News Column

Researchers from Academy of Sciences Report Recent Findings in Gene Therapy

May 29, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Data detailed on Biotechnology have been presented. According to news reporting originating from Paris, France, by NewsRx correspondents, research stated, "Gene therapy is quickly becoming a reality applicable in the clinic for inherited retinal diseases. Progress over the past decade has moved proof-of-concept gene therapies from bench to bedside."

Our news editors obtained a quote from the research from the Academy of Sciences, "The remarkable success in safety and efficacy, in the phase I/II clinical trials for the form of the severe childhood-onset blindness, Leber's Congenital Amaurosis (LCA) type II (due to mutations in the RPE65 gene) generated significant interest and opened up possibilities for a new era of retinal gene therapies. Success in these clinical trials was due to combining the favorable features of both the retina as a target organ and adeno-associated virus (AAV) as a vector. The retina offers several advantages for gene therapy approaches. It is an anatomically defined structure that is readily accessible for therapy and has some degree of immune privilege, making it suitable for application of viral vectors. AAV, on the other hand, is a non-pathogenic helper dependent virus that has little immunogenicity. This viral vector transduces quiescent cells efficiently and thanks to its small size diffuses well in the interneural matrix, making it suitable for applications in neural tissue. Building on this initial clinical success with LCA II, we have now many opportunities to extend this proof-of-concept to other retinal diseases."

According to the news editors, the research concluded: "This article will discuss what are some of the most imminent targets for such therapies and what are the challenges that we face in moving these therapies to the clinic."

For more information on this research see: Gene therapy for inherited retinal degenerations. Comptes Rendus Biologies, 2014;337(3):185-192. Comptes Rendus Biologies can be contacted at: Elsevier France-Editions Scientifiques Medicales Elsevier, 23 Rue Linois, 75724 Paris, France. (Elsevier -; Comptes Rendus Biologies -

The news editors report that additional information may be obtained by contacting D. Dalkara, Academy Sci, Inst France, F-75006 Paris, France (see also Biotechnology).

Keywords for this news article include: Biotechnology, Paris, France, Europe, Gene Therapy, Bioengineering, Clinical Trials and Studies

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Source: Gene Therapy Weekly

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