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Newsletter January 2016

News: Stanford bringing gene editing to patients with deadly diseases
Tiny vials of recently repaired blood cells are thriving in a Stanford incubator, proof that a powerful new gene-editing technique is fixing errant genes that cause so much human suffering. Until recently, gene therapy was laborious, crude and unsafe for human testing. But the new technology, called CRISPR-Cas9, acts as a microscopic scalpel, performing genomic surgery with a precision, efficiency and affordability once thought unimaginable. The research being done at the Stanford School of Medicine, led by Dr. Matthew Porteus, is part of an accelerating research movement made possible using the new technique to try to cure genetic diseases such as sickle cell anemia and muscular dystrophy. These labs are steadily advancing through cell-based and animal trials, as fledgling biotech companies raise large sums of money needed to bring the therapies to market.

News: UniQure Announces Preliminary Topline Results from Low-Dose Cohort in Hemophilia B Phase I/II Gene Therapy Clinical Trial
uniQure N.V. (Nasdaq: QURE), a leader in human gene therapy, today announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure’s novel AAV5/FIX gene therapy, AMT-060. All five patients in the low-dose cohort had Factor IX (FIX) phenotypic features of severe or moderately-severe hemophilia including documented Factor IX (FIX) levels less than 1-2% and required chronic treatment with prophylactic recombinant FIX (rFIX) therapy at the time of enrollment.

News: CRISPR Gene Therapy Has Cured a Mouse With Muscular Dystrophy
For the first time, a living mammal was cured of a genetic disease with a treatment we could use on humans.Continuing mankind’s formidable strut towards an age of gods and monsters, for the first time ever researchers have successfully treated a genetic disease in a living mammal with a method that could be used on humans. Thanks to CRISPR — the incredibly powerful genetic engineering process — and some bioengineers at Duke University, an adult mouse with muscular dystrophy will enter 2016 much healthier. The findings are out today in a paper at Science.

News: Zika Virus information website launched
Zika virus is an insect-borne virus, of the flavivirus genus, that is spread by Aedes mosquitoes. The most common symptoms of Zika virus disease (Zika) are fever, rash, joint pain, and red eye. The illness is usually mild with symptoms lasting from several days to a week. The symptoms of Zika are similar to those of dengue and chikungunya, which are diseases caused by other viruses spread by the same type of mosquitoes. The virus spreads now rapidly through South-America and reached already Texas. More information and recent news can be found on a recent launched website: Zika Virus Net at The objectives of Zika Virus Net are to be the public and professional information resource for zika fever and to serve as a network in the exchange of information and news related to zika.

Conference: Symposium and educational session of the Netherlands Society of Gene and Cell Therapy (NVGCT)
The 2016 NVGCT Spring Symposium will be held on March 10 and 11, 2016 in Lunteren, The Netherlands. The theme is gene delivery. Several stablished keynote speakers have already confirmed their presence. In addition several educational lectures will be presented. Register now before 31 January 2016.
Recent clinical successes have boosted the interest for Gene Therapy. The number of trials both for acquired and inherited diseases will increase significantly in the near future. Because of this and the changes in legislation the NVGCT has decided to also organize an educational session in collaboration with UniQure, the COGEM, Bureau GGO, ARM and the CCMO, on how to fill in the applications to help your with the obtaining the permits in an efficient way and to start performing your gene therapy trial in the Netherlands without delays.


- 25 - 27 January 2016, Phacilitate Cell & Gene Therapy World 2016, Washington DC, MD
- 3 - 4 March 2016, 18th International Conference on Cell and Gene Therapy - ICCGT 2016, Singapore
- 7 - 9 March 2016, International Society for BioProcess Technology 6th Spring Meeting - Viral vectors and vaccines, Washington, DC
- 9 - 11 March 2016, French Society of Cell and Gene Therapy (SFTCG) Annual Congress, Marseille, France
- 10 - 11 March 2016, Spring Symposium of the Netherlands Society of Gene & Cell Therapy (NVGCT), Lunteren, The Netherlands
- 15 April 2016, Annual conference of the British Society for Gene and Cell Therapy (BSGCT), London, UK
- 4 - 7 May 2016, American Society of Gene and Cell Therapy (ASGCT) 19th Annual Meeting, Washington, DC
- 19 – 21 May 2016, 5th International Conference and Exhibition on Cell & Gene Therapy, San Antonio, USA
- 25 - 28 May 2016, International Society for Cellular Therapy (ISCT) 22th Annual Meeting, Singapore
- 20 - 22 April 2016, World Orphan Drug Congress USA 2016, New York, NY
- 28 – 30 July 2016, The 22th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokyo, Japan
- 18 – 21 October 2016, XXIV Congress of the European Society of Gene and Cell Therapy (ESGCT) and the International Society for Stem Cell Research, Florence, Italy
- 10 - 13 May 2017, American Society of Gene and Cell Therapy (ASGCT) 20th Annual Meeting, Washington, DC
- 27 - 28 November 2017, 19th International Conference on Cell and Gene Therapy - ICCGT 2017, Istanbul, Turkey


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. Other information websites created and maintained by the owner of Gene Therapy Net are Dengue Virus Net, Influenza Virus Net, Ebola Virus Net, Chikungunya Virus Net and Zika Virus Net.
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