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Newsletter February 2016

News: U.S. Intelligence Official Calls Gene Editing a Threat
That’s according to James Clapper, U.S. director of national intelligence, who on Tuesday, in the annual worldwide threat assessment report of the U.S. intelligence community, added gene editing to a list of threats posed by “weapons of mass destruction and proliferation.” Gene editing refers to several novel ways to alter the DNA inside living cells. The most popular method, CRISPR, has been revolutionizing scientific research, leading to novel animals and crops, and is likely to power a new generation of gene treatments for serious diseases. It is gene editing’s relative ease of use that worries the U.S. intelligence community, according to the assessment. “Given the broad distribution, low cost, and accelerated pace of development of this dual-use technology, its deliberate or unintentional misuse might lead to far-reaching economic and national security implications,” the report said.

News: Cell and gene therapy in Cambridge, London and Oxford: an invitation to collaborate
The golden triangle is the world-leading life sciences cluster of Cambridge, London, Oxford and the greater south east region of England. It comprises multiple award-winning research institutions, thousands of talented scientists, deep experience in clinical trials, and a thriving global business and life sciences community. Key to the region’s success has been its collaborative mindset – a drive to embrace and exchange ideas with scientists and professionals from around the world. This report features Life Sciences Minister George Freeman, the world’s first dedicated life sciences minister. Along with case studies and interviews with high profile academics and decision-makers, this report will help to understand the current cell and gene research climate in London.

News: British researchers get green light to genetically modify human embryos
Britain’s first genetically modified human embryos could be created within months, after scientists were granted permission by the fertility regulator to carry out the procedure. The Human Fertilisation and Embryology Authority (HFEA) regulator approved a licence application by Kathy Niakan, a stem cell scientist at the Francis Crick Institute in London, to perform so-called genome editing on human embryos. The decision permits Niakan to study the embryos for 14 days for research purposes only. It does not permit them to be implanted into women. Niakan’s research is aimed at finding the genes at play in the early days of human fertilisation.

News: AGTC and BCM Families Foundation Announce Collaboration to Develop AAV-Based Gene Therapy for Blue Cone Monochromacy
Applied Genetic Technologies Corporation (Nasdaq:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare eye diseases, and the BCM Families Foundation, a non-profit organization focused on eradicating Blue Cone Monochromacy (BCM), today announced a collaboration to develop an AAV-based gene therapy for the disease. Blue Cone Monochromacy, also known as X-linked achromatopsia, is a rare genetic disease of the retina that almost exclusively affects males. It is a hereditary condition linked to the X chromosome that manifests with a partial dysfunction of the cones of the retina. BCM can result in reduced visual acuity, impaired color vision, photosensitivity, myopia and infantile-onset nystagmus. These manifestations are similar to those in achromatopsia, caused by mutations in the CNGB3 or CNGA3 gene, for each of which AGTC has ongoing clinical development activities.

Site update: Gene Editing Tools Explained
Gene editing, or genome editing with engineered nucleases is a type of genetic engineering in which DNA is inserted, deleted or replaced in the genome of an organism using engineered nucleases, or "molecular scissors." These nucleases create site-specific double-strand breaks (DSBs) at desired locations in the genome. The induced double-strand breaks are repaired through nonhomologous end-joining (NHEJ) or homologous recombination (HR), resulting in targeted mutations ('edits').There are three families of engineered nucleases being used that are now explained on Gene Therapy Net: Zinc finger nucleases (ZFNs), Transcription Activator-Like Effector-based Nucleases (TALENs), and CRISPR-Cas system

- 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
- 20 - 22 April 2016, ESGCT Spring School,Hannover, Germany
- 20 - 22 April 2016, World Orphan Drug Congress USA 2016, New York, NY
- 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
- 14 -16 September 2016, 22th Annual Meeting of the German Society for Gene Therapy (DG-GT e.V.), Heidelberg, Germany
- 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
- 16 – 18 November 2016, 15th Annual Gene Therapy Symposium for Heart, Lung, and Blood Diseases, Sonoma, California
- 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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