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Cellectis Unveils a Non-Viral Gene Therapy Approach for Sickle Cell Disease in Nature Communications
Cellectis announced a breakthrough in gene therapy for Sickle Cell Disease (SCD) using a non-viral approach published in Nature Communications. The therapy employs TALEN® technology for precise HBB gene correction in hematopoietic stem and progenitor cells (HSPCs). This method achieves over 50% expression of normal hemoglobin in treated cells, effectively correcting the sickle cell phenotype without adverse effects. The preclinical data shows promising therapeutic potential and efficient engraftment in animal models, indicating readiness for clinical application. This innovative approach offers hope for addressing SCD and other genetic diseases.
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