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Newsletter June 2010

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News: First-in-man gene therapy for advanced heart failure promising in small study
In the first clinical test of a gene therapy for heart failure, administration of a gene that upregulates an enzyme involved in myocardial contraction and relaxation appeared to improve symptoms, functional status, and ventricular volumes in patients with severe systolic heart failure. The small dose-finding study, reported here at the Heart Failure Congress 2010, sponsored by the Heart Failure Association of the European Society of Cardiology, is something of milestone and, its researchers say, paves the way for studying the novel treatment in larger clinical-outcomes trials.

News: Gene Therapy Reverses Muscle Weakness in MD
Patients with certain forms of muscular dystrophy (MD) may be able to achieve long-term restored muscle control with gene therapy, according to research presented Friday at the annual meeting of the American Society of Gene & Cell Therapy, held May 17 to 22 in Washington, D.C. Following a successful trial that showed gene expression in patients three months after receiving a genetic transfer, Jerry R. Mendell, M.D., of Nationwide Children's Hospital in Columbus, Ohio, and colleagues conducted a randomized controlled trial of three patients with limb girdle MD (LGMD) in which the extensor digitorum brevis muscle received genetic therapy while the control side received saline to evaluate whether long-term gene expression was achievable.

News: Gene Therapy Plus Chemotherapy Ups Odds for Success in Treating 'Bubble Boy' Disease
Gene therapy combined with chemotherapy may lead to immune system improvement in patients with bubble boy disease, according to a study to be presented at the American Society of Gene & Cell Therapy 13th Annual Meeting. Beginning in 2001, researchers from the University of California Los Angeles and the National Human Genome Research Institute, a division of the National Institutes of Health, treated 10 patients with adenosine deaminase severe combined immunodeficiency (ADA-SCID), commonly referred to as bubble boy disease.

- 13 - 15 June 2010, The 9th Annual Congress of La Société Francophone de Thérapie Cellulaire et Génique (SFTCG), Paris, France
- 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
- 26 August - 4 September 2010, Viral Vectors in Gene Therapy: Applications and Novel Production Methods, A.I. Virtanen Institute, Kuopio, Finland
- 27 - 29 September 2010, International Society for Cell and Gene Therapy of Cancer (ISCGT) Congress, Doha, Quatar
- 6 - 9 October 2010, 17th Annual Meeting German Society for Gene Therapy (DG-GT e.V.), Munich, LMU Campus Grosshadern, Germany
- 7 October 2010, COGEM Symposium: GM viruses as medicine: panacea or pandora's box?, Amsterdam, The Netherlands
- 22 - 25 October 2010, European Society of Gene and Cell Therapy (ESGCT) 18th Annual Congress, Milan, Italy
- 17 - 19 November 2010, 9th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases. Focus Topic: Gene Expression, Sonoma, CA
- 24 - 26 January 2011, Phacilitate 7th Annual Cell & Gene Therapy Forum 2011, Washington DC, MD
- 4 - 6 May 2011, Australasian Gene Therapy Society (AGTS) 7th Meeting, Melbourne, Australia
- 18 - 22 May 2011, American Society of Gene and Cell Therapy (ASGCT) 14th Annual Meeting, Seattle, WA
- 29 May - 2 June 2011, Gordon Research Conference: Virusses & Cells, Lucca (Barga), Italy
- 27 - 31 October 2011, British Society of Gene Therapy (BSGT) 8th Annual Conference, Brighton, UK


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