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Newsletter July 2010

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News: Study Shows Hope for Gene Therapy
Researchers have launched a new gene-therapy trial for children with a rare disease known as "bubble boy syndrome," reflecting fresh hopes that the strategy of delivering working genes can be used to treat many intractable ailments. In the new study, sponsored in the U.S. by investigators at Children's Hospital Boston and expected to open at five sites around the world, scientists plan to enroll 20 boys with SCID-X1, which stands for severe combined immunodeficiency, X-linked—a genetic condition that affects boys and leaves them unable to fight germs. Without treatment, which is currently possible only by bone-marrow transplantation, most children die before age one.

News: A Step Closer to Gene Therapy for HIV
In a first step toward a gene-based therapy for HIV, researchers have successfully modified stem cells to resist the virus and used them to treat patients. Two years after the stem cells were used in a pilot study with four patients, descendants of those modified cells could still be found in their blood and bone marrow, along with some of the anti-HIV proteins the engineered cells were designed to produce, according to John Rossi, PhD, of City of Hope in Duarte, Calif., and colleagues.

News: Oncolytic vaccine therapy shows promise in melanoma, SCC studies
Efficacy and safety results of phase 2 studies provide support for undertaking pivotal trials investigating an oncolytic vaccine therapy, granulocyte colony-stimulating factor (GM-CSF)-encoding oncolytic herpes simplex virus (OncoVEXGM-CSF, BioVex), for treating metastatic melanoma (MM) and head and neck squamous cell carcinoma (SCC). The herpes simplex virus on which OncoVEX-GM-CSF is based is genetically modified to replicate selectively in tumor cells and engineered to express a gene for GM-CSF, an immune-stimulating protein. The vaccine is injected into tumor deposits. As the virus replicates, it causes tumor cell lysis, leading to cell death and production of an immunogenic "soup" containing tumor antigens and GM-CSF. Through these mechanisms, the vaccine can eradicate the local tumor and serve as a vaccine matching each patient's tumor antigen profile that can induce a systemic immune response, which is, hopefully, sufficiently potent to eradicate tumor cells at distant, uninjected sites as well as to prevent future recurrences.

Conference: Advanced symposium and EMBO practical course, Viral Vectors in Gene Therapy
The course will offer a complete review of knowledge and technology on the adeno-associated (AAV)-, baculovirus (BV)- and lentiviral (LV)-based vectors for gene therapy.
With the first EMBO Eurolabcourse in Evry (France) in 2002 a series of courses was started with a new training format which combines a symposium open to all (plenary lectures, selected oral communications, poster sessions and round table discussions) with a practical course for 25-30 participants selected on application. This format allows maximal interactivity between the participants coming from research institutions of academia and industry, health organizations, regulatory agencies and patients associations from many countries worldwide. On one hand, the students and young researchers who participated in our previous courses implemented rapidly their career thanks to the new knowledge acquired. Many are now team leaders, invited speakers in the meetings and/or organizers of events in their countries. On the other hand, the industries got opportunities of new business in exploiting innovations from academia and extended their marketing area. More information can be found on the symposium web site

The Symposium, August 26 - 29, 2010 will present vectorology concepts, different vectors families, discuss their characteristics including transduction efficiency, host genome interaction, toxicity, perspectives of clinical applications and industrial/commercial exploitations.
Deadline for registration: July 31, 2010.

the EMBO Practical course, August 30 - September 4, 2010 laboratory sessions to 25 selected participants who will learn how produce, purify, and characterise AAV, BV and LV vectors including their biological properties and functional assessment in vivo

Advanced symposium and EMBO practical course: Viral Vectors in Gene Therapy

- 26 August - 4 September 2010, Viral Vectors in Gene Therapy: Applications and Novel Production Methods, A.I. Virtanen Institute, Kuopio, Finland
- 24 - 27 September 2010, Cellular Therapy of Cancer Symposium - ATTACK Project Meeting, Montpellier, France
- 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
- 16 - 19 March 2011, 6th International Conference on Oncolytic Viruses as Cancer Therapeutics, Las Vegas, NV
- 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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