Publications by authors named "Kawthar M Alenezi"
Mol Biotechnol
January 2025
Article Synopsis
- The CRISPR-Cas9 system is a groundbreaking gene editing tool being researched for treating thalassemia and sickle cell disease (SCD).
- A study reviewed clinical trials from multiple databases, identifying 6 eligible studies involving 115 patients, which used CRISPR/Cas9 to target specific gene enhancers and promoters.
- Results showed that patients experienced increased fetal hemoglobin, improved hemoglobin levels, transfusion independence in thalassemia, and reduced pain episodes in SCD, indicating the potential of CRISPR/Cas9 as a one-time functional cure for these blood disorders.
