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Gene Therapy: Past, Present, and Future
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This program will include a discussion of off-label treatment and investigational agents not approved by the FDA for use in the United States.
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Overview of Hemophilia Treatment
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History of Gene Therapy in Hemophilia
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AAV Vector
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First Wave of AAV Clinical Trials
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First Wave of AAV Clinical Trials (cont)
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Advances Following the First Wave
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AAV8 Vector in Hemophilia B
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Host-Immune Response
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Innovations in Gene Therapy in Other Diseases
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Current Clinical Trials in Hemophilia B
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Current Clinical Trial in Hemophilia A
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The Future
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Gene Therapy and the Treatment Paradigm
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Gene Therapy and Children
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Applicability of the AAV Vector
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Ongoing Concerns
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Identifying Endpoints for Clinical Trials
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Other Technologies
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Concluding Remarks
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Abbreviations
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