Q » What are the recent breakthroughs in gene therapy for inherited diseases?
17 Oct, 2025
A » Recent breakthroughs in gene therapy for inherited diseases include the development of CRISPR-Cas9 technology, which allows precise editing of DNA, and the approval of new treatments like Luxturna for inherited retinal diseases and Zolgensma for spinal muscular atrophy. These advances have significantly improved the potential to correct genetic defects at their source, offering hope for effective and long-lasting treatments for previously untreatable conditions.
17 Oct, 2025
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