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Children's Hospital of Philadelphia Researchers Develop Promising Tools to Enhance Gene Therapy Delivery for Hard-to-Treat Diseases

Adeno-associated viral (AAV) vectors are promising tools that can transport modified genetic material into the nuclei of cells in target tissues impacted by challenging diseases, offering the possibility of a one-time precision therapy to inherited diseases.

May 20, 2025


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Angell Street Psychiatry
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