Viral Vectors in Gene Therapy

EP: 1.Overview of Gene Therapy Approaches and Impacts

EP: 2.Key Factors in Gene Vector Selection

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EP: 3.Viral Vectors in Gene Therapy

EP: 4.Navigating Gene Therapy Eligibility: The Role of Genetic Testing

EP: 5.Evaluating Transformative Gene Therapies

EP: 6.Shifting the Treatment Paradigm in Spinal Muscular Atrophy

EP: 7.Developments in Muscular Dystrophy Gene Therapy

EP: 8.Review of Clinical Evidence for Delandistrogene Moxeparvovec for DMD

EP: 9.Driving Clinical Insights: Harnessing Functional Assessments in DMD Research

EP: 10.Duchenne Muscular Dystrophy Treatment Landscape Review

EP: 11.Strategic Insights Into Evolving Gene Therapy Trends

EP: 12.Sustainable Gene Therapy Coverage Approaches

EP: 13.Interpreting Clinical Data to Guide Coverage Decisions for Rare Disease Therapies

EP: 14.Coverage of Gene Therapies: Balancing Cost, Clinical Impact, and Durability of Effect

EP: 15.Innovative Payment Models for Gene Therapies

EP: 16.Ethical Implications of Patient Access Restrictions

EP: 17.Enhancing Collaboration for Patient-Centered Care in Rare 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.