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Newsletter September 2009

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News: The Second Coming of Gene Therapy
For years, gene therapy produced tons of hype but no results. Recently, though, new approaches have yielded its first successes: breakthrough treatments for blindness, cancer, and the deadly bubble boy disease. “For the first two years of her life, my daughter, Katlyn, was knocking on heaven’s door every day,” says Daisy Demerchant, a 26-year-old mom living in Centreville, New Brunswick, just north of Maine. “Two months after she was born she started getting sick, and she never got better.” At six months Katlyn was diagnosed with “bubble boy” disease, formally known as severe combined immunodeficiency (SCID), which robs the immune system of the ability to fight infection. There are many causes of this disorder; in Katlyn’s case it was lack of the enzyme adeno­sine deaminase, or ADA, which rids the body of a natural toxin called deoxyadenosine. When the toxin builds up, it destroys T and B lymphocytes, the body’s infection-fighting immune cells. As a result, Katlyn’s immune cells were dying.
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News: Scientists Construct 'Off Switch' For Parkinson Therapy
A common antibiotic can function as an "off switch" for a gene therapy being developed for Parkinson's disease, according to University of Florida researchers writing online in advance of September's Molecular Therapy. The discovery in rats answers an important question — how can new, therapeutic genes that have been irrevocably delivered to the human brain to treat Parkinson's be controlled if the genes unexpectedly start causing problems? Meanwhile, in a review of Parkinson treatments, the researchers say that prior experimental attempts using growth factors — naturally occurring substances that cause cells to grow and divide — to rescue dying brain cells may have failed because they occurred too late in the course of the disease. Together, the findings suggest that gene therapy to enable the brain to retain its ability to produce dopamine, a neurotransmitter that falls in critically short supply in Parkinson's patients, could be safely attempted during earlier stages of the disease with an added likelihood of success.
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Conferences
- 14 September 2009, Regulation of Advanced Therapy Medicinal Products in Europe, from Clinic to Market (Cell Therapy, Gene Therapy, Tissue Engineered Products), San Francisco, CA
- 2 -4 November 2009, Viral Vectors and Vaccines 16th International Meeting: For Pandemic Viruses, Cancer Vaccines, and Gene Therapy, New Orleans, LO
- 18 - 20 November 2009, 8th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, CA
- 18 - 21 November 2009, In Vivo Barriers to Gene Delivery, Cold Spring Harbor, NY
- 21 - 25 November 2009, European Society of Gene and Cell Therapy (ESGCT) 17th Annual Congress, Hannover, Germany
- 25 - 27 January 2010, Phacilitate Cell & Gene Therapy Forum 2010, Washington DC, MD
- 2 - 4 March 2010, DNA Vaccines 2010, New Orleans, LA
- 19 - 21 March 2010, British Society of Gene Therapy (BSGT) 7th Annual Conference, Royal Holloway University London, UK
- 19 – 23 May 2010, American Society of Gene and Cell Therapy (ASGCT) 13th Annual Meeting, Washington DC, MD
- 22 – 26 May 2010, International Society for Cellular Therapy (ISCT) 16th Annual Meeting, Philadelphia, PA
- 26 - 29 June 2010, 3nd International symposium on Cellular Delivery of Therapeutic Macromolecules, Cardiff, UK
- 1 - 3 July 2010, The 16th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tochigi-ken Sougou Bunka Center, Utsunomiya-city Tochigi, Japan
- 22 - 25 October 2010, European Society of Gene and Cell Therapy (ESGCT) 18th Annual Congress, Milan, Italy
- November 2010, Viral Vectors and Vaccines 17th International Meeting, Point Clear, AL

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