Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Maria Kinali; Virginia Arechavala-Gomeza; Lucy Feng; Sebahattin Cirak; David Hunt; Carl Adkin; Michela Guglieri; Emma Ashton; Stephen Abbs; Petros Nihoyannopoulos; Maria Elena Garralda; Mary Rutherford; Caroline McCulley; Linda Popplewell; Ian R Graham; George Dickson; Matthew J A Wood; Dominic J Wells; Steve D Wilton; Ryszard Kole; Volker Straub; Kate Bushby; Caroline Sewry; Jennifer E Morgan; Francesco Muntoni

doi:10.1016/S1474-4422(09)70211-X

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

Maria Kinali, Virginia Arechavala-Gomeza, Lucy Feng, Sebahattin Cirak, David Hunt, Carl Adkin, Michela Guglieri, Emma Ashton, Stephen Abbs, Petros Nihoyannopoulos, Maria Elena Garralda, Mary Rutherford, Caroline McCulley, Linda Popplewell, Ian R Graham, George Dickson, Matthew J A Wood, Dominic J Wells, Steve D Wilton, Ryszard KoleVolker Straub, Kate Bushby, Caroline Sewry, Jennifer E Morgan, Francesco Muntoni

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

Original language	English
Pages (from-to)	918-928
Number of pages	11
Journal	Lancet Neurology
Volume	8
Issue number	10
Early online date	26 Aug 2009
DOIs	https://doi.org/10.1016/S1474-4422(09)70211-X
Publication status	Published - Oct 2009

Keywords

Adolescent
Child
Dose-Response Relationship, Drug
Dystrophin
Gene Therapy
Humans
Immunohistochemistry
Male
Muscle, Skeletal
Muscular Dystrophy, Duchenne
Oligonucleotides
Reverse Transcriptase Polymerase Chain Reaction
Single-Blind Method

Access to Document

10.1016/S1474-4422(09)70211-X

http://www.sciencedirect.com/science/article/pii/S147444220970211X

Cite this

Kinali, M., Arechavala-Gomeza, V., Feng, L., Cirak, S., Hunt, D., Adkin, C., Guglieri, M., Ashton, E., Abbs, S., Nihoyannopoulos, P., Garralda, M. E., Rutherford, M., McCulley, C., Popplewell, L., Graham, I. R., Dickson, G., Wood, M. J. A., Wells, D. J., Wilton, S. D., ... Muntoni, F. (2009). Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurology, 8(10), 918-928. https://doi.org/10.1016/S1474-4422(09)70211-X

@article{be4130b031354732951af084af667c1b,

title = "Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study",

keywords = "Adolescent, Child, Dose-Response Relationship, Drug, Dystrophin, Gene Therapy, Humans, Immunohistochemistry, Male, Muscle, Skeletal, Muscular Dystrophy, Duchenne, Oligonucleotides, Reverse Transcriptase Polymerase Chain Reaction, Single-Blind Method",

author = "Maria Kinali and Virginia Arechavala-Gomeza and Lucy Feng and Sebahattin Cirak and David Hunt and Carl Adkin and Michela Guglieri and Emma Ashton and Stephen Abbs and Petros Nihoyannopoulos and Garralda, {Maria Elena} and Mary Rutherford and Caroline McCulley and Linda Popplewell and Graham, {Ian R} and George Dickson and Wood, {Matthew J A} and Wells, {Dominic J} and Wilton, {Steve D} and Ryszard Kole and Volker Straub and Kate Bushby and Caroline Sewry and Morgan, {Jennifer E} and Francesco Muntoni",

year = "2009",

month = oct,

doi = "10.1016/S1474-4422(09)70211-X",

language = "English",

volume = "8",

pages = "918--928",

journal = "Lancet Neurology",

issn = "1474-4422",

publisher = "Lancet Publishing Group",

number = "10",

}

Kinali, M, Arechavala-Gomeza, V, Feng, L, Cirak, S, Hunt, D, Adkin, C, Guglieri, M, Ashton, E, Abbs, S, Nihoyannopoulos, P, Garralda, ME, Rutherford, M, McCulley, C, Popplewell, L, Graham, IR, Dickson, G, Wood, MJA, Wells, DJ, Wilton, SD, Kole, R, Straub, V, Bushby, K, Sewry, C, Morgan, JE & Muntoni, F 2009, 'Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study', Lancet Neurology, vol. 8, no. 10, pp. 918-928. https://doi.org/10.1016/S1474-4422(09)70211-X

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. / Kinali, Maria; Arechavala-Gomeza, Virginia; Feng, Lucy et al.
In: Lancet Neurology, Vol. 8, No. 10, 10.2009, p. 918-928.

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

TY - JOUR

T1 - Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

AU - Kinali, Maria

AU - Arechavala-Gomeza, Virginia

AU - Feng, Lucy

AU - Cirak, Sebahattin

AU - Hunt, David

AU - Adkin, Carl

AU - Guglieri, Michela

AU - Ashton, Emma

AU - Abbs, Stephen

AU - Nihoyannopoulos, Petros

AU - Garralda, Maria Elena

AU - Rutherford, Mary

AU - McCulley, Caroline

AU - Popplewell, Linda

AU - Graham, Ian R

AU - Dickson, George

AU - Wood, Matthew J A

AU - Wells, Dominic J

AU - Wilton, Steve D

AU - Kole, Ryszard

AU - Straub, Volker

AU - Bushby, Kate

AU - Sewry, Caroline

AU - Morgan, Jennifer E

AU - Muntoni, Francesco

PY - 2009/10

Y1 - 2009/10

KW - Adolescent

KW - Child

KW - Dose-Response Relationship, Drug

KW - Dystrophin

KW - Gene Therapy

KW - Humans

KW - Immunohistochemistry

KW - Male

KW - Muscle, Skeletal

KW - Muscular Dystrophy, Duchenne

KW - Oligonucleotides

KW - Reverse Transcriptase Polymerase Chain Reaction

KW - Single-Blind Method

U2 - 10.1016/S1474-4422(09)70211-X

DO - 10.1016/S1474-4422(09)70211-X

M3 - Article

C2 - 19713152

SN - 1474-4422

VL - 8

SP - 918

EP - 928

JO - Lancet Neurology

JF - Lancet Neurology

IS - 10

ER -