Nat Rev Drug DiscovJClub
Translating base editors: a decade of progress from the bench to the clinic.
Nature Reviews Drug Discovery · · Review
Gaudelli, Komor
Abstract ↗AI summary
The abstract is read at the publisher; the summary is JClub's.
A decade after their discovery in 2016, base editors have evolved into versatile tools used in clinical trials for multiple human diseases, including sickle cell disease and leukaemias.
- Why it matters: Understanding the progress of base editors from research tools to therapeutic platforms highlights their potential to transform gene therapy and address unmet medical needs.
- What they did: The review traces the mechanistic development of base editors, including the creation of adenine and prime editors, and discusses their application in trials targeting diverse conditions.
- The result: This progress enables the potential for precise, chemical correction of genetic mutations in human patients, advancing the field toward widespread clinical use.
The findingWhy it mattersWhat they didThe result