Gene therapy then and now: A look back at changes in the field over the past 25 years

2025/02/28 by Dan Wang, Gregg Stevens, Terence R. Flotte · 1 voice
Biochemistry, Genetics and Molecular Biology · #CRISPR and Genetic Engineering #RNA Interference and Gene Delivery #Virus-based gene therapy research

paper · doi:10.1016/j.ymthe.2025.02.040

openalex publication_date 2025/02/28 · openalex created_date 2025/10/10 · openalex updated_date 2026/08/03

Abstract

Since the inception of Molecular Therapy in 2000, the field of gene therapy has made remarkable progress, evolving from no approved clinical products to 23 clinical gene therapy products today. In this review, we aim to capture the transformative changes in the field by surveying the literature over this period, with a particular focus on advancements in gene delivery vector technology, disease and tissue targeting, and the revolutionary molecular tools that have become central to the field. We also discuss the current challenges facing gene therapy and the need for greater collaboration to ensure its accessibility worldwide.

Citations

• A Programmable Dual-RNA–Guided DNA Endonuclease in Adaptive Bacterial Immunity
• Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage
• Ribozyme-activated mRNA trans-ligation enables large gene delivery to treat muscular dystrophies

Discussions

• ➡️ Wang, D., Stevens, G., & Flotte, T. R. (2025b). Gene Therapy then and now: A look back at changes in the field over the past 25 years. Molecular Therapy. DOI: doi.org/10.1016/j.ym... [bsky, 0 points, 0 comments]

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