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While the actual clinical application of gene therapies still faces challenges 304 such as potential toxicity, immunogenicity, and delivery efficiency, the approval of these biologics for clinical trials highlights the growing recognition and validation of AAV-mediated gene therapy as a promising and innovative treatment approach

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While convenient, these services typically charge $200-$400+ monthly, provide limited personalized medical supervision, offer no local support or accountability, and may source from compounding pharmacies of uncertain quality

doi: 10.1001/jama.2024.18581 49 DandonaPChaudhuriAGhanimH