Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Sebahattin Cirak; Virginia Arechavala-Gomeza; Michela Guglieri; Lucy Feng; Silvia Torelli; Karen Anthony; Stephen Abbs; Maria Elena Garralda; John Bourke; Dominic J. Wells; George Dickson; Matthew J. A. Wood; Steve D. Wilton; Volker Straub; Ryszard Kole; Stephen B. Shrewsbury; Caroline Sewry; Jennifer E. Morgan; Kate Bushby; Francesco Muntoni

doi:10.1016/S0140-6736(11)60756-3

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Stephen Abbs, Maria Elena Garralda, John Bourke, Dominic J. Wells, George Dickson, Matthew J. A. Wood, Steve D. Wilton, Volker Straub, Ryszard Kole, Stephen B. Shrewsbury, Caroline Sewry, Jennifer E. Morgan, Kate Bushby, Francesco Muntoni

Research output: Contribution to journal › Article › peer-review

Original language	English
Pages (from-to)	595-605
Number of pages	11
Journal	The Lancet
Volume	378
Issue number	9791
Early online date	25 Jul 2011
DOIs	https://doi.org/10.1016/S0140-6736(11)60756-3
Publication status	Published - 13 Aug 2011

Keywords

EXPRESSION
PROTEIN
SARCOLEMMA
EXERCISE
MUSCLE
ABNORMALITIES
PHENOTYPE
PRO051
MOUSE
NNOS

Access to Document

10.1016/S0140-6736(11)60756-3

http://www.sciencedirect.com/science/article/pii/S0140673611607563#

Cite this

Cirak, S., Arechavala-Gomeza, V., Guglieri, M., Feng, L., Torelli, S., Anthony, K., Abbs, S., Garralda, M. E., Bourke, J., Wells, D. J., Dickson, G., Wood, M. J. A., Wilton, S. D., Straub, V., Kole, R., Shrewsbury, S. B., Sewry, C., Morgan, J. E., Bushby, K., & Muntoni, F. (2011). Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. The Lancet, 378(9791), 595-605. https://doi.org/10.1016/S0140-6736(11)60756-3

@article{a2a5aee2ed1b405facb2fad14e06c18d,

title = "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study",

keywords = "EXPRESSION, PROTEIN, SARCOLEMMA, EXERCISE, MUSCLE, ABNORMALITIES, PHENOTYPE, PRO051, MOUSE, NNOS",

author = "Sebahattin Cirak and Virginia Arechavala-Gomeza and Michela Guglieri and Lucy Feng and Silvia Torelli and Karen Anthony and Stephen Abbs and Garralda, {Maria Elena} and John Bourke and Wells, {Dominic J.} and George Dickson and Wood, {Matthew J. A.} and Wilton, {Steve D.} and Volker Straub and Ryszard Kole and Shrewsbury, {Stephen B.} and Caroline Sewry and Morgan, {Jennifer E.} and Kate Bushby and Francesco Muntoni",

year = "2011",

month = aug,

day = "13",

doi = "10.1016/S0140-6736(11)60756-3",

language = "English",

volume = "378",

pages = "595--605",

journal = "The Lancet",

issn = "1474-547X",

publisher = "Elsevier B.V.",

number = "9791",

}

Cirak, S, Arechavala-Gomeza, V, Guglieri, M, Feng, L, Torelli, S, Anthony, K, Abbs, S, Garralda, ME, Bourke, J, Wells, DJ, Dickson, G, Wood, MJA, Wilton, SD, Straub, V, Kole, R, Shrewsbury, SB, Sewry, C, Morgan, JE, Bushby, K & Muntoni, F 2011, 'Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study', The Lancet, vol. 378, no. 9791, pp. 595-605. https://doi.org/10.1016/S0140-6736(11)60756-3

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. / Cirak, Sebahattin; Arechavala-Gomeza, Virginia; Guglieri, Michela et al.
In: The Lancet, Vol. 378, No. 9791, 13.08.2011, p. 595-605.

Research output: Contribution to journal › Article › peer-review

TY - JOUR

T1 - Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

AU - Cirak, Sebahattin

AU - Arechavala-Gomeza, Virginia

AU - Guglieri, Michela

AU - Feng, Lucy

AU - Torelli, Silvia

AU - Anthony, Karen

AU - Abbs, Stephen

AU - Garralda, Maria Elena

AU - Bourke, John

AU - Wells, Dominic J.

AU - Dickson, George

AU - Wood, Matthew J. A.

AU - Wilton, Steve D.

AU - Straub, Volker

AU - Kole, Ryszard

AU - Shrewsbury, Stephen B.

AU - Sewry, Caroline

AU - Morgan, Jennifer E.

AU - Bushby, Kate

AU - Muntoni, Francesco

PY - 2011/8/13

Y1 - 2011/8/13

KW - EXPRESSION

KW - PROTEIN

KW - SARCOLEMMA

KW - EXERCISE

KW - MUSCLE

KW - ABNORMALITIES

KW - PHENOTYPE

KW - PRO051

KW - MOUSE

KW - NNOS

U2 - 10.1016/S0140-6736(11)60756-3

DO - 10.1016/S0140-6736(11)60756-3

M3 - Article

SN - 1474-547X

VL - 378

SP - 595

EP - 605

JO - The Lancet

JF - The Lancet

IS - 9791

ER -

Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K et al. Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study. The Lancet. 2011 Aug 13;378(9791):595-605. Epub 2011 Jul 25. doi: 10.1016/S0140-6736(11)60756-3