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Newsletter February 2013

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News: Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases
In the latest issue of the journal Molecular Therapy, Professor Jacques P. Tremblay (president of the Association of Gene Therapy of Quebec and researcher in the Research center of the Centre Hospitalier Universitaire (CHU) of Quebec) launches a call - with 50 other world experts in gene therapy - for the creation of an International Gene Therapy Consortium for Monogenic Diseases. The bases of this consortium will be established during a workshop, which will be held during the congress of the American Society of Gene and Cell Therapy (ASGCT) in Salt Lake City in May 2013.
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News: Another Bump in the Road to Gene Therapy?
By Ricki Lewis - I am astonished, once again, by the complexity and unpredictability of science. Last week, a paper in the Proceedings of the National Academy of Science (PNAS) reported that gene therapy to treat a form of blindness called Leber congenital amaurosis type 2 (LCA2) doesn’t stop degeneration of the rods and cones – the photoreceptor cells that provide vision. Gene therapy sends genetic instructions for a protein called RPE65 into a layer of cells that supports the rods and cones – the retinal pigment epithelium, or RPE. The protein is essential for the eye to use vitamin A. And the gene therapy works, so far.
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News: New Gene Therapy Extends Lives Of Children With Rare Brain Disorder
Chalk up another victory for gene therapy. A recent clinical trial has shown that gene therapy can be used to extend the lives of children with a rare brain disorder that typically proves fatal in the first few years of life. The oldest child to benefit from this reversal of fortune has reached an amazing 18 years of age.
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News: Positive gene therapy results in large mammals of Duchenne muscular dystrophy
Usually, results from a new study help scientists inch their way toward an answer whether they are battling a health problem or are on the verge of a technological breakthrough. Once in a while, those results give them a giant leap forward. In a preliminary study in a canine model of Duchenne muscular dystrophy (DMD), University of Missouri scientists showed exactly such a leap using gene therapy to treat muscular dystrophy. The results of the study will be published in the journal Molecular Therapy on Jan. 15, 2013.
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Site Update: Gene Therapy Legislation in Europe
The section regarding the gene therapy legislation in Europe has recently been extensively updated. The clinical trail regulations and market registration procedure by the EMA have been rewritten and is up to date now. Special attention has been given to the Advanced Therapy Medicinal Products (ATMPs) regulation and the role of the Committee for Advanced Therapies (CAT) and Committee for Medicinal Products for Human Use (CHMP).
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Conference: NVGCT Spring Symposium March 15 & 16, 2013
The 2013 Spring Symposium of the Netherlands Society of Gene & Cell Therapy will be held on March 15 & 16, 2013. Venue: Congrescentrum De Werelt, Lunteren The Netherlands.

Confirmed speakers: Jörn Aldag (uniQure) & Dick van Bekkum (Cinderella therapeutics). Keynote speaker: Bobby Gaspar (UCL Institute of Child Health, London, UK), Gene therapy for primary immune deficiencies.


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Conferences
- 28 February - 2 March 2013, 19th Annual Meeting German Society for Gene Therapy (DG-GT e.V.), Munich, Germany
- 15 - 16 March 2013, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 22 March 2013, French Society of Cell and Gene Therapy (SFTCG) Annual Congress 2013, Paris, France
- 17 - 19 April 2013, Annual conference of the British Society for Gene Therapy (BSGT), London, UK
- 22 April 2013, 8th Annual Conference of The Israeli Society of Gene Therapy (ISGCT), Jerusalem, Israel
- 22 - 25 April 2013, International Society for Cellular Therapy (ISCT) 19th Annual Meeting, Auckland, New Zealand
- 5 - 10 May 2013, Gordon Research Conference: Virusses & Cells, Lucca (Barga), Italy
- 8 – 10 May 2013, 8th Australasian Gene Therapy Society Meeting (AGTS), Sydney, Australia
- 15 – 18 May 2013, American Society of Gene and Cell Therapy (ASGCT) 16th Annual Meeting, Salt Lake City, Utah
- 4 – 6 July 2013, The 19th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Okayama, Japan
- 12 – 14 August 2013, International Conference on Genetic Engineering & Genetically Modified Organisms, Raleigh, North Carolina
- 23 – 25 October 2013, 2nd International Conference and Exhibition on Cell & Gene Therapy, Orlanda, Florida
- 24 – 28 October 2013, VII Congress of the Spanish Society of Gene (SETGyC) and Cell Therapy, and XXI Congress of the European Society of Gene and Cell Therapy (ESGCT), Madrid, Spain
- 20 – 22 November 2013, 12th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California

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