Evolving therapeutic strategies for Duchenne muscular dystrophy: targeting downstream events

JG Tidball, M Wehling-Henricks - Pediatric Research, 2004 - nature.com
Duchenne muscular dystrophy (DMD) is a progressive, lethal, muscle wasting disease that
affects 1 of 3500 boys born worldwide. The disease results from mutation of the dystrophin …

Myoblast transplantation

T Partridge - Neuromuscular Disorders, 2002 - Elsevier
Myoblast transplantation was the first quasi-gene therapy to be suggested for Duchenne
muscular dystrophy. Animal experiments established the principles that the missing gene …

In vivo targeted repair of a point mutation in the canine dystrophin gene by a chimeric RNA/DNA oligonucleotide

RJ Bartlett, S Stockinger, MM Denis, WT Bartlett… - Nature …, 2000 - nature.com
In the canine model of Duchenne muscular dystrophy in golden retrievers (GRMD), a point
mutation within the splice acceptor site of intron 6 leads to deletion of exon 7 from the …

Regenerative medicine applications of mesenchymal stem cells

S Hosseini, L Taghiyar, F Safari… - Cell Biology and …, 2018 - Springer
A major research challenge is to develop therapeutics that assist with healing damaged
tissues and organs because the human body has limited ability to restore the majority of …

Effect of creatine monohydrate in improving cellular energetics and muscle strength in ambulatory Duchenne muscular dystrophy patients: a randomized, placebo …

B Banerjee, U Sharma, K Balasubramanian… - Magnetic resonance …, 2010 - Elsevier
Randomized, placebo-controlled single blinded study was carried out to evaluate the effect
of oral creatine supplementation on cellular energetics, manual muscle test (MMT) score …

Human muscle-derived CLEC14A-positive cells regenerate muscle independent of PAX7

A Marg, H Escobar, N Karaiskos, SA Grunwald… - Nature …, 2019 - nature.com
Skeletal muscle stem cells, called satellite cells and defined by the transcription factor PAX7,
are responsible for postnatal muscle growth, homeostasis and regeneration. Attempts to …

Antioxidant levels represent a major determinant in the regenerative capacity of muscle stem cells

KL Urish, JB Vella, M Okada, BM Deasy… - Molecular biology of …, 2009 - Am Soc Cell Biol
Stem cells are classically defined by their multipotent, long-term proliferation, and self-
renewal capabilities. Here, we show that increased antioxidant capacity represents an …

Efficacy of myoblast transplantation in nonhuman primates following simple intramuscular cell injections: toward defining strategies applicable to humans

D Skuk, M Goulet, B Roy, JP Tremblay - Experimental neurology, 2002 - Elsevier
Nonhuman primates were used to define myoblast transplantation strategies applicable to
humans. Nevertheless, previous experiments were based on the use of myotoxins …

Adult stem cell sources for skeletal and smooth muscle tissue engineering

S Salemi, JA Prange, V Baumgartner… - Stem Cell Research & …, 2022 - Springer
Introduction Tissue engineering is an innovative field with enormous developments in recent
years. These advances are not only in the understanding of how stem cells can be isolated …

Successful myoblast transplantation in primates depends on appropriate cell delivery and induction of regeneration in the host muscle

D Skuk, B Roy, M Goulet, JP Tremblay - Experimental neurology, 1999 - Elsevier
Myoblast transplantation (MT) may be a potential treatment for severe recessive hereditary
myopathies. The limited results of MT in clinical trials led us to improve this technique in …