-
1
-
-
84859181514
-
Evidence-based path to newborn screening for Duchenne muscular dystrophy
-
Mendell JR, Shilling C, Leslie ND, Flanigan KM, Al-Dahhak R, Gastier-Foster J, et al. Evidence-based path to newborn screening for Duchenne muscular dystrophy. Ann Neurol. 2012;71(3):304-13. doi: 10. 1002/ana. 23528
-
(2012)
Ann Neurol.
, vol.71
, Issue.3
, pp. 304-313
-
-
Mendell, J.R.1
Shilling, C.2
Leslie, N.D.3
Flanigan, K.M.4
Al-Dahhak, R.5
Gastier-Foster, J.6
-
2
-
-
0023718118
-
An explanation for the phenotypic differences between patients bearing partial deletions of theDMDlocus
-
Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM. An explanation for the phenotypic differences between patients bearing partial deletions of theDMDlocus. Genomics. 1988;2(1):90-5.
-
(1988)
Genomics.
, vol.2
, Issue.1
, pp. 90-95
-
-
Monaco, A.P.1
Bertelson, C.J.2
Liechti-Gallati, S.3
Moser, H.4
Kunkel, L.M.5
-
3
-
-
34748906143
-
Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications
-
Aartsma-Rus A, van Ommen G-JB. Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications. RNA. 2007;13(10):1609-24. doi: 10. 1261/rna. 653607
-
(2007)
RNA
, vol.13
, Issue.10
, pp. 1609-1624
-
-
Aartsma-Rus, A.1
Van Ommen, G.-J.B.2
-
4
-
-
84873734105
-
RNA-Guided Human Genome Engineering via Cas9
-
Mali P, Yang L, Esvelt KM, Aach J, Guell M, DiCarlo JE, et al. RNA-Guided Human Genome Engineering via Cas9. Science. 2013;339:823-7. doi: 10. 1126/science. 1232033
-
(2013)
Science.
, vol.339
, pp. 823-827
-
-
Mali, P.1
Yang, L.2
Esvelt, K.M.3
Aach, J.4
Guell, M.5
DiCarlo, J.E.6
-
5
-
-
84873729095
-
Multiplex genome engineering using crispr/cas systems
-
Cong L, Ran AF, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex Genome Engineering Using CRISPR/Cas Systems. Science. 2013;339:819-23. doi: 10. 1126/science. 1231143
-
(2013)
Science.
, vol.339
, pp. 819-823
-
-
Cong, L.1
Ran, A.F.2
Cox, D.3
Lin, S.4
Barretto, R.5
Habib, N.6
-
6
-
-
84865070369
-
A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
-
Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity. Science. 2012;337:816-22. doi: 10. 1126/science. 1225829
-
(2012)
Science.
, vol.337
, pp. 816-822
-
-
Jinek, M.1
Chylinski, K.2
Fonfara, I.3
Hauer, M.4
Doudna, J.A.5
Charpentier, E.6
-
7
-
-
41949122156
-
Generation and characterization of transgenic mice with the full-length human DMD gene
-
't Hoen PAC, de Meijer EJ, Boer JM, Vossen RHAM, Turk R, Maatman RGHJ, et al. Generation and characterization of transgenic mice with the full-length human DMD gene. J Biol Chem. 2008;283(9):5899-907. doi: 10. 1074/jbc. M709410200
-
(2008)
J Biol Chem.
, vol.283
, Issue.9
, pp. 5899-5907
-
-
T'Hoen, P.A.C.1
De Meijer, E.J.2
Boer, J.M.3
Vossen, R.H.A.M.4
Turk, R.5
Maatman, R.G.H.J.6
-
8
-
-
72849135317
-
Latent TGF-binding protein 4 modifies muscular dystrophy in mice
-
Heydemann A, Ceco E, Lim JE, Hadhazy M, Ryder P, Moran JL, et al. Latent TGF-binding protein 4 modifies muscular dystrophy in mice. JCI. 2009;119(12):3703-12. doi: 10. 1172/JCI39845
-
(2009)
JCI
, vol.119
, Issue.12
, pp. 3703-3712
-
-
Heydemann, A.1
Ceco, E.2
Lim, J.E.3
Hadhazy, M.4
Ryder, P.5
Moran, J.L.6
-
9
-
-
84962204038
-
Effect of genetic background on the dystrophic phenotype in mdx mice
-
Coley WD, Bogdanik L, Vila MC, Yu Q, van der Meulen JH, Rayavarapu S, et al. Effect of genetic background on the dystrophic phenotype in mdx mice. Hum Mol Genet. 2016;25(1):130-45. doi: 10. 1093/hmg/ddv460
-
(2016)
Hum Mol Genet.
, vol.25
, Issue.1
, pp. 130-145
-
-
Coley, W.D.1
Bogdanik, L.2
Vila, M.C.3
Yu, Q.4
Van Der Meulen, J.H.5
Rayavarapu, S.6
-
10
-
-
84899083880
-
Annexin A6 modifies muscular dystrophy by mediating sarcolemmal repair
-
Swaggart KA, Demonbreun AR, Vo AH, Swanson KE, Kim EY, Fahrenbach JP, et al. Annexin A6 modifies muscular dystrophy by mediating sarcolemmal repair. PNAS. 2014;111(16):6004-9. doi: 10. 1073/pnas. 1324242111
-
(2014)
PNAS
, vol.111
, Issue.16
, pp. 6004-6009
-
-
Swaggart, K.A.1
Demonbreun, A.R.2
Vo, A.H.3
Swanson, K.E.4
Kim, E.Y.5
Fahrenbach, J.P.6
-
11
-
-
84962787321
-
A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-DerivedMuscle Cells
-
Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, et al. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-DerivedMuscle Cells. Cell Stem Cell. 2016;18(4):533-40. doi: 10. 1016/j. stem. 2016. 01. 021
-
(2016)
Cell Stem Cell.
, vol.18
, Issue.4
, pp. 533-540
-
-
Young, C.S.1
Hicks, M.R.2
Ermolova, N.V.3
Nakano, H.4
Jan, M.5
Younesi, S.6
-
12
-
-
80055107411
-
DNA transfection of mammalian skeletal muscles using in vivo electroporation
-
DiFranco M, Quinonez M, Capote J, Vergara J. DNA transfection of mammalian skeletal muscles using in vivo electroporation. JoVE. 2009;(32):1-7. doi: 10. 3791/1520
-
(2009)
JoVE.
, Issue.32
, pp. 1-7
-
-
DiFranco, M.1
Quinonez, M.2
Capote, J.3
Vergara, J.4
-
13
-
-
84922735816
-
In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system
-
Maddalo D, Manchado E, Concepcion CP, Bonetti C, Vidigal JA, Han Y-C, et al. In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system. Nature. 2014;516(7531):423-7. doi: 10. 1038/nature13902
-
(2014)
Nature.
, vol.516
, Issue.7531
, pp. 423-427
-
-
Maddalo, D.1
Manchado, E.2
Concepcion, C.P.3
Bonetti, C.4
Vidigal, J.A.5
Han, Y.-C.6
-
14
-
-
0028910144
-
Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin
-
Thanh LT, Nguyen TM, Helliwell TR, Morris GE. Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin. Am J Hum Genet. 1995;56(3): 725-31.
-
(1995)
Am J Hum Genet.
, vol.56
, Issue.3
, pp. 725-731
-
-
Thanh, L.T.1
Nguyen, T.M.2
Helliwell, T.R.3
Morris, G.E.4
-
15
-
-
84873729095
-
Multiplex genome engineering using CRISPR/Cas systems
-
Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339(6121):819-23. doi: 10. 1126/science. 1231143
-
(2013)
Science.
, vol.339
, Issue.6121
, pp. 819-823
-
-
Cong, L.1
Ran, F.A.2
Cox, D.3
Lin, S.4
Barretto, R.5
Habib, N.6
-
16
-
-
0027365063
-
Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy
-
Man N, Morris GE. Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy. Am J Hum Genet. 1993;52:1057-66.
-
(1993)
Am J Hum Genet.
, vol.52
, pp. 1057-1066
-
-
Man, N.1
Morris, G.E.2
-
17
-
-
84871061937
-
Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy
-
Kendall GC, Mokhonova EI, Moran M, Sejbuk NE, Wang DW, SilvaO, et al. Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy. Sci Transl Med. 2012;4:164ra160. doi: 10. 1126/scitranslmed. 3005054
-
(2012)
Sci Transl Med.
, vol.4
, pp. 164ra160
-
-
Kendall, G.C.1
Mokhonova, E.I.2
Moran, M.3
Sejbuk, N.E.4
Wang, D.W.5
Silva, O.6
|