Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways.
Details
Serval ID
serval:BIB_6FC559463B37
Type
Article: article from journal or magazin.
Publication sub-type
Review (review): journal as complete as possible of one specific subject, written based on exhaustive analyses from published work.
Collection
Publications
Institution
Title
Central Nervous System Gene Therapy: Present Developments and Emerging Trends Accelerating Industry-Academia Pathways.
Journal
Human gene therapy
ISSN
1557-7422 (Electronic)
ISSN-L
1043-0342
Publication state
Published
Issued date
09/2022
Peer-reviewed
Oui
Volume
33
Number
17-18
Pages
913-922
Language
english
Notes
Publication types: Journal Article ; Research Support, Non-U.S. Gov't
Publication Status: ppublish
Publication Status: ppublish
Abstract
The recent success of first central nervous system gene therapies has reinvigorated the growing community of gene therapy researchers and strengthened the field's market position. We are witnessing an increase of clinical trials with long-term efficiency mainly for neurometabolic, neurodegenerative, and neurodevelopmental diseases caused by loss-of-function mutations. The ever-expanding knowledge and accessibility to the most advanced tools allow enrichment of applications to more complex diseases. This gradually contributes toward sealing the gap between top diseases impacting current global health and those toward which gene therapy development is currently aimed. In this study, we highlight innovative therapeutic approaches that have reached the clinics and outline the latest improvements of vector design and targeting. Finally, we address the pressing challenges faced by clinical trials and the direction they are heading.
Keywords
Central Nervous System, Genetic Therapy, brain disease, central nervous system, clinical trial, gene therapy, viral vectors
Pubmed
Web of science
Create date
13/09/2022 9:50
Last modification date
31/03/2023 7:11