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 …

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 …

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 …

[HTML][HTML] Skeletal muscle stem cells from PSC-derived teratomas have functional regenerative capacity

SSK Chan, RW Arpke, A Filareto, N Xie, MP Pappas… - Cell Stem Cell, 2018 - cell.com
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 …

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 …

[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 …

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 …

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 …

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 …

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 …