Q » What are the recent breakthroughs in gene therapy for inherited diseases?

David

17 Oct, 2025

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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.

Michael

17 Oct, 2025

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A »Recent breakthroughs in gene therapy have shown promising results for inherited diseases, such as the use of CRISPR-Cas9 technology to edit faulty genes directly. Exciting advances include successful treatments for conditions like sickle cell anemia and certain types of congenital blindness, which offer hope for a wide range of genetic disorders. As research continues, these therapies may transform the future of genetic medicine by providing more effective, tailored treatments.

William

17 Oct, 2025

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A »Recent breakthroughs in gene therapy for inherited diseases include CRISPR-Cas9 gene editing, which has shown promise in treating sickle cell anemia and muscular dystrophy. Viral vectors have also improved, enabling more efficient gene delivery. Additionally, ex vivo gene therapy has successfully treated severe combined immunodeficiency and other genetic disorders.

James

17 Oct, 2025

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