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https://musculardystrophynews.com/2020/02/27/new-crispr-cas9-delivery-improves-muscle-function-dmd-mice-study/
Feb 27, 2020 · Researchers have improved a gene editing technique that may more efficiently correct DMD gene mutations in people with Duchenne muscular dystrophy (DMD), restore the normal function of the dystrophin protein, and improve muscle function, a study in mice shows.. The study, “ Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV …4.4/5(9)
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2852878/
Nov 11, 2009 · Gene delivery to muscle. We injected the vector AAV1-FS344 under the control of the ubiquitously strong cytomegalovirus promoter (CMV-FS) or the muscle creatine kinase promoter (MCK-FS), which provides muscle-specific gene expression, into the right quadriceps muscle of six normal cynomolgus macaques.Author: Janaiah Kota, Chalonda R. Handy, Chalonda R. Handy, Amanda M. Haidet, Amanda M. Haidet, Chrystal L. ...
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC19498/
Nov 26, 1996 · Skeletal muscle is a useful target to evaluate this approach because of its large mass, vascularity, and accessibility . Since muscle fibers are nondividing, effective gene delivery could potentially result in long-lived protein production.Author: Paul D. Kessler, Gregory M. Podsakoff, Xiaojuan Chen, Susan A. McQuiston, Peter C. Colosi, Laura A. ...
https://www.pnas.org/content/93/24/14082
Nov 26, 1996 · Gene therapy has been proposed for several inherited and acquired protein deficiencies. The feasibility of this approach is supported by studies showing that gene delivery to skeletal muscle (1, 2), vascular smooth muscle , and liver can result in systemic levels of therapeuticAuthor: Paul D. Kessler, Gregory M. Podsakoff, Xiaojuan Chen, Susan A. McQuiston, Peter C. Colosi, Laura A. ...
https://www.researchgate.net/publication/5425229_Gene_Delivery_to_Muscle
The delivery of genes to skeletal muscle by myoblast implantation, DNA injection, or viral transduction has therapeutic applications for human neuromuscular and systemic disorders, many of which ...
https://currentprotocols.onlinelibrary.wiley.com/doi/abs/10.1002/0471142905.hg1304s31
The delivery of genes to skeletal muscle by myoblast implantation, DNA injection, or viral transduction has therapeutic applications for human neuromuscular and systemic disorders, many of which are now represented by transgenic or “knockout” mouse models.Author: Matthew L. Springer, Thomas A. Rando, Helen M. Blau
http://www.nature.com/articles/3301874
Jan 14, 2003 · Transgenes can be delivered to muscle using viral or non-viral systems. Although the pioneer study used naked plasmid DNA for gene delivery, 1 the low efficiency of …
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