Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies
JV Chakkalakal, J Thompson, RJ Parks… - The FASEB …, 2005 - Wiley Online Library
Although the molecular defect causing Duchenne/Becker muscular dystrophy (DMD/BMD)
was identified nearly 20 years ago, the development of effective therapeutic strategies has …
was identified nearly 20 years ago, the development of effective therapeutic strategies has …
Potential of adipose-derived stem cells in muscular regenerative therapies
SV Forcales - Frontiers in Aging Neuroscience, 2015 - frontiersin.org
Regenerative capacity of skeletal muscles resides in satellite cells, a self-renewing
population of muscle cells. Several studies are investigating epigenetic mechanisms that …
population of muscle cells. Several studies are investigating epigenetic mechanisms that …
Persistence and survival of autologous muscle derived cells versus bovine collagen as potential treatment of stress urinary incontinence
T Yokoyama, N Yoshimura, R Dhir, Z QU… - The Journal of …, 2001 - Elsevier
PURPOSE: We explored the use of autologous muscle derived cells as a method of treating
stress urinary incontinence. We determined whether urethral muscle derived cell injection is …
stress urinary incontinence. We determined whether urethral muscle derived cell injection is …
[HTML][HTML] Skeletal muscle stem cells from PSC-derived teratomas have functional regenerative capacity
Derivation of functional skeletal muscle stem cells from pluripotent cells without genetic
modification has proven elusive. Here we show that teratomas formed in adult skeletal …
modification has proven elusive. Here we show that teratomas formed in adult skeletal …
Gene delivery to muscle
ML Springer, TA Rando, HM Blau - Current protocols in human …, 2001 - Wiley Online Library
The delivery of genes to skeletal muscle by myoblast implantation, DNA injection, or viral
transduction has therapeutic applications for human neuromuscular and systemic disorders …
transduction has therapeutic applications for human neuromuscular and systemic disorders …
[HTML][HTML] Recent advances in innovative therapeutic approaches for Duchenne muscular dystrophy: from discovery to clinical trials
Y Shimizu-Motohashi, S Miyatake… - American journal of …, 2016 - ncbi.nlm.nih.gov
Duchenne muscular dystrophy (DMD) is an X-linked progressive degenerative muscle
disorder caused by the absence of dystrophin. There is no curative therapy, although …
disorder caused by the absence of dystrophin. There is no curative therapy, although …
Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells
R Kumar-Singh, JS Chamberlain - Human Molecular Genetics, 1996 - academic.oup.com
Adenovirus-mediated gene transfer to muscle is a promising technology for gene therapy of
Duchenne muscular dystrophy (DMD). However, currently available recombinant …
Duchenne muscular dystrophy (DMD). However, currently available recombinant …
Assessment of risks associated with cardiovascular gene therapy in human subjects
JM Isner, PR Vale, JF Symes, DW Losordo - Circulation research, 2001 - Am Heart Assoc
Clinical trials of cardiovascular gene therapy, whether using viral (53%) or nonviral (47%)
vectors, have thus far disclosed no evidence indicative of inflammatory or other …
vectors, have thus far disclosed no evidence indicative of inflammatory or other …
Skeletal muscle tissue engineering: which cell to use?
JM Fishman, A Tyraskis, P Maghsoudlou… - … Engineering Part B …, 2013 - liebertpub.com
Tissue-engineered skeletal muscle is urgently required to treat a wide array of devastating
congenital and acquired conditions. Selection of the appropriate cell type requires …
congenital and acquired conditions. Selection of the appropriate cell type requires …
Flow cytometric characterization of myogenic cell populations obtained via the preplate technique: potential for rapid isolation of muscle-derived stem cells
RJ Jankowski, C Haluszczak, M Trucco… - Human gene …, 2001 - liebertpub.com
Myoblast transplantation has been investigated as a therapy for muscle-related diseases
and as a gene delivery vehicle for therapeutic recombinant proteins. Clinical successes …
and as a gene delivery vehicle for therapeutic recombinant proteins. Clinical successes …