Dr. Alex Kolevzon on Gene Therapy, SHANK3 & the Future of Treatment
Dr. Alexander Kolevzon
Clinician-scientist focused on neurodevelopmental disorders
Welcome to Generation Hope. In this wide-ranging interview, Dr. Alex Kolevzon (Mount Sinai) sits down with Ron to discuss the science, the trials, and the human side of gene therapy for rare neurodevelopmental disorders. In this episode we cover: • Dr. Kolevzon’s path into autism research and why focusing on genetics (like SHANK3) matters. • The evolution from symptom-based treatments to gene-targeted approaches. • Clinical progress: what early trials look like (design, dosing, age cohorts) and how behavioral therapies will work alongside gene therapy. • Biomarkers and outcome measures — EEG, imaging, and how those tools help stratify patients. • Neonatal sequencing and the ethical questions around early genetic screening. • Real family perspectives and why these advances could be transformative for children and adults living with Phelan-McDermid, Rett, STXBP1, Angelman and more. • A cautiously optimistic take on timelines and the potential societal impact — what a future with disease-modifying therapies might look like. If you care about gene therapy, rare genetic disorders, or the future of neurodevelopmental medicine, this conversation offers both scientific insight and real-world hope. 📌 Visit our site for more interviews, updates, and resources: GENERationHope.co 👍 Like this video if you found it helpful and subscribe to be notified when new interviews drop. 🔔 Hit the bell to get updates about the documentary and Gene Therapy breakthroughs.
Key topics
- clinical trials
- SHANK3
- gene therapy
- outcome measures
Chapters
- 1. Introduction
- 2. Main conversation
- 3. What families should understand
Transcript
Transcript coming soon. This page is already connected to the published YouTube interview, and the edited transcript can be added here for search and accessibility.
Related explainers and articles
What Is Gene Therapy?
Gene therapy is a treatment approach that tries to address disease at the level of the gene, often by adding, replacing, silencing, or editing genetic instructions.
What Is Phelan-McDermid Syndrome?
Phelan-McDermid Syndrome is a rare genetic condition often linked to changes involving SHANK3 on chromosome 22.
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