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Updated: Aug 13, 2025

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells
Published on: October 6, 2023
Are we prepared to deliver gene-targeted therapies for rare diseases?
Timothy W Yu1, Stephen F Kingsmore2, Robert C Green3
1Division of Genetics and Genomics, Harvard Medical School, Boston, Massachusetts, USA.
Abstract:
The cost and time needed to conduct whole-genome sequencing (WGS) have decreased significantly in the last 20 years. At the same time, the number of conditions with a known molecular basis has steadily increased, as has the number of investigational new drug applications for novel gene-based therapeutics. The prospect of precision gene-targeted therapy for all seems in reach… or is it? Here we consider practical and strategic considerations that need to be addressed to establish a foundation for the early, effective, and equitable delivery of these treatments.
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