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Volumn 36, Issue 10, 2014, Pages 979-990

Synthetic biology and therapeutic strategies for the degenerating brain: Synthetic biology approaches can transform classical cell and gene therapies, to provide new cures for neurodegenerative diseases

Author keywords

Artificial cell systems; Genome editing; Neurodegeneration; Synthetic proteins

Indexed keywords

MESSENGER RNA; NEUROTROPHIC FACTOR; NUCLEASE; PROTEIN; SYNTHETIC DNA; TRANSCRIPTION FACTOR;

EID: 84908244830     PISSN: 02659247     EISSN: 15211878     Source Type: Journal    
DOI: 10.1002/bies.201400094     Document Type: Article
Times cited : (20)

References (117)
  • 1
    • 84901308529 scopus 로고    scopus 로고
    • Brain-mapping projects to join forces
    • Reardon S. 2014. Brain-mapping projects to join forces. Nat News doi: 10.1038/nature.2014.14871
    • (2014) Nat News
    • Reardon, S.1
  • 2
    • 84884594132 scopus 로고    scopus 로고
    • Prevalence of adult Huntington's disease in the UK based on diagnoses recorded in general practice records
    • Evans SJ, Douglas I, Rawlins MD, Wexler NS, et al. 2013. Prevalence of adult Huntington's disease in the UK based on diagnoses recorded in general practice records. J Neurol Neurosurg Psychiatry 84: 1156-60.
    • (2013) J Neurol Neurosurg Psychiatry , vol.84 , pp. 1156-1160
    • Evans, S.J.1    Douglas, I.2    Rawlins, M.D.3    Wexler, N.S.4
  • 4
    • 79958234542 scopus 로고    scopus 로고
    • Depression and risk of developing dementia
    • Byers AL, Yaffe K. 2011. Depression and risk of developing dementia. Nat Rev Neurol 7: 323-31.
    • (2011) Nat Rev Neurol , vol.7 , pp. 323-331
    • Byers, A.L.1    Yaffe, K.2
  • 5
    • 80052376956 scopus 로고    scopus 로고
    • The size and burden of mental disorders and other disorders of the brain in Europe 2010
    • Wittchen HU, Jacobi F, Rehm J, Gustavsson A, et al. 2011. The size and burden of mental disorders and other disorders of the brain in Europe 2010. Eur Neuropsychopharmacol 21: 655-79.
    • (2011) Eur Neuropsychopharmacol , vol.21 , pp. 655-679
    • Wittchen, H.U.1    Jacobi, F.2    Rehm, J.3    Gustavsson, A.4
  • 6
    • 84878227058 scopus 로고    scopus 로고
    • BDNF-based synaptic repair as a disease-modifying strategy for neurodegenerative diseases
    • Lu B, Nagappan G, Guan X, Nathan PJ, et al. 2013. BDNF-based synaptic repair as a disease-modifying strategy for neurodegenerative diseases. Nat Rev Neurosci 14: 401-16.
    • (2013) Nat Rev Neurosci , vol.14 , pp. 401-416
    • Lu, B.1    Nagappan, G.2    Guan, X.3    Nathan, P.J.4
  • 8
    • 79957590886 scopus 로고    scopus 로고
    • Parkinson's disease: the quintessential neuropsychiatric disorder
    • Weintraub D, Burn DJ. 2011. Parkinson's disease: the quintessential neuropsychiatric disorder. Mov Disord 26: 1022-31.
    • (2011) Mov Disord , vol.26 , pp. 1022-1031
    • Weintraub, D.1    Burn, D.J.2
  • 9
    • 84898033416 scopus 로고    scopus 로고
    • The many questions on the use of biomarkers for neurodegenerative diseases in clinical practice
    • Menendez-Gonzalez M. 2014. The many questions on the use of biomarkers for neurodegenerative diseases in clinical practice. Front Aging Neurosci 6: 45.
    • (2014) Front Aging Neurosci , vol.6 , pp. 45
    • Menendez-Gonzalez, M.1
  • 10
    • 84863454666 scopus 로고    scopus 로고
    • Programmable single-cell mammalian biocomputers
    • Auslander S, Auslander D, Muller M, Wieland M, et al. 2012. Programmable single-cell mammalian biocomputers. Nature 487: 123-7.
    • (2012) Nature , vol.487 , pp. 123-127
    • Auslander, S.1    Auslander, D.2    Muller, M.3    Wieland, M.4
  • 11
    • 84868619337 scopus 로고    scopus 로고
    • Genetic programs constructed from layered logic gates in single cells
    • Moon TS, Lou C, Tamsir A, Stanton BC, et al. 2012. Genetic programs constructed from layered logic gates in single cells. Nature 491: 249-53.
    • (2012) Nature , vol.491 , pp. 249-253
    • Moon, T.S.1    Lou, C.2    Tamsir, A.3    Stanton, B.C.4
  • 12
    • 84868538087 scopus 로고    scopus 로고
    • Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice
    • Garriga-Canut M, Agustin-Pavon C, Herrmann F, Sanchez A, et al. 2012. Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice. Proc Natl Acad Sci USA 109: E3136-45.
    • (2012) Proc Natl Acad Sci USA , vol.109 , pp. E3136-E3145
    • Garriga-Canut, M.1    Agustin-Pavon, C.2    Herrmann, F.3    Sanchez, A.4
  • 13
    • 80052456127 scopus 로고    scopus 로고
    • Multi-input RNAi-based logic circuit for identification of specific cancer cells
    • Xie Z, Wroblewska L, Prochazka L, Weiss R, et al. 2011. Multi-input RNAi-based logic circuit for identification of specific cancer cells. Science 333: 1307-11.
    • (2011) Science , vol.333 , pp. 1307-1311
    • Xie, Z.1    Wroblewska, L.2    Prochazka, L.3    Weiss, R.4
  • 14
    • 84892765883 scopus 로고    scopus 로고
    • Genome-scale CRISPR-Cas9 knockout screening in human cells
    • Shalem O, Sanjana NE, Hartenian E, Shi X, et al. 2014. Genome-scale CRISPR-Cas9 knockout screening in human cells. Science 343: 84-7.
    • (2014) Science , vol.343 , pp. 84-87
    • Shalem, O.1    Sanjana, N.E.2    Hartenian, E.3    Shi, X.4
  • 15
    • 79551685675 scopus 로고    scopus 로고
    • A TALE nuclease architecture for efficient genome editing
    • Miller JC, Tan S, Qiao G, Barlow KA, et al. 2011. A TALE nuclease architecture for efficient genome editing. Nat Biotechnol 29: 143-8.
    • (2011) Nat Biotechnol , vol.29 , pp. 143-148
    • Miller, J.C.1    Tan, S.2    Qiao, G.3    Barlow, K.A.4
  • 16
    • 18944373328 scopus 로고    scopus 로고
    • Highly efficient endogenous human gene correction using designed zinc-finger nucleases
    • Urnov FD, Miller JC, Lee YL, Beausejour CM, et al. 2005. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435: 646-51.
    • (2005) Nature , vol.435 , pp. 646-651
    • Urnov, F.D.1    Miller, J.C.2    Lee, Y.L.3    Beausejour, C.M.4
  • 17
    • 84890170191 scopus 로고    scopus 로고
    • Synthetic biology: evolution or revolution? A co-founder's perspective
    • Gardner TS, Hawkins K. 2013. Synthetic biology: evolution or revolution? A co-founder's perspective. Curr Opin Chem Biol 17: 871-7.
    • (2013) Curr Opin Chem Biol , vol.17 , pp. 871-877
    • Gardner, T.S.1    Hawkins, K.2
  • 18
    • 17844374314 scopus 로고    scopus 로고
    • A synthetic multicellular system for programmed pattern formation
    • Basu S, Gerchman Y, Collins CH, Arnold FH, et al. 2005. A synthetic multicellular system for programmed pattern formation. Nature 434: 1130-4.
    • (2005) Nature , vol.434 , pp. 1130-1134
    • Basu, S.1    Gerchman, Y.2    Collins, C.H.3    Arnold, F.H.4
  • 19
    • 84900846464 scopus 로고    scopus 로고
    • Genetically encoded sender-receiver system in 3D mammalian cell culture
    • Carvalho A, Menendez DB, Senthivel VR, Zimmermann T, et al. 2014. Genetically encoded sender-receiver system in 3D mammalian cell culture. ACS Synth Biol 3: 264-72.
    • (2014) ACS Synth Biol , vol.3 , pp. 264-272
    • Carvalho, A.1    Menendez, D.B.2    Senthivel, V.R.3    Zimmermann, T.4
  • 20
    • 33645870422 scopus 로고    scopus 로고
    • Production of the antimalarial drug precursor artemisinic acid in engineered yeast
    • Ro DK, Paradise EM, Ouellet M, Fisher KJ, et al. 2006. Production of the antimalarial drug precursor artemisinic acid in engineered yeast. Nature 440: 940-3.
    • (2006) Nature , vol.440 , pp. 940-943
    • Ro, D.K.1    Paradise, E.M.2    Ouellet, M.3    Fisher, K.J.4
  • 21
    • 78651406505 scopus 로고    scopus 로고
    • Distributed biological computation with multicellular engineered networks
    • Regot S, Macia J, Conde N, Furukawa K, et al. 2011. Distributed biological computation with multicellular engineered networks. Nature 469: 207-11.
    • (2011) Nature , vol.469 , pp. 207-211
    • Regot, S.1    Macia, J.2    Conde, N.3    Furukawa, K.4
  • 23
    • 84865688581 scopus 로고    scopus 로고
    • Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression
    • Yu D, Pendergraff H, Liu J, Kordasiewicz HB, et al. 2012. Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression. Cell 150: 895-908.
    • (2012) Cell , vol.150 , pp. 895-908
    • Yu, D.1    Pendergraff, H.2    Liu, J.3    Kordasiewicz, H.B.4
  • 24
    • 84879086501 scopus 로고    scopus 로고
    • Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients
    • Bartus RT, Baumann TL, Siffert J, Herzog CD, et al. 2013. Safety/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients. Neurology 80: 1698-701.
    • (2013) Neurology , vol.80 , pp. 1698-1701
    • Bartus, R.T.1    Baumann, T.L.2    Siffert, J.3    Herzog, C.D.4
  • 25
    • 84897076939 scopus 로고    scopus 로고
    • Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial
    • Palfi S, Gurruchaga JM, Ralph GS, Lepetit H, et al. 2014. Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson's disease: a dose escalation, open-label, phase 1/2 trial. Lancet 383: 1138-46.
    • (2014) Lancet , vol.383 , pp. 1138-1146
    • Palfi, S.1    Gurruchaga, J.M.2    Ralph, G.S.3    Lepetit, H.4
  • 26
    • 84879873039 scopus 로고    scopus 로고
    • Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy
    • Biffi A, Montini E, Lorioli L, Cesani M, et al. 2013. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science 341: 1233158.
    • (2013) Science , vol.341 , pp. 1233158
    • Biffi, A.1    Montini, E.2    Lorioli, L.3    Cesani, M.4
  • 27
    • 78650031174 scopus 로고    scopus 로고
    • Huntington's disease: from molecular pathogenesis to clinical treatment
    • Ross CA, Tabrizi SJ. 2011. Huntington's disease: from molecular pathogenesis to clinical treatment. Lancet Neurol 10: 83-98.
    • (2011) Lancet Neurol , vol.10 , pp. 83-98
    • Ross, C.A.1    Tabrizi, S.J.2
  • 28
    • 84867403458 scopus 로고    scopus 로고
    • Increased encapsulated cell biodelivery of nerve growth factor in the brain by transposon-mediated gene transfer
    • Fjord-Larsen L, Kusk P, Emerich DF, Thanos C, et al. 2012. Increased encapsulated cell biodelivery of nerve growth factor in the brain by transposon-mediated gene transfer. Gene Ther 19: 1010-7.
    • (2012) Gene Ther , vol.19 , pp. 1010-1017
    • Fjord-Larsen, L.1    Kusk, P.2    Emerich, D.F.3    Thanos, C.4
  • 29
    • 84864530993 scopus 로고    scopus 로고
    • Targeted delivery of nerve growth factor via encapsulated cell biodelivery in Alzheimer disease: a technology platform for restorative neurosurgery
    • Wahlberg LU, Lind G, Almqvist PM, Kusk P, et al. 2012. Targeted delivery of nerve growth factor via encapsulated cell biodelivery in Alzheimer disease: a technology platform for restorative neurosurgery. J Neurosurg 117: 340-7.
    • (2012) J Neurosurg , vol.117 , pp. 340-347
    • Wahlberg, L.U.1    Lind, G.2    Almqvist, P.M.3    Kusk, P.4
  • 30
    • 0033613487 scopus 로고    scopus 로고
    • The biotech death of Jesse Gelsinger
    • Stolberg SG. 1999. The biotech death of Jesse Gelsinger. NY Times Mag 136-40: 49-50.
    • (1999) NY Times Mag , vol.136-140 , pp. 49-50
    • Stolberg, S.G.1
  • 31
    • 84895546715 scopus 로고    scopus 로고
    • Pre-clinical assessment of immune responses to adeno-associated virus (AAV) vectors
    • Basner-Tschakarjan E, Bijjiga E, Martino AT. 2014. Pre-clinical assessment of immune responses to adeno-associated virus (AAV) vectors. Front Immunol 5: 28.
    • (2014) Front Immunol , vol.5 , pp. 28
    • Basner-Tschakarjan, E.1    Bijjiga, E.2    Martino, A.T.3
  • 32
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
    • Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, et al. 2008. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 118: 3143-50.
    • (2008) J Clin Invest , vol.118 , pp. 3143-3150
    • Howe, S.J.1    Mansour, M.R.2    Schwarzwaelder, K.3    Bartholomae, C.4
  • 33
    • 84882261767 scopus 로고    scopus 로고
    • Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy
    • Papayannakos C, Daniel R. 2013. Understanding lentiviral vector chromatin targeting: working to reduce insertional mutagenic potential for gene therapy. Gene Ther 20: 581-8.
    • (2013) Gene Ther , vol.20 , pp. 581-588
    • Papayannakos, C.1    Daniel, R.2
  • 34
    • 84871922836 scopus 로고    scopus 로고
    • Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses
    • Ciesielska A, Hadaczek P, Mittermeyer G, Zhou S, et al. 2013. Cerebral infusion of AAV9 vector-encoding non-self proteins can elicit cell-mediated immune responses. Mol Ther 21: 158-66.
    • (2013) Mol Ther , vol.21 , pp. 158-166
    • Ciesielska, A.1    Hadaczek, P.2    Mittermeyer, G.3    Zhou, S.4
  • 35
    • 62549094869 scopus 로고    scopus 로고
    • Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response
    • Hadaczek P, Forsayeth J, Mirek H, Munson K, et al. 2009. Transduction of nonhuman primate brain with adeno-associated virus serotype 1: vector trafficking and immune response. Hum Gene Ther 20: 225-37.
    • (2009) Hum Gene Ther , vol.20 , pp. 225-237
    • Hadaczek, P.1    Forsayeth, J.2    Mirek, H.3    Munson, K.4
  • 36
    • 84893307092 scopus 로고    scopus 로고
    • AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction
    • Samaranch L, San Sebastian W, Kells AP, Salegio EA, et al. 2014. AAV9-mediated expression of a non-self protein in nonhuman primate central nervous system triggers widespread neuroinflammation driven by antigen-presenting cell transduction. Mol Ther 22: 329-37.
    • (2014) Mol Ther , vol.22 , pp. 329-337
    • Samaranch, L.1    San Sebastian, W.2    Kells, A.P.3    Salegio, E.A.4
  • 37
  • 38
    • 84874616818 scopus 로고    scopus 로고
    • Synergistic and tunable human gene activation by combinations of synthetic transcription factors
    • Perez-Pinera P, Ousterout DG, Brunger JM, Farin AM, et al. 2013. Synergistic and tunable human gene activation by combinations of synthetic transcription factors. Nat Methods 10: 239-42.
    • (2013) Nat Methods , vol.10 , pp. 239-242
    • Perez-Pinera, P.1    Ousterout, D.G.2    Brunger, J.M.3    Farin, A.M.4
  • 39
    • 84880571335 scopus 로고    scopus 로고
    • CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes
    • Gilbert LA, Larson MH, Morsut L, Liu Z, et al. 2013. CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes. Cell 154: 442-51.
    • (2013) Cell , vol.154 , pp. 442-451
    • Gilbert, L.A.1    Larson, M.H.2    Morsut, L.3    Liu, Z.4
  • 40
    • 84884907424 scopus 로고    scopus 로고
    • CRISPR RNA-guided activation of endogenous human genes
    • Maeder ML, Linder SJ, Cascio VM, Fu Y, et al. 2013. CRISPR RNA-guided activation of endogenous human genes. Nat Methods 10: 977-9.
    • (2013) Nat Methods , vol.10 , pp. 977-979
    • Maeder, M.L.1    Linder, S.J.2    Cascio, V.M.3    Fu, Y.4
  • 41
    • 84862663712 scopus 로고    scopus 로고
    • Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis
    • Kordasiewicz HB, Stanek LM, Wancewicz EV, Mazur C, et al. 2012. Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron 74: 1031-44.
    • (2012) Neuron , vol.74 , pp. 1031-1044
    • Kordasiewicz, H.B.1    Stanek, L.M.2    Wancewicz, E.V.3    Mazur, C.4
  • 42
    • 32044445564 scopus 로고    scopus 로고
    • Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron
    • Singh NK, Singh NN, Androphy EJ, Singh RN. 2006. Splicing of a critical exon of human survival motor neuron is regulated by a unique silencer element located in the last intron. Mol Cell Biol 26: 1333-46.
    • (2006) Mol Cell Biol , vol.26 , pp. 1333-1346
    • Singh, N.K.1    Singh, N.N.2    Androphy, E.J.3    Singh, R.N.4
  • 43
    • 41549168514 scopus 로고    scopus 로고
    • Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice
    • Hua Y, Vickers TA, Okunola HL, Bennett CF. et al. 2008. Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice. Am J Hum Genet 82: 834-48.
    • (2008) Am J Hum Genet , vol.82 , pp. 834-848
    • Hua, Y.1    Vickers, T.A.2    Okunola, H.L.3    Bennett, C.F.4
  • 44
    • 67449135902 scopus 로고    scopus 로고
    • Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy
    • Williams JH, Schray RC, Patterson CA, Ayitey SO. et al. 2009. Oligonucleotide-mediated survival of motor neuron protein expression in CNS improves phenotype in a mouse model of spinal muscular atrophy. J Neurosci 29: 7633-8.
    • (2009) J Neurosci , vol.29 , pp. 7633-7638
    • Williams, J.H.1    Schray, R.C.2    Patterson, C.A.3    Ayitey, S.O.4
  • 45
    • 84892373557 scopus 로고    scopus 로고
    • Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials
    • Zanetta C, Nizzardo M, Simone C, Monguzzi E, et al. 2014. Molecular therapeutic strategies for spinal muscular atrophies: current and future clinical trials. Clin Ther 36: 128-40.
    • (2014) Clin Ther , vol.36 , pp. 128-140
    • Zanetta, C.1    Nizzardo, M.2    Simone, C.3    Monguzzi, E.4
  • 46
    • 84898015288 scopus 로고    scopus 로고
    • Molecular mechanisms of long ncRNAs in neurological disorders
    • Vucicevic D, Schrewe H, Orom UA. 2014. Molecular mechanisms of long ncRNAs in neurological disorders. Front Genet 5: 48.
    • (2014) Front Genet , vol.5 , pp. 48
    • Vucicevic, D.1    Schrewe, H.2    Orom, U.A.3
  • 47
    • 84936770369 scopus 로고    scopus 로고
    • Antisense oligonucleotide therapy for the treatment of C9ORF72 ALS/FTD diseases
    • in press
    • Riboldi G, Zanetta C, Ranieri M, Nizzardo M, et al. 2014. Antisense oligonucleotide therapy for the treatment of C9ORF72 ALS/FTD diseases. Mol Neurobiol in press, doi: 10.1007/s12035-014-8724-7
    • (2014) Mol Neurobiol
    • Riboldi, G.1    Zanetta, C.2    Ranieri, M.3    Nizzardo, M.4
  • 48
    • 79960148982 scopus 로고    scopus 로고
    • Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design
    • Martin JN, Wolken N, Brown T, Dauer WT, et al. 2011. Lethal toxicity caused by expression of shRNA in the mouse striatum: implications for therapeutic design. Gene Ther 18: 666-73.
    • (2011) Gene Ther , vol.18 , pp. 666-673
    • Martin, J.N.1    Wolken, N.2    Brown, T.3    Dauer, W.T.4
  • 49
    • 79957923807 scopus 로고    scopus 로고
    • An alpha-synuclein AAV gene silencing vector ameliorates a behavioral deficit in a rat model of Parkinson's disease, but displays toxicity in dopamine neurons
    • Khodr CE, Sapru MK, Pedapati J, Han Y, et al. 2011. An alpha-synuclein AAV gene silencing vector ameliorates a behavioral deficit in a rat model of Parkinson's disease, but displays toxicity in dopamine neurons. Brain Res 1395: 94-107.
    • (2011) Brain Res , vol.1395 , pp. 94-107
    • Khodr, C.E.1    Sapru, M.K.2    Pedapati, J.3    Han, Y.4
  • 50
    • 77949446144 scopus 로고    scopus 로고
    • Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system
    • Ehlert EM, Eggers R, Niclou SP, Verhaagen J. 2010. Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system. BMC Neurosci 11: 20.
    • (2010) BMC Neurosci , vol.11 , pp. 20
    • Ehlert, E.M.1    Eggers, R.2    Niclou, S.P.3    Verhaagen, J.4
  • 51
    • 44449121785 scopus 로고    scopus 로고
    • Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi
    • McBride JL, Boudreau RL, Harper SQ, Staber PD, et al. 2008. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci USA 105: 5868-73.
    • (2008) Proc Natl Acad Sci USA , vol.105 , pp. 5868-5873
    • McBride, J.L.1    Boudreau, R.L.2    Harper, S.Q.3    Staber, P.D.4
  • 52
    • 84860013215 scopus 로고    scopus 로고
    • Synthetic genetic polymers capable of heredity and evolution
    • Pinheiro VB, Taylor AI, Cozens C, Abramov M, et al. 2012. Synthetic genetic polymers capable of heredity and evolution. Science 336: 341-4.
    • (2012) Science , vol.336 , pp. 341-344
    • Pinheiro, V.B.1    Taylor, A.I.2    Cozens, C.3    Abramov, M.4
  • 53
    • 78650739853 scopus 로고    scopus 로고
    • Regulation of selected genome loci using de novo-engineered transcription activator-like effector (TALE)-type transcription factors
    • Morbitzer R, Romer P, Boch J, Lahaye T. 2010. Regulation of selected genome loci using de novo-engineered transcription activator-like effector (TALE)-type transcription factors. Proc Natl Acad Sci USA 107: 21617-22.
    • (2010) Proc Natl Acad Sci USA , vol.107 , pp. 21617-21622
    • Morbitzer, R.1    Romer, P.2    Boch, J.3    Lahaye, T.4
  • 54
    • 84898998655 scopus 로고    scopus 로고
    • Direct activation of human and mouse Oct4 genes using engineered TALE and Cas9 transcription factors
    • Hu J, Lei Y, Wong WK, Liu S, et al. 2014. Direct activation of human and mouse Oct4 genes using engineered TALE and Cas9 transcription factors. Nucleic Acids Res 42: 4375-90.
    • (2014) Nucleic Acids Res , vol.42 , pp. 4375-4390
    • Hu, J.1    Lei, Y.2    Wong, W.K.3    Liu, S.4
  • 55
    • 79952097561 scopus 로고    scopus 로고
    • Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa
    • Mussolino C, Sanges D, Marrocco E, Bonetti C, et al. 2011. Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa. EMBO Mol Med 3: 118-28.
    • (2011) EMBO Mol Med , vol.3 , pp. 118-128
    • Mussolino, C.1    Sanges, D.2    Marrocco, E.3    Bonetti, C.4
  • 56
    • 80053972328 scopus 로고    scopus 로고
    • Intramuscular administration of a VEGF zinc finger transcription factor activator (VEGF-ZFP-TF) improves functional outcomes in SOD1 rats
    • Kliem MA, Heeke BL, Franz CK, Radovitskiy I, et al. 2011. Intramuscular administration of a VEGF zinc finger transcription factor activator (VEGF-ZFP-TF) improves functional outcomes in SOD1 rats. Amyotroph Lateral Scler 12: 331-9.
    • (2011) Amyotroph Lateral Scler , vol.12 , pp. 331-339
    • Kliem, M.A.1    Heeke, B.L.2    Franz, C.K.3    Radovitskiy, I.4
  • 57
    • 0036912846 scopus 로고    scopus 로고
    • Induction of angiogenesis in a mouse model using engineered transcription factors
    • Rebar EJ, Huang Y, Hickey R, Nath AK, et al. 2002. Induction of angiogenesis in a mouse model using engineered transcription factors. Nat Med 8: 1427-32.
    • (2002) Nat Med , vol.8 , pp. 1427-1432
    • Rebar, E.J.1    Huang, Y.2    Hickey, R.3    Nath, A.K.4
  • 58
    • 78650044247 scopus 로고    scopus 로고
    • An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease
    • Laganiere J, Kells AP, Lai JT, Guschin D, et al. 2010. An engineered zinc finger protein activator of the endogenous glial cell line-derived neurotrophic factor gene provides functional neuroprotection in a rat model of Parkinson's disease. J Neurosci 30: 16469-74.
    • (2010) J Neurosci , vol.30 , pp. 16469-16474
    • Laganiere, J.1    Kells, A.P.2    Lai, J.T.3    Guschin, D.4
  • 59
    • 84884997788 scopus 로고    scopus 로고
    • Dose-dependent neuroprotection of VEGF(1)(6)(5) in Huntington's disease striatum
    • Ellison SM, Trabalza A, Tisato V, Pazarentzos E, et al. 2013. Dose-dependent neuroprotection of VEGF(1)(6)(5) in Huntington's disease striatum. Mol Ther 21: 1862-75.
    • (2013) Mol Ther , vol.21 , pp. 1862-1875
    • Ellison, S.M.1    Trabalza, A.2    Tisato, V.3    Pazarentzos, E.4
  • 60
    • 78951479577 scopus 로고    scopus 로고
    • Targeting DNA double-strand breaks with TAL effector nucleases
    • Christian M, Cermak T, Doyle EL, Schmidt C, et al. 2010. Targeting DNA double-strand breaks with TAL effector nucleases. Genetics 186: 757-61.
    • (2010) Genetics , vol.186 , pp. 757-761
    • Christian, M.1    Cermak, T.2    Doyle, E.L.3    Schmidt, C.4
  • 61
    • 84873734105 scopus 로고    scopus 로고
    • RNA-guided human genome engineering via Cas9
    • Mali P, Yang L, Esvelt KM, Aach J, et al. 2013. RNA-guided human genome engineering via Cas9. Science 339: 823-6.
    • (2013) Science , vol.339 , pp. 823-826
    • Mali, P.1    Yang, L.2    Esvelt, K.M.3    Aach, J.4
  • 62
    • 84894460947 scopus 로고    scopus 로고
    • A cut above the rest: targeted genome editing technologies in human pluripotent stem cells
    • Li M, Suzuki K, Kim NY, Liu GH, et al. 2014. A cut above the rest: targeted genome editing technologies in human pluripotent stem cells. J Biol Chem 289: 4594-9.
    • (2014) J Biol Chem , vol.289 , pp. 4594-4599
    • Li, M.1    Suzuki, K.2    Kim, N.Y.3    Liu, G.H.4
  • 63
    • 84895487305 scopus 로고    scopus 로고
    • Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV
    • Tebas P, Stein D, Tang WW, Frank I, et al. 2014. Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV. N Engl J Med 370: 901-10.
    • (2014) N Engl J Med , vol.370 , pp. 901-910
    • Tebas, P.1    Stein, D.2    Tang, W.W.3    Frank, I.4
  • 64
    • 84880570576 scopus 로고    scopus 로고
    • High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells
    • Fu Y, Foden JA, Khayter C, Maeder ML, et al. 2013. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Nat Biotechnol 31: 822-6.
    • (2013) Nat Biotechnol , vol.31 , pp. 822-826
    • Fu, Y.1    Foden, J.A.2    Khayter, C.3    Maeder, M.L.4
  • 65
    • 84861984839 scopus 로고    scopus 로고
    • Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme
    • Wang J, Friedman G, Doyon Y, Wang NS, et al. 2012. Targeted gene addition to a predetermined site in the human genome using a ZFN-based nicking enzyme. Genome Res 22: 1316-26.
    • (2012) Genome Res , vol.22 , pp. 1316-1326
    • Wang, J.1    Friedman, G.2    Doyon, Y.3    Wang, N.S.4
  • 66
    • 84884288934 scopus 로고    scopus 로고
    • Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
    • Ran FA, Hsu PD, Lin CY, Gootenberg JS, et al. 2013. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell 154: 1380-9.
    • (2013) Cell , vol.154 , pp. 1380-1389
    • Ran, F.A.1    Hsu, P.D.2    Lin, C.Y.3    Gootenberg, J.S.4
  • 67
    • 84884165315 scopus 로고    scopus 로고
    • DNA targeting specificity of RNA-guided Cas9 nucleases
    • Hsu PD, Scott DA, Weinstein JA, Ran FA, et al. 2013. DNA targeting specificity of RNA-guided Cas9 nucleases. Nat Biotechnol 31: 827-32.
    • (2013) Nat Biotechnol , vol.31 , pp. 827-832
    • Hsu, P.D.1    Scott, D.A.2    Weinstein, J.A.3    Ran, F.A.4
  • 68
    • 84882976110 scopus 로고    scopus 로고
    • Optical control of mammalian endogenous transcription and epigenetic states
    • Konermann S, Brigham MD, Trevino AE, Hsu PD, et al. 2013. Optical control of mammalian endogenous transcription and epigenetic states. Nature 500: 472-6.
    • (2013) Nature , vol.500 , pp. 472-476
    • Konermann, S.1    Brigham, M.D.2    Trevino, A.E.3    Hsu, P.D.4
  • 69
    • 84893642253 scopus 로고    scopus 로고
    • Cell-to-cell transmission of pathogenic proteins in neurodegenerative diseases
    • Guo JL, Lee VM. 2014. Cell-to-cell transmission of pathogenic proteins in neurodegenerative diseases. Nat Med 20: 130-8.
    • (2014) Nat Med , vol.20 , pp. 130-138
    • Guo, J.L.1    Lee, V.M.2
  • 70
    • 84885783467 scopus 로고    scopus 로고
    • Anti-tau antibodies that block tau aggregate seeding in vitro markedly decrease pathology and improve cognition in vivo
    • Yanamandra K, Kfoury N, Jiang H, Mahan TE, et al. 2013. Anti-tau antibodies that block tau aggregate seeding in vitro markedly decrease pathology and improve cognition in vivo. Neuron 80: 402-14.
    • (2013) Neuron , vol.80 , pp. 402-414
    • Yanamandra, K.1    Kfoury, N.2    Jiang, H.3    Mahan, T.E.4
  • 71
    • 84894033757 scopus 로고    scopus 로고
    • Is there still any hope for amyloid-based immunotherapy for Alzheimer's disease
    • Panza F, Logroscino G, Imbimbo BP, Solfrizzi V. 2014. Is there still any hope for amyloid-based immunotherapy for Alzheimer's disease? Curr Opin Psychiatry 27: 128-37.
    • (2014) Curr Opin Psychiatry , vol.27 , pp. 128-137
    • Panza, F.1    Logroscino, G.2    Imbimbo, B.P.3    Solfrizzi, V.4
  • 73
    • 84897450856 scopus 로고    scopus 로고
    • Kaleidoscopic protein-protein interactions in the life and death of ataxin-1: new strategies against protein aggregation
    • de Chiara C, Pastore A. 2014. Kaleidoscopic protein-protein interactions in the life and death of ataxin-1: new strategies against protein aggregation. Trends Neurosci 37: 211-8.
    • (2014) Trends Neurosci , vol.37 , pp. 211-218
    • de Chiara, C.1    Pastore, A.2
  • 74
    • 77749319356 scopus 로고    scopus 로고
    • Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein
    • Bauer PO, Goswami A, Wong HK, Okuno M, et al. 2010. Harnessing chaperone-mediated autophagy for the selective degradation of mutant huntingtin protein. Nat Biotechnol 28: 256-63.
    • (2010) Nat Biotechnol , vol.28 , pp. 256-263
    • Bauer, P.O.1    Goswami, A.2    Wong, H.K.3    Okuno, M.4
  • 75
    • 2942755950 scopus 로고    scopus 로고
    • The hybrid cytomegalovirus enhancer/chicken beta-actin promoter along with woodchuck hepatitis virus posttranscriptional regulatory element enhances the protective efficacy of DNA vaccines
    • Garg S, Oran AE, Hon H, Jacob J. 2004. The hybrid cytomegalovirus enhancer/chicken beta-actin promoter along with woodchuck hepatitis virus posttranscriptional regulatory element enhances the protective efficacy of DNA vaccines. J Immunol 173: 550-8.
    • (2004) J Immunol , vol.173 , pp. 550-558
    • Garg, S.1    Oran, A.E.2    Hon, H.3    Jacob, J.4
  • 76
    • 84872241791 scopus 로고    scopus 로고
    • Restricted transgene expression in the brain with cell-type specific neuronal promoters
    • Delzor A, Dufour N, Petit F, Guillermier M, et al. 2012. Restricted transgene expression in the brain with cell-type specific neuronal promoters. Hum Gene Ther Meth 23: 242-54.
    • (2012) Hum Gene Ther Meth , vol.23 , pp. 242-254
    • Delzor, A.1    Dufour, N.2    Petit, F.3    Guillermier, M.4
  • 77
    • 78049259570 scopus 로고    scopus 로고
    • A regulatory toolbox of MiniPromoters to drive selective expression in the brain
    • Portales-Casamar E, Swanson DJ, Liu L, de Leeuw CN, et al. 2010. A regulatory toolbox of MiniPromoters to drive selective expression in the brain. Proc Natl Acad Sci USA 107: 16589-94.
    • (2010) Proc Natl Acad Sci USA , vol.107 , pp. 16589-16594
    • Portales-Casamar, E.1    Swanson, D.J.2    Liu, L.3    de Leeuw, C.N.4
  • 78
    • 66149143984 scopus 로고    scopus 로고
    • Measuring the activity of BioBrick promoters using an in vivo reference standard
    • Kelly JR, Rubin AJ, Davis JH, Ajo-Franklin CM, et al. 2009. Measuring the activity of BioBrick promoters using an in vivo reference standard. J Biol Eng 3: 4.
    • (2009) J Biol Eng , vol.3 , pp. 4
    • Kelly, J.R.1    Rubin, A.J.2    Davis, J.H.3    Ajo-Franklin, C.M.4
  • 79
    • 0038664127 scopus 로고    scopus 로고
    • Identification and functional analysis of human transcriptional promoters
    • Trinklein ND, Aldred SJ, Saldanha AJ, Myers RM. 2003. Identification and functional analysis of human transcriptional promoters. Genome Res 13: 308-12.
    • (2003) Genome Res , vol.13 , pp. 308-312
    • Trinklein, N.D.1    Aldred, S.J.2    Saldanha, A.J.3    Myers, R.M.4
  • 80
    • 84895922623 scopus 로고    scopus 로고
    • Global clinical response in Cushing's syndrome patients treated with mifepristone
    • Katznelson L, Loriaux DL, Feldman D, Braunstein GD, et al. 2014. Global clinical response in Cushing's syndrome patients treated with mifepristone. Clin Endocrinol (Oxf) 80: 562-9.
    • (2014) Clin Endocrinol (Oxf) , vol.80 , pp. 562-569
    • Katznelson, L.1    Loriaux, D.L.2    Feldman, D.3    Braunstein, G.D.4
  • 81
    • 84880390816 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated, mifepristone-regulated transgene expression in the brain
    • Maddalena A, Tereshchenko J, Bahr M, Kugler S. 2013. Adeno-associated virus-mediated, mifepristone-regulated transgene expression in the brain. Mol Ther Nucleic Acids 2: e106.
    • (2013) Mol Ther Nucleic Acids , vol.2 , pp. e106
    • Maddalena, A.1    Tereshchenko, J.2    Bahr, M.3    Kugler, S.4
  • 82
    • 84872001455 scopus 로고    scopus 로고
    • Pharmaceutically controlled designer circuit for the treatment of the metabolic syndrome
    • Ye H, Charpin-El Hamri G, Zwicky K, Christen M, et al. 2013. Pharmaceutically controlled designer circuit for the treatment of the metabolic syndrome. Proc Natl Acad Sci USA 110: 141-6.
    • (2013) Proc Natl Acad Sci USA , vol.110 , pp. 141-146
    • Ye, H.1    Charpin-El Hamri, G.2    Zwicky, K.3    Christen, M.4
  • 83
    • 84884655331 scopus 로고    scopus 로고
    • Targeting neurons and photons for optogenetics
    • Packer AM, Roska B, Hausser M. 2013. Targeting neurons and photons for optogenetics. Nat Neurosci 16: 805-15.
    • (2013) Nat Neurosci , vol.16 , pp. 805-815
    • Packer, A.M.1    Roska, B.2    Hausser, M.3
  • 84
    • 84867370333 scopus 로고    scopus 로고
    • Light-inducible spatiotemporal control of gene activation by customizable zinc finger transcription factors
    • Polstein LR, Gersbach CA. 2012. Light-inducible spatiotemporal control of gene activation by customizable zinc finger transcription factors. J Am Chem Soc 134: 16480-3.
    • (2012) J Am Chem Soc , vol.134 , pp. 16480-16483
    • Polstein, L.R.1    Gersbach, C.A.2
  • 85
    • 84901946956 scopus 로고    scopus 로고
    • Parkinson's disease: recent advances
    • Katzenschlager R. 2014. Parkinson's disease: recent advances. J Neurol 261: 1031-6.
    • (2014) J Neurol , vol.261 , pp. 1031-1036
    • Katzenschlager, R.1
  • 86
    • 84869795204 scopus 로고    scopus 로고
    • Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy
    • 161ra52
    • Wykes RC, Heeroma JH, Mantoan L, Zheng K, et al. 2012. Optogenetic and potassium channel gene therapy in a rodent model of focal neocortical epilepsy. Sci Transl Med 4: 161ra52.
    • (2012) Sci Transl Med , vol.4
    • Wykes, R.C.1    Heeroma, J.H.2    Mantoan, L.3    Zheng, K.4
  • 87
    • 78649363586 scopus 로고    scopus 로고
    • An engineered mammalian band-pass network
    • Greber D, Fussenegger M. 2010. An engineered mammalian band-pass network. Nucleic Acids Res 38: e174.
    • (2010) Nucleic Acids Res , vol.38 , pp. e174
    • Greber, D.1    Fussenegger, M.2
  • 88
    • 78650633687 scopus 로고    scopus 로고
    • A tunable dual-promoter integrator for targeting of cancer cells
    • Nissim L, Bar-Ziv RH. 2010. A tunable dual-promoter integrator for targeting of cancer cells. Mol Syst Biol 6: 444.
    • (2010) Mol Syst Biol , vol.6 , pp. 444
    • Nissim, L.1    Bar-Ziv, R.H.2
  • 89
    • 84887315800 scopus 로고    scopus 로고
    • Reward-based hypertension control by a synthetic brain-dopamine interface
    • Rossger K, Charpin-El Hamri G, Fussenegger M. 2013. Reward-based hypertension control by a synthetic brain-dopamine interface. Proc Natl Acad Sci USA 110: 18150-5.
    • (2013) Proc Natl Acad Sci USA , vol.110 , pp. 18150-18155
    • Rossger, K.1    Charpin-El Hamri, G.2    Fussenegger, M.3
  • 90
    • 84898538354 scopus 로고    scopus 로고
    • Encapsulated cell therapy for neurodegenerative diseases: from promise to product
    • Emerich DF, Orive G, Thanos C, Tornoe J, et al. 2013. Encapsulated cell therapy for neurodegenerative diseases: from promise to product. Adv Drug Deliv Rev 67-68: 131-41.
    • (2013) Adv Drug Deliv Rev , vol.67-68 , pp. 131-141
    • Emerich, D.F.1    Orive, G.2    Thanos, C.3    Tornoe, J.4
  • 91
    • 84868631248 scopus 로고    scopus 로고
    • The mysterious motivational functions of mesolimbic dopamine
    • Salamone JD, Correa M. 2012. The mysterious motivational functions of mesolimbic dopamine. Neuron 76: 470-85.
    • (2012) Neuron , vol.76 , pp. 470-485
    • Salamone, J.D.1    Correa, M.2
  • 93
    • 84898447902 scopus 로고    scopus 로고
    • Plasma phospholipids identify antecedent memory impairment in older adults
    • Mapstone M, Cheema AK, Fiandaca MS, Zhong X, et al. 2014. Plasma phospholipids identify antecedent memory impairment in older adults. Nat Med 20: 415-8.
    • (2014) Nat Med , vol.20 , pp. 415-418
    • Mapstone, M.1    Cheema, A.K.2    Fiandaca, M.S.3    Zhong, X.4
  • 94
    • 84899525577 scopus 로고    scopus 로고
    • Astrocyte Kir4.1 ion channel deficits contribute to neuronal dysfunction in Huntington's disease model mice
    • Tong X, Ao Y, Faas GC, Nwaobi SE, et al. 2014. Astrocyte Kir4.1 ion channel deficits contribute to neuronal dysfunction in Huntington's disease model mice. Nat Neurosci 17: 694-703.
    • (2014) Nat Neurosci , vol.17 , pp. 694-703
    • Tong, X.1    Ao, Y.2    Faas, G.C.3    Nwaobi, S.E.4
  • 95
    • 84908256835 scopus 로고    scopus 로고
    • Is KIR4.1 antibody a marker of multiple sclerosis
    • Supplement P4.130
    • Nerrant E, Charif M, Ayrignac X, Carra-Dalliere C, et al. 2014. Is KIR4.1 antibody a marker of multiple sclerosis? Neurology 82: Supplement P4.130.
    • (2014) Neurology , vol.82
    • Nerrant, E.1    Charif, M.2    Ayrignac, X.3    Carra-Dalliere, C.4
  • 96
    • 79960213497 scopus 로고    scopus 로고
    • Detection of multiple disease indicators by an autonomous biomolecular computer
    • Gil B, Kahan-Hanum M, Skirtenko N, Adar R, et al. 2011. Detection of multiple disease indicators by an autonomous biomolecular computer. Nano Lett 11: 2989-96.
    • (2011) Nano Lett , vol.11 , pp. 2989-2996
    • Gil, B.1    Kahan-Hanum, M.2    Skirtenko, N.3    Adar, R.4
  • 97
    • 84879205624 scopus 로고    scopus 로고
    • Aging and neurogenesis in the adult forebrain: what we have learned and where we should go from here
    • Hamilton LK, Joppe SE, Cochard LM, Fernandes KJ. 2013. Aging and neurogenesis in the adult forebrain: what we have learned and where we should go from here. Eur J Neurosci 37: 1978-86.
    • (2013) Eur J Neurosci , vol.37 , pp. 1978-1986
    • Hamilton, L.K.1    Joppe, S.E.2    Cochard, L.M.3    Fernandes, K.J.4
  • 98
    • 84896732922 scopus 로고    scopus 로고
    • Neurogenesis in the striatum of the adult human brain
    • Ernst A, Alkass K, Bernard S, Salehpour M, et al. 2014. Neurogenesis in the striatum of the adult human brain. Cell 156: 1072-83.
    • (2014) Cell , vol.156 , pp. 1072-1083
    • Ernst, A.1    Alkass, K.2    Bernard, S.3    Salehpour, M.4
  • 101
    • 79960833952 scopus 로고    scopus 로고
    • Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations
    • Soldner F, Laganiere J, Cheng AW, Hockemeyer D, et al. 2011. Generation of isogenic pluripotent stem cells differing exclusively at two early onset Parkinson point mutations. Cell 146: 318-31.
    • (2011) Cell , vol.146 , pp. 318-331
    • Soldner, F.1    Laganiere, J.2    Cheng, A.W.3    Hockemeyer, D.4
  • 102
    • 84883857778 scopus 로고    scopus 로고
    • Genetic correction of tauopathy phenotypes in neurons derived from human induced pluripotent stem cells
    • Fong H, Wang C, Knoferle J, Walker D, et al. 2013. Genetic correction of tauopathy phenotypes in neurons derived from human induced pluripotent stem cells. Stem Cell Rep 1: 226-34.
    • (2013) Stem Cell Rep , vol.1 , pp. 226-234
    • Fong, H.1    Wang, C.2    Knoferle, J.3    Walker, D.4
  • 103
    • 84862551266 scopus 로고    scopus 로고
    • Modeling brain disease in a dish: really
    • Marchetto MC, Gage FH. 2012. Modeling brain disease in a dish: really? Cell Stem Cell 10: 642-5.
    • (2012) Cell Stem Cell , vol.10 , pp. 642-645
    • Marchetto, M.C.1    Gage, F.H.2
  • 104
    • 84889855901 scopus 로고    scopus 로고
    • One-step generation of different immunodeficient mice with multiple gene modifications by CRISPR/Cas9 mediated genome engineering
    • Zhou J, Shen B, Zhang W, Wang J, et al. 2014. One-step generation of different immunodeficient mice with multiple gene modifications by CRISPR/Cas9 mediated genome engineering. Int J Biochem Cell Biol 46: 49-55.
    • (2014) Int J Biochem Cell Biol , vol.46 , pp. 49-55
    • Zhou, J.1    Shen, B.2    Zhang, W.3    Wang, J.4
  • 105
    • 84874617789 scopus 로고    scopus 로고
    • Efficient genome editing in zebrafish using a CRISPR-Cas system
    • Hwang WY, Fu Y, Reyon D, Maeder ML, et al. 2013. Efficient genome editing in zebrafish using a CRISPR-Cas system. Nat Biotechnol 31: 227-9.
    • (2013) Nat Biotechnol , vol.31 , pp. 227-229
    • Hwang, W.Y.1    Fu, Y.2    Reyon, D.3    Maeder, M.L.4
  • 106
    • 84894081986 scopus 로고    scopus 로고
    • Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos
    • Niu Y, Shen B, Cui Y, Chen Y, et al. 2014. Generation of gene-modified cynomolgus monkey via Cas9/RNA-mediated gene targeting in one-cell embryos. Cell 156: 836-43.
    • (2014) Cell , vol.156 , pp. 836-843
    • Niu, Y.1    Shen, B.2    Cui, Y.3    Chen, Y.4
  • 107
    • 84896142186 scopus 로고    scopus 로고
    • TALEN-mediated gene mutagenesis in rhesus and cynomolgus monkeys
    • Liu H, Chen Y, Niu Y, Zhang K, et al. 2014. TALEN-mediated gene mutagenesis in rhesus and cynomolgus monkeys. Cell Stem Cell 14: 323-8.
    • (2014) Cell Stem Cell , vol.14 , pp. 323-328
    • Liu, H.1    Chen, Y.2    Niu, Y.3    Zhang, K.4
  • 108
    • 84864288917 scopus 로고    scopus 로고
    • Lesions of ventrolateral prefrontal or anterior orbitofrontal cortex in primates heighten negative emotion
    • Agustin-Pavon C, Braesicke K, Shiba Y, Santangelo AM, et al. 2012. Lesions of ventrolateral prefrontal or anterior orbitofrontal cortex in primates heighten negative emotion. Biol Psychiatry 72: 266-72.
    • (2012) Biol Psychiatry , vol.72 , pp. 266-272
    • Agustin-Pavon, C.1    Braesicke, K.2    Shiba, Y.3    Santangelo, A.M.4
  • 109
    • 84892681247 scopus 로고    scopus 로고
    • Common marmoset as a new model animal for neuroscience research and genome editing technology
    • Kishi N, Sato K, Sasaki E, Okano H. 2014. Common marmoset as a new model animal for neuroscience research and genome editing technology. Dev Growth Differ 56: 53-62.
    • (2014) Dev Growth Differ , vol.56 , pp. 53-62
    • Kishi, N.1    Sato, K.2    Sasaki, E.3    Okano, H.4
  • 110
    • 84863693043 scopus 로고    scopus 로고
    • An effector-reduced anti-beta-amyloid (Abeta) antibody with unique abeta binding properties promotes neuroprotection and glial engulfment of Abeta
    • Adolfsson O, Pihlgren M, Toni N, Varisco Y, et al. 2012. An effector-reduced anti-beta-amyloid (Abeta) antibody with unique abeta binding properties promotes neuroprotection and glial engulfment of Abeta. J Neurosci 32: 9677-89.
    • (2012) J Neurosci , vol.32 , pp. 9677-9689
    • Adolfsson, O.1    Pihlgren, M.2    Toni, N.3    Varisco, Y.4
  • 111
    • 79956077225 scopus 로고    scopus 로고
    • Intrastriatal transplantation of microcarrier-bound human retinal pigment epithelial cells versus sham surgery in patients with advanced Parkinson's disease: a double-blind, randomised, controlled trial
    • Gross RE, Watts RL, Hauser RA, Bakay RA, et al. 2011. Intrastriatal transplantation of microcarrier-bound human retinal pigment epithelial cells versus sham surgery in patients with advanced Parkinson's disease: a double-blind, randomised, controlled trial. Lancet Neurol 10: 509-19.
    • (2011) Lancet Neurol , vol.10 , pp. 509-519
    • Gross, R.E.1    Watts, R.L.2    Hauser, R.A.3    Bakay, R.A.4
  • 112
    • 84860130544 scopus 로고    scopus 로고
    • Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease
    • Mittermeyer G, Christine CW, Rosenbluth KH, Baker SL, et al. 2012. Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease. Hum Gene Ther 23: 377-81.
    • (2012) Hum Gene Ther , vol.23 , pp. 377-381
    • Mittermeyer, G.1    Christine, C.W.2    Rosenbluth, K.H.3    Baker, S.L.4
  • 113
    • 84892394291 scopus 로고    scopus 로고
    • Long-term clinical outcome of fetal cell transplantation for Parkinson disease: two case reports
    • Kefalopoulou Z, Politis M, Piccini P, Mencacci N, et al. 2014. Long-term clinical outcome of fetal cell transplantation for Parkinson disease: two case reports. JAMA Neurol 71: 83-7.
    • (2014) JAMA Neurol , vol.71 , pp. 83-87
    • Kefalopoulou, Z.1    Politis, M.2    Piccini, P.3    Mencacci, N.4
  • 114
    • 79958247418 scopus 로고    scopus 로고
    • p53 Gene repair with zinc finger nucleases optimised by yeast 1-hybrid and validated by Solexa sequencing
    • Herrmann F, Garriga-Canut M, Baumstark R, Fajardo-Sanchez E, et al. 2011. p53 Gene repair with zinc finger nucleases optimised by yeast 1-hybrid and validated by Solexa sequencing. PLoS One 6: e20913.
    • (2011) PLoS One , vol.6 , pp. e20913
    • Herrmann, F.1    Garriga-Canut, M.2    Baumstark, R.3    Fajardo-Sanchez, E.4
  • 115
    • 0034749283 scopus 로고    scopus 로고
    • Stimulation of homologous recombination through targeted cleavage by chimeric nucleases
    • Bibikova M, Carroll D, Segal DJ, Trautman JK, et al. 2001. Stimulation of homologous recombination through targeted cleavage by chimeric nucleases. Mol Cell Biol 21: 289-97.
    • (2001) Mol Cell Biol , vol.21 , pp. 289-297
    • Bibikova, M.1    Carroll, D.2    Segal, D.J.3    Trautman, J.K.4
  • 116
    • 78651270582 scopus 로고    scopus 로고
    • TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain
    • Li T, Huang S, Jiang WZ, Wright D, et al. 2011. TAL nucleases (TALNs): hybrid proteins composed of TAL effectors and FokI DNA-cleavage domain. Nucleic Acids Res 39: 359-72.
    • (2011) Nucleic Acids Res , vol.39 , pp. 359-372
    • Li, T.1    Huang, S.2    Jiang, W.Z.3    Wright, D.4
  • 117
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using CRISPR/Cas systems
    • Cong L, Ran FA, Cox D, Lin S, et al. 2013. Multiplex genome engineering using CRISPR/Cas systems. Science 339: 819-23.
    • (2013) Science , vol.339 , pp. 819-823
    • Cong, L.1    Ran, F.A.2    Cox, D.3    Lin, S.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.