Horae Gene Therapy Center
Genetics and Genomics | Nervous System Diseases | Therapeutics
The National Institute of Neurological Disorders and Stroke (NINDS) held a workshop titled "Next generation strategies for gene-targeted therapies of central nervous system (CNS) disorders" in September 2019 in Bethesda, MD, USA. The meeting brought together a multi-disciplinary group of experts in the field of CNS-directed gene-targeted therapy delivery from academia, industry, advocacy, and the government. The group was charged with identifying the key challenges and gaps in this evolving field, as well as suggesting potential solutions. The workshop was divided into four sessions: (1) control of level and location, (2) improving delivery and distribution, (3) enhancing models and manufacturing, and (4) impacting patients. Prior to the workshop, NINDS established working groups of key opinion leaders (KOLs) for each session. In pre-meeting teleconferences, KOLs were tasked with identifying the research gaps and key obstacles that delay and/or prevent gene-targeted therapies to move into the clinic. This approach allowed for the workshop to begin with problem-solving discussions and strategy development, as the key issues had been established. The overall purpose of the workshop was to consider knowledge gaps and potential strategies to inform the community around CNS gene-targeted therapies, including but not limited to researchers and funders.
AAV vectors, antisense oligonucleotides, central nervous system, gene-targeted therapies, ultra-rare diseases
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DOI of Published Version
Morris JA, Boshoff CH, Schor NF, Wong LM, Gao G, Davidson BL. Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary. Mol Ther. 2021 Dec 1;29(12):3332-3344. doi: 10.1016/j.ymthe.2021.09.010. Epub 2021 Sep 20. PMID: 34547463; PMCID: PMC8636163. Link to article on publisher's site
Molecular therapy : the journal of the American Society of Gene Therapy
Morris JA, Boshoff CH, Schor NF, Wong LM, Gao G, Davidson BL. (2021). Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary. Open Access Publications by UMass Chan Authors. https://doi.org/10.1016/j.ymthe.2021.09.010. Retrieved from https://escholarship.umassmed.edu/oapubs/4969
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