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Newsletter November 2009

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News: Gene Therapy Halts Brain Disease in Two Boys
Researchers have used a modified AIDS virus to halt a devastating brain disease in two young boys. The treatment, in which the virus delivered a therapeutic gene, marks the first time gene therapy has been successfully used against X-linked adrenoleukodystrophy (ALD)--a disorder that is always fatal if untreated. With this proof of principle, scientists hope versions of the AIDS virus engineered to carry different genes can now be applied to a variety of other diseases. ALD is caused by a defect in an X chromosome gene that produces a protein called ALD. Cells need this transporter protein to break down certain fats; without it, the fats build up and damage the myelin sheathing that protects nerves. In X-linked ALD, which strikes mainly boys, patients develop neurological symptoms such as seizures and loss of vision around age 6 to 8, and within months they become paralyzed, deaf, and eventually die.
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News: Gene Therapy Repairs Injured Human Donor Lungs For The First Time
For the first time, scientists in the McEwen Centre for Regenerative Medicine, University Health Network have successfully used gene therapy to repair injured human donor lungs, making them potentially suitable for transplantation into patients. This technique could significantly expand the number of donor lungs by using organs that are currently discarded, and improve outcomes after transplantation. In their pioneering work, a team of researchers led by Dr. Shaf Keshavjee, Senior Scientist at the McEwen Centre for Regenerative Medicine, University Health Network and Director of the Lung Transplant Program, University Health Network developed a technique of ex vivo gene delivery to donor lungs, before they are implanted into a recipient's body. The technique was shown to be simple and effective in improving lung function.
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News: One Shot Of Gene Therapy, And Children With Congenital Blindness Can Now See
Born with a retinal disease that made him legally blind, and would eventually leave him totally sightless, the nine-year-old boy used to sit in the back of the classroom, relying on the large print on an electronic screen and assisted by teacher aides. Now, after a single injection of genes that produce light-sensitive pigments in the back of his eye, he sits in front with classmates and participates in class without extra help. In the playground, he joins his classmates in playing his first game of softball.
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Gene Therapy Poll: What will be the future of gene therapy?
Gene Therapy Net started this poll a couple of weeks ago. Most voters think that gene therapy will be a vital part of future health care, while others are more sceptic. Those visitors of Gene Therapy Net think that it will work only for 'simple' inherited disease like X-SCID or that more time needed to evaluate potential. What do you think?
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Conferences
- 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

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25 - 27 January 2010, Phacilitate Cell & Gene Therapy Forum 2010, Washington DC, MD
- 1 February 2010, Lentiviruses Gene Therapy Workshop: A Joint Israeli/UK Academic Meeting, Jerusalem, Israel
- 2 - 4 March 2010, DNA Vaccines 2010, New Orleans, LA
- 11 - 12 March 2010, Dutch Society of Gene Therapy (NVGT) Spring Meeting, The Netherlands
- 19 - 21 March 2010, British Society of Gene Therapy (BSGT) 7th Annual Conference, Royal Holloway University London, UK
- 22 - 24 April 2010, 6th International Meeting on Gene Therapy of Arthritis and Related Disorders, London, UK
- 5 May 2010, 7th Annual Conference of the Israeli Society of Gene & Cell Therapy (ISGCT), Tel Aviv, Israel
- 17 – 18 May 2010, ASGCT Clinical Trials Training Course, Washington DC, MD
- 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

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Gene Therapy Net is the information resource for basic and clinical research in gene therapy, and the site serves as a network in the exchange of gene therapy information and breaking news items. Visitors can keep track of the latest scientific papers, conference announcements, gene therapy jobs, regulations and guidelines, www.genetherapynet.com