Muscling in: Gene therapies for muscular dystrophy target RNA.

Nature Medicine
Joel R Chamberlain, Jeffrey S Chamberlain

Abstract

Muscle diseases can take many forms, from the progressive muscle degeneration of dystrophies to the childhood cancer rhabdomyosarcoma. In 'Bench to Bedside', Joel R. Chamberlain and Jeffrey S. Chamberlain discuss studies using antisense oligonucleotides to treat Duchenne muscular dystrophy and myotonic dystrophy. In 'Bedside to Bench', Simone Hettmer and Amy J. Wagers examine the implications of clinical studies describing a type of rhabdomyosarcoma that resembles acute leukemia. The findings dovetail with other studies suggesting that some of these cancers might originate outside of muscle tissue and highlight the need for a better understanding of the cells that give rise to this condition.

References

Jul 27, 2004·Nature Medicine·Paul GregorevicJeffrey S Chamberlain
Dec 28, 2007·The New England Journal of Medicine·Judith C van DeutekomGert-Jan B van Ommen
May 2, 2008·Nature·Ling-Bo LiNancy M Bonini
Jan 24, 2009·Proceedings of the National Academy of Sciences of the United States of America·Saovaros SvastiRyszard Kole
Mar 17, 2009·Annals of Neurology·Toshifumi YokotaEric Hoffman
May 5, 2009·Current Opinion in Genetics & Development·Andrea L H ArnettJeffrey S Chamberlain
May 21, 2009·Molecular Therapy : the Journal of the American Society of Gene Therapy·Aurélie GoyenvalleKay E Davies
Jul 18, 2009·Science·Thurman M WheelerCharles A Thornton

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Citations

May 25, 2011·Muscle & Nerve·Erin Pennock Foff, Mani S Mahadevan
Jun 1, 2010·Muscle & Nerve·Gregory T CarterJeffrey S Chamberlain
May 26, 2010·Journal of Child Neurology·Guy L OdomJeffrey S Chamberlain

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