Gene Therapy Delivery
5 videos · last 6 months shuffled daily, then older videos newest first
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40:09 Next Steps After the Alector Trial in GRN FTD
Behavioral neurologist Dr. Chow explains for Cure GRN how clinical trials and endpoints work, what the completed Alector trial gave the GRN-related FTD field, what participants can learn after a trial and how to join a new one.
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43:43 CAR-T Therapy in Neuroimmune Disease
Dr. Barbara Willekens of the University of Antwerp explains CAR-T cell therapy, why it is being explored in autoimmune disease including emerging trials in NMOSD and MOGAD, and how patients are selected for trials, for SRNA.
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39:57 AAIC 2026 Findings for Familial Alzheimer's Families
Youngtimers reviews findings from AAIC 2026 and the DIAD Family Conference for familial Alzheimer's families, including results for tau drugs E2814 and diranersen, blood tests and prevention trials starting earlier.
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2:44 Pfizer on Gene Therapy and AAV Vectors for Rare Disease
Pfizer says 6,000 of the 7,000 known Rare Diseases are genetic in origin and discusses how gene therapy using AAV Vectors can potentially be standardized and streamlined to help a patient manage these genetic based diseases without ongoing treatment.
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19:37 AAV-Based Gene Therapy in Parkinson's Disease
Michael S. Okun, MD, and Joohi Jimenez-Shahed, MD, review AAV-based gene therapy strategies for Parkinson's disease, safety and durability, and endpoints and outcomes from ongoing gene therapy studies, in a Medscape education programme.