CRISPR in Medicine: A Systematic Review of Clinical Trials and Therapeutic Applications - PubMed
12 hours ago
- #Clinical Trials
- #Gene Editing
- #CRISPR
- CRISPR/Cas9 technology is a revolutionary tool in medicine with potential for treating various diseases.
- The systematic review evaluates efficacy, safety, and applicability of CRISPR/Cas9 in clinical trials.
- CRISPR successfully treated β-thalassemia and sickle cell disease, achieving high transfusion independence.
- In B-cell acute lymphoblastic leukemia, CRISPR-engineered CAR-T cells achieved an 83.3% complete remission rate.
- CRISPR-based CAR-T cells showed promising results in B-cell non-Hodgkin's lymphoma.
- CRISPR improved vision in retinal degeneration and reduced symptoms in hereditary angioedema and transthyretin amyloidosis.
- Challenges include optimizing delivery systems, minimizing off-target effects, and addressing ethical considerations.