In April 2016 Manchester eScholar was replaced by the University of Manchester’s new Research Information Management System, Pure. In the autumn the University’s research outputs will be available to search and browse via a new Research Portal. Until then the University’s full publication record can be accessed via a temporary portal and the old eScholar content is available to search and browse via this archive.

Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy.

Gregory, L G; Waddington, S N; Holder, M V; Mitrophanous, K A; Buckley, S M K; Mosley, K L; Bigger, B W; Ellard, F M; Walmsley, L E; Lawrence, L; Al-Allaf, F; Kingsman, S; Coutelle, C; Themis, M

Gene therapy. 2004;11(14):1117-25.

Access to files

Full-text and supplementary files are not available from Manchester eScholar. Full-text is available externally using the following links:

Full-text held externally

Abstract

Gene therapy for Duchenne muscular dystrophy has so far not been successful because of the difficulty in achieving efficient and permanent gene transfer to the large number of affected muscles and the development of immune reactions against vector and transgenic protein. In addition, the prenatal onset of disease complicates postnatal gene therapy. We have therefore proposed a fetal approach to overcome these barriers. We have applied beta-galactosidase expressing equine infectious anaemia virus (EIAV) lentiviruses pseudotyped with VSV-G by single or combined injection via different routes to the MF1 mouse fetus on day 15 of gestation and describe substantial gene delivery to the musculature. Highly efficient gene transfer to skeletal muscles, including the diaphragm and intercostal muscles, as well as to cardiac myocytes was observed and gene expression persisted for at least 15 months after administration of this integrating vector. These findings support the concept of in utero gene delivery for therapeutic and long-term prevention/correction of muscular dystrophies and pave the way for a future application in the clinic.

Bibliographic metadata

Content type:
Publication type:
Publication form:
Published date:
Language:
eng
Journal title:
Abbreviated journal title:
ISSN:
Place of publication:
England
Volume:
11
Issue:
14
Pagination:
1117-25
Digital Object Identifier:
10.1038/sj.gt.3302268
Pubmed Identifier:
15141156
Pii Identifier:
3302268
Access state:
Active

Institutional metadata

University researcher(s):

Record metadata

Manchester eScholar ID:
uk-ac-man-scw:56476
Created by:
Bigger, Brian
Created:
14th October, 2009, 10:17:09
Last modified by:
Bigger, Brian
Last modified:
4th May, 2010, 15:38:21

Can we help?

The library chat service will be available from 11am-3pm Monday to Friday (excluding Bank Holidays). You can also email your enquiry to us.