Next Generation Strategies for Gene-Targeted Therapies of Central Nervous System Disorders Workshop

September 26, 2019 - September 27, 2019

Contact: Ling Wong
Contact Email: ling.wong@nih.gov
Location:

Bethesda North Marriott Hotel and Conference Center
Meeting Room: White Oak B
5701 Marinelli Road
North Bethesda, MD 20852


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NINDS Gene Therapy Workshop flyer

Recent advances in the gene therapy field have contributed to renewed interest in gene replacement, gene silencing, and genome editing as approaches to gene-targeted therapeutic strategies. This workshop will bring together a multi-disciplinary group of experts from academia, government, and industry to identify key challenges/gaps and opportunities, and to provide insight on how NIH could facilitate these next generation strategies for patients with neurological or neuromuscular disorders.

Related Resources

Speaker ListAgenda

Recent advances in the gene therapy field have contributed to renewed interest in gene replacement, gene silencing, and genome editing as approaches to gene-targeted therapeutic strategies.

This workshop brought together a multi-disciplinary group of experts from academia, government, industry, and patient advocacy. Participants joined working group discussions in advance of the workshop to identify key challenges and knowledge gaps. During the meeting, they further detailed those challenges/gaps and suggested potential solutions. The four working groups focused on: 1) Control of expression level & location, 2) Improving delivery and distribution, 3) Enhancing pre-clinical models & manufacturing, and 4) Impacting patients. Participants discussed a variety of collaboration models and infrastructure needs, and suggested potential next steps for the research community, NINDS, and NIH.

Conclusions:
We will produce and disseminate a white paper summarizing the key discussion points and recommendations.

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