Viral vector-mediated gene transfer for CNS disease

Expert Opinion on Biological Therapy

Volumn 10, Issue 3, 2010, Pages 381-394

  Snyder, Brooke R ^a   Boulis, Nicholas M ^a   Federici, Thais ^a  

^a EMORY UNIVERSITY   (United States)

Author keywords

Brain; Gene delivery; Gene replacement; Neuromodulation; Neuroprotection; Spinal cord

Indexed keywords

ADENOVIRUS VECTOR; AROMATIC LEVO AMINO ACID DECARBOXYLASE; ASPARTOACYLASE; BETA INTERFERON; CAPSID PROTEIN; CLN2 PROTEIN; COPPER ZINC SUPEROXIDE DISMUTASE; GALANIN; GANCICLOVIR; GLUTAMATE DECARBOXYLASE 65; GLUTAMATE DECARBOXYLASE 67; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; HERPESVIRUS VECTOR; LENTIVIRUS VECTOR; LEVODOPA; NERVE GROWTH FACTOR; NEUROPEPTIDE Y; NEURTURIN; PARVOVIRUS VECTOR; PEPTIDASE; PREPROENKEPHALIN; PROTEIN P53; RETROVIRUS VECTOR; SMALL INTERFERING RNA; THYMIDINE KINASE; TYROSINE 3 MONOOXYGENASE; UNCLASSIFIED DRUG;

ADRENOLEUKODYSTROPHY; ALZHEIMER DISEASE; AMPLICON; AMYOTROPHIC LATERAL SCLEROSIS; BLOOD BRAIN BARRIER; BRAIN HEMORRHAGE; BRAIN TUMOR; CANAVAN DISEASE; CANCER PAIN; CENTRAL NERVOUS SYSTEM DISEASE; CHRONIC PAIN; CLINICAL TRIAL; DRUG DISTRIBUTION; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG INDUCED HEADACHE; EPILEPSY; EX VIVO GENE TRANSFER; GENE EXPRESSION; GENE REPLACEMENT THERAPY; GENE SILENCING; GENE TRANSFER; GENETIC TRANSDUCTION; GLIOBLASTOMA; HUMAN; HUMORAL IMMUNITY; HUNTINGTON CHOREA; INBORN ERROR OF METABOLISM; LYSOSOME STORAGE DISEASE; MOTOR NEURON DISEASE; NEUROMODULATION; NEURONAL CEROID LIPOFUSCINOSIS; NEUROPROTECTION; NEUROTOXICITY; NONHUMAN; PARKINSON DISEASE; REVIEW; RNA INTERFERENCE; SPINAL CORD INJURY; STEM CELL TRANSPLANTATION; SUICIDE GENE THERAPY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIROGENESIS; VIRUS CAPSID; VIRUS EXPRESSION; VIRUS TRANSCRIPTION;

CENTRAL NERVOUS SYSTEM DISEASES; GENE THERAPY; GENETIC VECTORS; HUMANS; VIRUSES;

EID: 76949088009     PISSN: 14712598     EISSN: None     Source Type: Journal    
DOI: 10.1517/14712590903514074     Document Type: Review

References (107)

1. Watson JD, Crick FH. Molecular structure of nucleic acids; a structure for deoxyribose nucleic acid. Nature 1953;171(4356):737-738
2. Friedmann T, Roblin R. Gene therapy for human genetic disease? Science 1972;175(25):949-955
3. Morrow JF. The prospects for gene therapy in humans. Ann NY Acad Sci 1976;265:13-21
4. Aposhian HV. The use of DNA for gene therapy-the need, experimental approach, and implications. Perspect Biol Med 1970;14(1):98-108
5. Blaese RM, Culver KW, Miller AD, et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995;270(5235):475-480
6. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288(5466):669-672
7. Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab 2003;80(1-2):148-158
8. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302(5644):415-419
9. Gene Therapy Clinical Trials Worldwide. John Wiley and Sons Ltd., 2007, Available from: http://www.wiley.co.uk/ genetherapy/clinical. [Last accessed 1 December 2010]
10. Friedmann T. The future for gene therapy-a reevaluation. Ann NY Acad Sci 1976;265:141-152
11. Rainov NG, Ren H. Clinical trials with retrovirus mediated gene therapy-what have we learned? J Neurooncol 2003;65(3):227-236
12. Stewart HJ, Leroux-Carlucci MA, Sion CJ, et al. Development of inducible EIAV-based lentiviral vector packaging and producer cell lines. Gene Ther 2009;16(6):805-814
13. Azzouz M, Martin-Rendon E, Barber RD, et al. Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J Neurosci 2002;22(23):10302-10312
14. Kutner RH, Zhang XY, Reiser J. Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors. Nat Protoc 2009;4(4):495-505
15. Valori CF, Ning K, Wyles M, Azzouz M. Development and applications of non-HIV-based lentiviral vectors in neurological disorders. Curr Gene Ther 2008;8(6):406-418
16. Lundberg C, Bjorklund T, Carlsson T, et al. Applications of lentiviral vectors for biology and gene therapy of neurological disorders. Curr Gene Ther 2008;8(6):461-473
17. Cronin J, Zhang XY, Reiser J. Altering the tropism of lentiviral vectors through pseudotyping. Curr Gene Ther 2005;5(4):387-398
18. Azzouz M, Le T, Ralph GS, et al. Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J Clin Invest 2004;114(12):1726-1731
19. Tanase K, Teng Q, Krishnaney AA, et al. Cervical spinal cord delivery of a rabies G protein pseudotyped lentiviral vector in the SOD-1 transgenic mouse. Invited submission from the Joint Section Meeting on Disorders of the Spine and Peripheral Nerves, March 2004. J Neurosurg Spine 2004;1(1):128-136
20. Consiglio A, Gritti A, Dolcetta D, et al. Robust in vivo gene transfer into adult mammalian neural stem cells by lentiviral vectors. Proc Natl Acad Sci USA 2004;101(41):14835-14840
21. Capowski EE, Schneider BL, Ebert AD, et al. Lentiviral vector-mediated genetic modification of human neural progenitor cells for ex vivo gene therapy. J Neurosci Methods 2007;163(2):338-349
22. Harrow S, Papanastassiou V, Harland J, et al. HSV1716 injection into the brain adjacent to tumour following surgical resection of high-grade glioma: safety data and long-term survival. Gene Ther 2004;11(22):1648-1658
23. Wolfe D, Mata M, Fink DJ. A human trial of HSV-mediated gene transfer for the treatment of chronic pain. Gene Ther 2009;16(4):455-460
24. Fraefel C. Gene delivery using helper virus-free HSV-1 amplicon vectors. Curr Protoc Neurosci 2007 Jul;Chapter 4:Unit 4 14
25. Asadi-Moghaddam K, Chiocca EA. Prodrug-activation gene therapy. In: Kaplitt MG, During MJ, editors, Gene therapy in the central nervous system: from bench to bedside. San Diego, CA: Elsevier, Inc; 2006. p. 291-302
26. Thomas CE, Schiedner G, Kochanek S, et al. Peripheral infection with adenovirus causes unexpected long-term brain inflammation in animals injected intracranially with first-generation, but not with high-capacity, adenovirus vectors: toward realistic long-term neurological gene therapy for chronic diseases. Proc Natl Acad Sci USA 2000;97(13):7482-7487
27. Barcia C, Jimenez-Dalmaroni M, Kroeger KM, et al. One-year expression from high-capacity adenoviral vectors in the brains of animals with pre-existing anti-adenoviral immunity: clinical implications. Mol Ther 2007;15(12):2154-2163
28. Nandi S, Lesniak MS. Adenoviral virotherapy for malignant brain tumors. Expert Opin Biol Ther 2009;9(6):737-747
29. Immonen A, Vapalahti M, Tyynela K, et al. AdvHSV-tk gene therapy with intravenous ganciclovir improves survival in human malignant glioma: a randomised, controlled study. Mol Ther 2004;10(5):967-972
30. Bessis N, GarciaCozar FJ, Boissier MC. Immune responses to gene therapy vectors: influence on vector function and effector mechanisms. Gene Ther 2004;11(Suppl 1):S10-7
31. Haberman RP, Samulski RJ, McCown TJ. Attenuation of seizures and neuronal death by adeno-associated virus vector galanin expression and secretion. Nat Med 2003;9(8):1076-1080
32. Guy J, Qi X, Muzyczka N, Hauswirth WW. Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve. Arch Ophthalmol 1999;117(7):929-937
33. Wu Z, Asokan A, Samulski RJ. Adeno-associated virus serotypes: vector toolkit for human gene therapy. Mol Ther 2006;14(3):316-327
34. McCown TJ. Adeno-associated virus (AAV) vectors in the CNS. Curr Gene Ther 2005;5(3):333-338
35. Mueller C, Flotte TR. Clinical gene therapy using recombinant adeno-associated virus vectors. Gene Ther 2008;15(11):858-863
36. Bartlett JS, Samulski RJ, McCown TJ. Selective and rapid uptake of adeno-associated virus type 2 in brain. Hum Gene Ther 1998;9(8):1181-1186
37. McFarland NR, Lee JS, Hyman BT, McLean PJ. Comparison of transduction efficiency of recombinant AAV serotypes 1, 2, 5, and 8 in the rat nigrostriatal system. J Neurochem 2009;109(3):838-845
38. Liu G, Martins IH, Chiorini JA, Davidson BL. Adeno-associated virus type 4 (AAV4) targets ependyma and astrocytes in the subventricular zone and RMS. Gene Ther 2005;12(20):1503-1508
39. Tenenbaum L, Chtarto A, Lehtonen E, et al. Recombinant AAV-mediated gene delivery to the central nervous system. J Gene Med 2004;6(Suppl 1):S212-22
40. Davidson BL, Stein CS, Heth JA, et al. Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. Proc Natl Acad Sci USA 2000;97(7):3428-3432
41. Towne C, Raoul C, Schneider BL, Aebischer P. Systemic AAV6 delivery mediating RNA interference against SOD1: neuromuscular transduction does not alter disease progression in fALS mice. Mol Ther 2008;16(6):1018-1025
42. Duque S, Joussemet B, Riviere C, et al. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol Ther 2009;17(7):1187-1196
43. Foust KD, Nurre E, Montgomery CL, et al. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat Biotechnol 2009;27(1):59-65
44. Broekman ML, Comer LA, Hyman BT, Sena-Esteves M. Adeno-associated virus vectors serotyped with AAV8 capsid are more efficient than AAV-1 or -2 serotypes for widespread gene delivery to the neonatal mouse brain. Neuroscience 2006;138(2):501-510
45. Burger C, Gorbatyuk OS, Velardo MJ, et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol Ther 2004;10(2):302-317
46. Maheshri N, Koerber JT, Kaspar BK, Schaffer DV. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nat Biotechnol 2006;24(2):198-204
47. Li W, Asokan A, Wu Z, et al. Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles. Mol Ther 2008;16(7):1252-1260
48. Shevtsova Z, Malik JM, Michel U, et al. Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo. Exp Physiol 2005;90(1):53-59
49. Eberling JL, Jagust WJ, Christine CW, et al. Results from a phase I safety trial of hAADC gene therapy for parkinson disease. Neurology 2008;70(21):1980-1983
50. Federici T, Riley J, Park J, et al. Preclinical safety validation of a stabilized viral vector direct injection approach to the cervical spinal cord. Clin Transl Sci 2009;2(2):165-167
51. Riley J, Butler J, Park J, et al. Targeted spinal cord therapeutics delivery: stabilized platform and MER guidance validation. Stereotact Funct Neurosurg 2007;86(2):67-74
52. Riley J, Federici T, Park J, et al. Cervical spinal cord therapeutics delivery: preclinical safety validation of a stabilized microinjection platform. Neurosurgery; 65(4):754-61; discussion 761-762
53. Kordower JH, Herzog CD, Dass B, et al. Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys. Ann Neurol 2006;60(6):706-715
54. Herzog CD, Dass B, Holden JE, et al. Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys. Mov Disord 2007;22(8):1124-1132
55. Herzog CD, Dass B, Gasmi M, et al. Transgene expression, bioactivity, and safety of CERE-120 (AAV2-neurturin) following delivery to the monkey striatum. Mol Ther 2008;16(10):1737-1744
56. Towne C, Schneider BL, Kieran D, et al. Efficient transduction of non-human primate motor neurons after intramuscular delivery of recombinant AAV serotype 6. Gene Ther 2009: published online3 September 2009; doi: 10.1038/gt.2009.119
57. Boulis NM, Noordmans AJ, Song DK, et al. Adeno-associated viral vector gene expression in the adult rat spinal cord following remote vector delivery. Neurobiol Dis 2003;14(3):535-541
58. Kaspar BK, Llado J, Sherkat N, et al. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science 2003;301(5634):839-842
59. Hollis ER, 2nd, Kadoya K, et al. Efficient retrograde neuronal transduction utilizing self-complementary AAV1. Mol Ther 2008;16(2):296-301
60. Cearley CN, Wolfe JH. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther 2006;13(3):528-537
61. Mata M, Chattopadhyay M, Fink DJ. Gene therapy for the treatment of sensory neuropathy. Expert Opin Biol Ther 2006;6(5):499-507
62. Azzouz M, Ralph GS, Storkebaum E, et al. VEGF delivery with retrogradely transported lentivector prolongs survival in a mouse ALS model. Nature 2004;429(6990):413-417
63. Storek B, Harder NM, Banck MS, et al. Intrathecal long-term gene expression by self-complementary adeno-associated virus type 1 suitable for chronic pain studies in rats. Mol Pain 2006;2:4. Published online 30 January 2006, doi: 10.1186/1744-8069-2-4
64. Iwamoto N, Watanabe A, Yamamoto M, et al. Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1. J Gene Med 2009;11(6):498-505
65. Towne C, Pertin M, Beggah AT, et al. Recombinant adeno-associated virus serotype 6 (rAAV2/6)-mediated gene transfer to nociceptive neurons through different routes of delivery. Mol Pain 2009;5:52. Published online 8 September 2009, doi: 10.1186/1744-8069-5-52
66. Storek B, Reinhardt M, Wang C, et al. Sensory neuron targeting by self-complementary AAV8 via lumbar puncture for chronic pain. Proc Natl Acad Sci USA 2008;105(3):1055-1060
67. Finegold AA, Mannes AJ, Iadarola MJ. A paracrine paradigm for in vivo gene therapy in the central nervous system: treatment of chronic pain. Hum Gene Ther 1999;10(7):1251-1257
68. Poliani PL, Brok H, Furlan R, et al. Delivery to the central nervous system of a nonreplicative herpes simplex type 1 vector engineered with the interleukin 4 gene protects rhesus monkeys from hyperacute autoimmune encephalomyelitis. Hum Gene Ther 2001;12(8):905-920
69. Fedorova E, Battini L, Prakash-Cheng A, et al. Lentiviral gene delivery to CNS by spinal intrathecal administration to neonatal mice. J Gene Med 2006;8(4):414-424
70. McCarty DM, Dirosario J, Gulaid K, et al. Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice. Gene Ther 2009;16(11):1340-1352
71. Leone P, Janson CG, Bilaniuk L, et al. Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for canavan disease. Ann Neurol 2000;48(1):27-38
72. Janson C, McPhee S, Bilaniuk L, et al. Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain. Hum Gene Ther 2002;13(11):1391-1412
73. McPhee SW, Janson CG, Li C, et al. Immune responses to AAV in a phase I study for canavan disease. J Gene Med 2006;8(5):577-588
74. Hackett NR, Redmond DE, Sondhi D, et al. Safety of direct administration of AAV2CUhCLN2, a candidate treatment for the central nervous system manifestations of late infantile neuronal ceroid lipofuscinosis, to the brain of rats and nonhuman primates. Hum Gene Ther 2005;16(12):1484-1503
75. Worgall S, Sondhi D, Hackett NR, et al. Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther 2008;19(5):463-474
76. Sondhi D, Peterson DA, Edelstein AM, et al. Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis. Exp Neurol 2008;213(1):18-27
77. Kaplitt MG, Feigin A, Tang C, et al. Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for parkinson's disease: an open label, phase I trial. Lancet 2007;369(9579):2097-2105
78. Feigin A, Kaplitt MG, Tang C, et al. Modulation of metabolic brain networks after subthalamic gene therapy for Parkinson's disease. Proc Natl Acad Sci U S A 2007;104(49):19559-19564
79. Bankiewicz KS, Forsayeth J, Eberling JL, et al. Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC. Mol Ther 2006;14(4):564-570
80. Christine CW, Starr PA, Larson PS, et al. Safety and tolerability of putaminal AADC gene therapy for parkinson disease. Neurology 2009;73(18):1662-1669
81. Marks WJ Jr., Ostrem JL, Verhagen L, et al. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic parkinson's disease: an open-label, phase I trial. Lancet Neurol 2008;7(5):400-408
82. Siffert J, Marks WJ Jr, Starr PA, et al. AAV2-neurturin (CERE-120) for advanced parkinson's disease (PD): efficacy and safety results from a controlled Phase 2 clinical trial. Soc Neurosci 2009; Chicago, IL; 2009
83. Tuszynski MH, Thal L, Pay M, et al. A phase 1 clinical trial of nerve growth factor gene therapy for alzheimer disease. Nat Med 2005;11(5):551-555
84. Nagahara AH, Bernot T, Moseanko R, et al. Long-term reversal of cholinergic neuronal decline in aged non-human primates by lentiviral NGF gene delivery. Exp Neurol 2009;215(1):153-159
85. Bishop KM, Hofer EK, Mehta A, et al. Therapeutic potential of CERE-110 (AAV2-NGF): targeted, stable, and sustained NGF delivery and trophic activity on rodent basal forebrain cholinergic neurons. Exp Neurol 2008;211(2):574-584
86. Lang FF, Bruner JM, Fuller GN, et al. Phase I trial of adenovirus-mediated p53 gene therapy for recurrent glioma: biological and clinical results. J Clin Oncol 2003;21(13):2508-2518
87. Eck SL, Alavi JB, Alavi A, et al. Treatment of advanced CNS malignancies with the recombinant adenovirus H5.010RSVTK: a phase I trial. Hum Gene Ther 1996;7(12):1465-1482
88. Trask TW, Trask RP, Aguilar-Cordova E, et al. Phase I study of adenoviral delivery of the HSV-tk gene and ganciclovir administration in patients with current malignant brain tumors. Mol Ther 2000;1(2):195-203
89. Germano IM, Fable J, Gultekin SH, Silvers A. Adenovirus/herpes simplex-thymidine kinase/ganciclovir complex: preliminary results of a phase I trial in patients with recurrent malignant gliomas. J Neurooncol 2003;65(3):279-289
90. Osborne R. Ark floats gene therapy's boat, for now. Nat Biotechnol 2008;26(10):1057-1059
91. Langford G, Dayan A, Yla-Herttuala S, Eckland D. A preclinical assessment of the safety and biodistribution of an adenoviral vector containing the herpes simplex virus thymidine kinase gene (cerepro) after intracerebral administration. J Gene Med 2009;11(6):468-476
92. Chiocca EA, Smith KM, McKinney B, et al. A phase I trial of Ad.hIFN-beta gene therapy for glioma. Mol Ther 2008;16(3):618-626
93. Ramaswamy S, McBride JL, Herzog CD, et al. Neurturin gene therapy improves motor function and prevents death of striatal neurons in a 3-nitropropionic acid rat model of huntington's disease. Neurobiol Dis 2007;26(2):375-384
94. Ramaswamy S, McBride JL, Han I, et al. Intrastriatal CERE-120 (AAV-neurturin) protects striatal and cortical neurons and delays motor deficits in a transgenic mouse model of huntington's disease. Neurobiol Dis 2009;34(1):40-50
95. Harper SQ, Staber PD, He X, et al. RNA interference improves motor and neuropathological abnormalities in a huntington's disease mouse model. Proc Natl Acad Sci USA 2005;102(16):5820-5825
96. Hadaczek P, Forsayeth J, Mirek H, et al. Transduction of non-human primate brain with adeno-associated virus serotype 1: vector trafficking and immune response. Hum Gene Ther 2009;20:225-237
97. Richichi C, Lin EJ, Stefanin D, et al. Anticonvulsant and antiepileptogenic effects mediated by adeno-associated virus vector neuropeptide Y expression in the rat hippocampus. J Neurosci 2004;24(12):3051-3059
98. McCown TJ. Adeno-associated virus-mediated expression and constitutive secretion of galanin suppresses limbic seizure activity in vivo. Mol Ther 2006;14(1):63-68
99. Hoffmann B, Mayatepek E. Neurological manifestations in lysosomal storage disorders - from pathology to first therapeutic possibilities. Neuropediatrics 2005;36(5):285-289
100. Sands MS, Haskins ME. CNS-directed gene therapy for lysosomal storage diseases. Acta Paediatr Suppl 2008;97(457):22-27
101. Haskins M. Gene therapy for lysosomal storage diseases (LSDs) in large animal models. ILAR J 2009;50(2):112-121
102. Fraldi A, Hemsley K, Crawley A, et al. Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes. Hum Mol Genet 2007;16(22):2693-2702
103. Karolewski BA, Wolfe JH. Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol Ther 2006;14(1):14-24
104. Liu G, Martins I, Wemmie JA, et al. Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. J Neurosci 2005;25(41):9321-9327
105. Federici T, Boulis N. Gene-based treatment of motor neuron diseases. Muscle Nerve 2006;33(3):302-323
106. Ralph GS, Radcliffe PA, Day DM, et al. Silencing mutant SOD1 using RNAi protects against neurodegeneration and extends survival in an ALS model. Nat Med 2005;11(4):429-433
107. Raoul C, Abbas-Terki T, Bensadoun JC, et al. Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat Med 2005;11(4):423-428