Advancing CRISPR genome editing into gene therapy clinical trials: progress and future prospects.
Level 5 - mechanism / opinion, no new human data
Narrative review without systematic methodology or original data
PubMed 40160040 · doi:10.1017/erm.2025.10
What was done
This narrative review summarizes the development, clinical translation, and future directions of CRISPR genome-editing technologies (including CRISPR-Cas9, base editing, and prime editing) for human therapeutic applications across monogenic disorders, cancer, and infectious diseases.
What was found
The abstract reports no quantitative data or numerical outcomes. It notes the regulatory milestone of FDA approval for Casgevy (autologous CD34+ hematopoietic stem cell editing for sickle cell disease), ongoing clinical trials for transfusion-dependent beta-thalassaemia, and emerging research in oncology and HIV. Key ongoing challenges identified include off-target effects, suboptimal delivery systems, scalability, long-term safety, and ethical concerns regarding germline modifications.
Why it matters
The review outlines the state of clinical translation for genome-editing technologies as they progress from theoretical concepts to approved human therapeutics, highlighting current clinical milestones alongside remaining technical hurdles.
Limits
This is a narrative review providing no primary clinical trial data, quantitative synthesis, or formal risk-of-bias evaluation. No specific study counts, participant numbers, or efficacy metrics are reported in the abstract.
Cited by
- supports CRISPR gene editing is being evaluated in clinical trials as a potential cure for HIV infection.