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Development of AAV-Mediated Gene Therapy Approaches to Treat Skeletal Diseases

27 Mar 2024

Abstract excerpt

Adeno-associated viral (AAV) vectors have emerged as crucial tools in advancing gene therapy for skeletal diseases, offering the potential for sustained expression with low postinfection immunogenicity and pathogenicity. Preclinical studies support both the therapeutic efficacy and safety of these vectors, illustrating the promise of AAV-mediated gene therapy. Emerging technologies and innovations in AAV-mediated...

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PMID: 38534217
DOI: 10.1089/hum.2024.022

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