Biostrophin

Biostrophin is a drug which may serve as a vehicle for gene therapy, in the treatment of Duchenne and Becker muscular dystrophy.[1]

As mutations in the gene which codes for the protein dystrophin is the underlying defect responsible for both disorders, biostrophin will deliver a genetically-engineered, functional copy of the gene at the molecular level to affected muscle cells.[1] Dosage, as well as a viable means for systemic release of the drug in patients, is currently being investigated with the use of both canine and primate animal models.[2]

Biostrophin is being manufactured by Asklepios BioPharmaceuticals, Inc., with funding provided by the Muscular Dystrophy Association.[3]

See also

References

  1. ↑ 1.0 1.1 Khurdayan VK, Bozzo J, Prous JR (Oct 2005). "Chronicles in drug discovery". Drug news & perspectives 18 (8): 517–522. doi:10.1358/dnp.2005.18.8.953409. ISSN 0214-0934. PMID 16391721.
  2. ↑ http://www.ncbiotech.org/news_and_events/industry_news/duchenne.html Dec 2006
  3. ↑ "Asklepios Biopharmaceutical, Inc. Announces Cross License with GlaxoSmithKline". 2006-09-28.

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