Retroviral-Mediated Gene Transfer and Duchenne Muscular Dystrophy

1994 ◽  
pp. 391-410 ◽  
Author(s):  
Matthew G. Dunckley ◽  
George Dickson
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer

Immunity to Adeno-Associated Virus-Mediated Gene Transfer in a Random-Bred Canine Model of Duchenne Muscular Dystrophy

2006 ◽  
Vol 0 (0) ◽  
pp. 061218064941001
Author(s):  
Zejing Wang ◽  
James M. Allen ◽  
Stanley R. Riddell ◽  
Paul Gregorevic ◽  
Rainer Storb ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Canine Model ◽  
Adeno Associated Virus

Adeno-Associated Virus (AAV) Mediated Dystrophin Gene Transfer Studies and Exon Skipping Strategies for Duchenne Muscular Dystrophy (DMD)

2015 ◽  
Vol 15 (4) ◽  
pp. 395-415 ◽  
Author(s):  
Klaudia Kawecka ◽  
Michael Theodoulides ◽  
Yalin Hasoglu ◽  
Susan Jarmin ◽  
Hanna Kymalainen ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Exon Skipping ◽  
Dystrophin Gene ◽  
Adeno Associated Virus

Immunological hurdles in the path to gene therapy for Duchenne muscular dystrophy

2002 ◽  
Vol 4 (23) ◽  
pp. 1-23 ◽  
Author(s):  
Dominic J. Wells ◽  
Aurora Ferrer ◽  
Kim E. Wells
Keyword(s):  
Gene Therapy ◽  
Immune Response ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Immune Responses ◽  
Muscle Fibres ◽  
Dystrophic Muscle ◽  
Western Blots ◽  
Muscle Wasting Disease

Patients with Duchenne muscular dystrophy (DMD), an X-linked lethal muscle-wasting disease, have abnormal expression of the protein dystrophin within their muscle fibres. In the mdx mouse model of this condition, both germline and neonatal somatic gene transfers of dystrophin cDNAs have demonstrated the potential of gene therapy in treating DMD. However, in many DMD patients, there appears to be no dystrophin expression when muscle biopsies are immunostained or western blots are performed. This raises the possibility that the expression of dystrophin following gene transfer might trigger a destructive immune response against this ‘neoantigen’. Immune responses can also be generated against the gene transfer vector used to transfect the dystrophic muscle, and the combined immune response could further damage the already inflamed muscle. These problems are now beginning to be investigated in immunocompetent mdx mice. Although much work remains to be done, there are promising indications that these immune responses might not prove as much of a concern as originally envisaged.

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

Gene Therapy ◽  
2004 ◽  
Vol 11 (14) ◽  
pp. 1117-1125 ◽  
Author(s):  
L G Gregory ◽  
S N Waddington ◽  
M V Holder ◽  
K A Mitrophanous ◽  
S M K Buckley ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
In Utero ◽  
Highly Efficient ◽  
Muscle Groups

scholarly journals Gene Therapy for Duchenne Muscular Dystrophy

2021 ◽  
pp. 1-14
Author(s):  
Nertiyan Elangkovan ◽  
George Dickson
Keyword(s):  
Gene Therapy ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Viral Vector ◽  
Cardiac Complications ◽  
Muscle Fibres ◽  
Large Animal ◽  
Large Animal Models ◽  
Advanced Therapy

Duchenne muscular dystrophy (DMD) is an X-linked, muscle wasting disease that affects 1 in 5000 males. Affected individuals become wheelchair bound by the age of twelve and eventually die in their third decade due to respiratory and cardiac complications. The disease is caused by mutations in the DMD gene that codes for dystrophin. Dystrophin is a structural protein that maintains the integrity of muscle fibres and protects them from contraction-induced damage. The absence of dystrophin compromises the stability and function of the muscle fibres, eventually leading to muscle degeneration. So far, there is no effective treatment for deteriorating muscle function in DMD patients. A promising approach for treating this life-threatening disease is gene transfer to restore dystrophin expression using a safe, non-pathogenic viral vector called adeno-associated viral (AAV) vector. Whilst microdystrophin gene transfer using AAV vectors shows extremely impressive therapeutic success so far in large animal models of DMD, translating this advanced therapy medicinal product from bench to bedside still offers scope for many optimization steps. In this paper, the authors review the current progress of AAV-microdystrophin gene therapy for DMD and other treatment strategies that may apply to a subset of DMD patients depending on the mutations they carry.

Immunity to Adeno-Associated Virus-Mediated Gene Transfer in a Random-Bred Canine Model of Duchenne Muscular Dystrophy

2007 ◽  
Vol 18 (1) ◽  
pp. 18-26 ◽  
Author(s):  
Zejing Wang ◽  
James M. Allen ◽  
Stanley R. Riddell ◽  
Paul Gregorevic ◽  
Rainer Storb ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Canine Model ◽  
Adeno Associated Virus

scholarly journals 390. Impact of a Treatment with Antioxidant on Gene Transfer Efficiency After Recombinant Adeno-Associated Vector Injection in a Mouse Model of Duchenne Muscular Dystrophy

2016 ◽  
Vol 24 ◽  
pp. S155
Author(s):  
Jean-Baptiste Dupont ◽  
Benoît Tournaire ◽  
Romain Durand ◽  
Béatrice Marolleau ◽  
Emilie Bertil ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Mouse Model ◽  
Transfer Efficiency ◽  
Gene Transfer Efficiency

Gene therapy for duchenne muscular dystrophy — Early experiences with liposome-mediated gene transfer

1996 ◽  
Vol 17 (1) ◽  
pp. 63-69 ◽  
Author(s):  
L. Vitiello ◽  
A. Chonnt ◽  
J.D. Wasserman ◽  
R.G. Worton
Keyword(s):  
Gene Therapy ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Gene Transfer ◽  
Early Experiences
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