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