Exploring the Use of Adeno-Associated Virus for Gene Therapy

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Presentation transcript:

Exploring the Use of Adeno-Associated Virus for Gene Therapy

Types of Gene Therapy

Rationale for Gene Therapy

Current Gene Therapy Vectors

Adeno-Associated Virus vs Vector

Different AAV Serotypes

Benefits of AAV

Historical View of AAV

AAV Gene Therapies

Practical Application of AAV Therapy

Overview of Early AAV Clinical Trials

Ongoing AAV Clinical Trials

Use of AAV in Patients With Hemophilia B

Use of AAV in Patients With Hemophilia B Study Results

Use of AAV in Patients With SMA

Examining SMA Genetics

Examining SMA Genetics (cont)

Disease Course of SMA

Challenges of CNS Delivery of Current Treatments

Characteristics of AVXS-101

AVXS-101 Clinical Trial in SMA Type 1 Results

AVXS-101 Clinical Trial in SMA Type 1 Results (cont)

Challenges to AAV Gene Therapy

Importance of Multidisciplinary Approach

Complications of Immune Response

Immune Response of Factor IX

Strategies to Combat Immune Response

Follistatin Gene Therapy Trial Patients With Becker Muscular Dystrophy

Current Barriers Under Investigation

Key Elements for Successful Gene Therapy

Concluding Remarks

Abbreviations

Abbreviations (cont)