CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications.
Level 3 - non-randomized controlled study
Systematic review of early-phase, primarily non-randomized human clinical trials
PubMed 41810550 · doi:10.1177/10430342251409714
What was done
A systematic review following PRISMA 2020 guidelines searched PubMed, Scopus, Web of Science, and the Cochrane databases through November 2024 for clinical trials assessing the efficacy, safety, and applicability of CRISPR/Cas9 gene editing across human diseases. Methodological quality was evaluated using the Cochrane risk of bias tool. A total of 17 clinical studies were included.
What was found
In nonmalignant hematological diseases (sickle cell disease and beta-thalassemia), CRISPR therapy led to high rates of transfusion independence and the elimination of vaso-occlusive crises. In malignant hematological conditions, CRISPR-edited CAR-T cell therapy achieved an 83.3% complete remission rate in B-cell acute lymphoblastic leukemia, alongside positive responses in B-cell non-Hodgkin lymphoma. In solid oncology (including lung cancer), CRISPR editing was safely implemented. In genetic disorders, CRISPR treatment improved visual function in retinal degeneration and decreased disease symptoms in both hereditary angioedema and transthyretin amyloidosis, with predominantly mild adverse events reported.
Why it matters
This review aggregates early human clinical trial data confirming that CRISPR/Cas9 technology provides measurable therapeutic utility across diverse genetic, oncologic, and hematologic indications. It establishes a benchmark of response rates and safety outcomes across published clinical studies.
Limits
Only 17 trials across varied disease categories were identified, reflecting small aggregate sample sizes characteristic of early-phase testing. Specific numerical efficacy and safety rates are largely omitted from the abstract aside from the 83.3% remission rate in B-cell ALL. Delivery optimization, immunogenicity, and long-term risks such as off-target edits remain key unresolved concerns.
Cited by
- supports CRISPR-engineered CAR T-cells are currently in clinical trials for solid tumors.