Citation: Ep. Hoffman et D. Dressman, Molecular pathophysiology and targeted therapeutics for muscular dystrophy, TRENDS PHAR, 22(9), 2001, pp. 465-470
Citation: Es. Gordon et Ep. Hoffman, The ABC's of limb-girdle muscular dystrophy: alpha-sarcoglycanopathy, Bethlem myopathy, calpainopathy and more, CURR OP NEU, 14(5), 2001, pp. 567-573
Authors:
Colantuoni, C
Jeon, OH
Hyder, K
Chenchik, A
Khimani, AH
Narayanan, V
Hoffman, EP
Kaufmann, WE
Naidu, S
Pevsner, J
Citation: C. Colantuoni et al., Gene expression profiling in postmortem Rett syndrome brain: Differential gene expression and patient classification, NEUROBIOL D, 8(5), 2001, pp. 847-865
Authors:
Hayashi, YK
Tezak, Z
Momoi, T
Nonaka, I
Garcia, CA
Hoffman, EP
Arahata, K
Citation: Yk. Hayashi et al., Massive muscle cell degeneration in the early stage of merosin-deficient congenital muscular dystrophy, NEUROMUSC D, 11(4), 2001, pp. 350-359
Authors:
Wagner, KR
Hamed, S
Hadley, DW
Gropman, AL
Burstein, AH
Escolar, DM
Hoffman, EP
Fischbeck, KH
Citation: Kr. Wagner et al., Gentamicin treatment of Duchenne and Becker muscular dystrophy due to nonsense mutations, ANN NEUROL, 49(6), 2001, pp. 706-711
Authors:
Wu, FF
Takahashi, MP
Pegoraro, E
Angelini, C
Colleselli, P
Cannon, SC
Hoffman, EP
Citation: Ff. Wu et al., A new mutation in a family with cold-aggravated myotonia disrupts Na+ channel inactivation, NEUROLOGY, 56(7), 2001, pp. 878-884
Authors:
Hoffbuhr, K
Devaney, JM
LaFleur, B
Sirianni, N
Scacheri, C
Giron, J
Schuette, J
Innis, J
Marino, M
Philippart, M
Narayanan, V
Umansky, R
Kronn, D
Hoffman, EP
Naidu, S
Citation: K. Hoffbuhr et al., MeCP2 mutations in children with and without the phenotype of Rett syndrome, NEUROLOGY, 56(11), 2001, pp. 1486-1495
Citation: Kw. Liang et al., Targeted delivery of plasmid DNA to myogenic cells via transferrin-conjugated peptide nucleic acid, MOL THER, 1(3), 2000, pp. 236-243
Authors:
Fanin, M
Hoffman, EP
Angelini, C
Pegoraro, E
Citation: M. Fanin et al., Private beta- and gamma-sarcoglycan gene mutations: Evidence of a founder effect in northern Italy, HUM MUTAT, 16(1), 2000, pp. 13-17
Authors:
Pegoraro, E
Fanin, M
Trevisan, CP
Angelini, C
Hoffman, EP
Citation: E. Pegoraro et al., A novel laminin alpha 2 isoform in severe laminin alpha 2 deficient congenital muscular dystrophy, NEUROLOGY, 55(8), 2000, pp. 1128-1134
Authors:
Scacheri, PC
Hoffman, EP
Fratkin, JD
Semino-Mora, C
Senchak, A
Davis, MR
Laing, NG
Vedanarayanan, V
Subramony, SH
Citation: Pc. Scacheri et al., A novel ryanodine receptor gene mutation causing both cores and rods in congenital myopathy, NEUROLOGY, 55(11), 2000, pp. 1689-1696
Authors:
Xiao, X
Li, J
Tsao, YP
Dressman, D
Hoffman, EP
Watchko, JF
Citation: X. Xiao et al., Full functional rescue of a complete muscle (TA) in dystrophic hamsters byadeno-associated virus vector-directed gene therapy, J VIROLOGY, 74(3), 2000, pp. 1436-1442
Citation: Yw. Chen et al., Expression profiling in the muscular dystrophies: Identification of novel aspects of molecular pathophysiology, J CELL BIOL, 151(6), 2000, pp. 1321-1336
Citation: Ep. Hoffman et J. Giron, Molecular diagnosis and genetic counseling of the manifesting carrier of Duchenne muscular dystrophy, METH MOL M, 43, 2000, pp. 173-188
Authors:
Akkaraju, GR
Huard, J
Hoffman, EP
Goins, WF
Pruchnic, R
Watkins, SC
Cohen, JB
Glorioso, JC
Citation: Gr. Akkaraju et al., Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle, J GENE MED, 1(4), 1999, pp. 280-289
Authors:
Bouri, K
Feero, WG
Myerburg, MM
Wickham, TJ
Kovesdi, I
Hoffman, EP
Clemens, PR
Citation: K. Bouri et al., Polylysine modification of adenoviral fiber protein enhances muscle cell transduction, HUM GENE TH, 10(10), 1999, pp. 1633-1640
Authors:
Li, J
Dressman, D
Tsao, YP
Sakamoto, A
Hoffman, EP
Xiao, X
Citation: J. Li et al., rAAV vector-mediated sarcoglycan gene transfer in a hamster model for limbgirdle muscular dystrophy, GENE THER, 6(1), 1999, pp. 74-82