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Viral Vectors in Gene Therapy

Opinion
Video

Emma Ciafaloni, MD, FAAN, describes the key differences among viral vectors used in gene therapy, highlighting their applications and strengths in targeting specific diseases.

This is a video synopsis/summary of a Peer Exchange involving Ryan Haumschild, PharmD, MS, MBA; Jessica Nance, MD, MS; Kimberly C. Chen, DO, MSHLM; Emma Ciafaloni, MD, FAAN; and Mary Pak, MD, FACP.

Ciafaloni provides an in-depth analysis of viral vectors used in gene therapy, focusing on retroviruses, adenoviruses, and adeno-associated viruses (AAVs). She contrasts their characteristics, such as integration into the genome, payload capacity, and tissue-targeting abilities. Retroviruses, like lentivirus, offer stable expression in both dividing and nondividing cells, suitable for diseases like hematologic disorders. Adenoviruses, being episomal, are advantageous for nondividing cells but pose challenges with immune responses and high-dose requirements. AAVs, with their efficient transduction and low immunogenicity, are promising for systemic delivery but face limitations in cargo size and redosing due to immune responses. Ciafaloni emphasizes the importance of selecting the right vector based on disease type and patient characteristics, acknowledging the ongoing efforts to improve vector efficiency, reduce toxicity, and lower manufacturing costs. Haumschild underscores the critical role of proper vector selection in optimizing gene therapy outcomes, underscoring the need for precision in patient identification and treatment strategies.

Video synopsis is AI-generated and reviewed by AJMC® editorial staff.

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