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Challenges and Opportunities in Advancing Gene Therapy: Insights from FDA Roundtable
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Challenges and Opportunities in Advancing Gene Therapy: Insights from FDA Roundtable
The story of KJ Muldoon, a 10-month-old infant whose life-threatening CSP1 deficiency was stabilized through a custom CRISPR therapy, highlights the transformative potential of genetic medicine. This personalized treatment allowed KJ to resume a normal diet without requiring a liver transplant, exemplifying the promise of genetic therapies to treat rare diseases. Despite such successes, the gene therapy field faces significant challenges, as dis…
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