Gen terapisi

**Genome Editing for Sickle Cell Disease: CRISPR/Cas9 as a Potential Cure**

Sickle cell disease, a debilitating genetic disorder, is poised to be revolutionized by genome editing techniques like CRISPR/Cas9. This innovative approach holds promise for permanent treatment by targeting the root cause of the disease: faulty hemoglobin genes.

CRISPR/CAS9: A Revolutionary Tool for Lysosomal Storage Disease Treatment Lysosomal storage diseases (LSDs) are a group of rare genetic disorders characterized by the accumulation of toxic substances within cells due to defective lysosomal function. CRISPR/CAS9, a revolutionary gene editing technology, offers unprecedented potential for treating LSDs by precisely correcting the Devamı…