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Articles tagged with exonskipping

Muscles regeneration with genetically exon-skipped stem cells.

According to a research by _Prof.Luis Garcia and Prof. Yvan TorrenteStem cells, mainly satellite cells, were obtained from muscle bioptic material of Duchenne boys whose dystrophin gene had a dele...

Tags: Muscle generation, exonskipping, stem cells.

Treatment of Duchenne Muscular Dystrophy by Exon Skipping

PORTLAND, OR — January 21, 2009 — AVI BioPharma, Inc. (NASDAQ: AVII), a developer of RNA–based drugs, today announced results from a Phase 1 trial of its drug candidate AVI–465...

Tags: intramuscular IM injection, exonskipping, dystrophin.

Crossing the Valley of Despair- A review by Dr Karl Bettelheim

Article submitted by Dr Karl Bettelheim published on the 3rd of June 2009Although there have been some very good studies published recently especially on the use of viruses to transfer human genes...

Tags: gene therapy, exonskipping, utrophin upregulation, immunodeficiency XSCID, EGFR gene, gefitinib.

Gene therapy in Muscular Dystrophy. - A review by Dr Karl Bettelheim

Article submitted by Dr Karl Bettelheim published on the 18th of July 2009There has recently appeared an excellent review (1) of current studies on gene therapy for muscular dystrophies. While poi...

Tags: Exonskipping, phosphorodiamidate morpholino oligomers, Gene therapy.