UMMS Affiliation

Horae Gene Therapy Center

Publication Date


Document Type



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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This is an open access article under the CC BY-NC-ND license (

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

Journal/Book/Conference Title

Molecular therapy : the journal of the American Society of Gene Therapy

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Link to Article in PubMed

PubMed ID


Creative Commons License

Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License
This work is licensed under a Creative Commons Attribution-Noncommercial-No Derivative Works 4.0 License.