Translating cell therapies for neurodegenerative diseases: Huntington's disease as a model disorder.

ABSTRACT: There has been substantial progress in the development of regenerative medicine strategies for CNS disorders over the last decade, with progression to early clinical studies for some conditions. However, there are multiple challenges along the translational pipeline, many of which are common across diseases and pertinent to multiple donor cell types. These include defining the point at which the preclinical data are sufficiently compelling to permit progression to the first clinical studies; scaling-up, characterization, quality control and validation of the cell product; design, validation and approval of the surgical device; and operative procedures for safe and effective delivery of cell product to the brain. Furthermore, clinical trials that incorporate principles of efficient design a

SUBMITTER: Rosser AE 

PROVIDER: S-EPMC9166564 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature