Dr. Guoping Feng (MIT) on SHANK3 Gene Therapy, Brain Disorders, and What’s Coming Next
Dr. Guoping Feng
MIT neuroscientist and genetic medicine researcher
About this conversation
Welcome — I’m Ron Kleiman, and this is GENEration Hope. In this episode, I’m joined by Dr. Guoping Feng, Professor of Neuroscience at MIT, affiliated with the McGovern Institute, the Yang Tan Collective, and the Broad Institute. 
We talk about: • Why he left medicine to pursue research that could lead to treatments for kids  • The urgency of moving faster — because “kids are growing up every day”  • Gene therapy delivery, the blood–brain barrier, and what’s changing with new vectors  • How AI and machine learning are speeding up vector design and behavioral testing  • The miniSHANK3 approach: why the full SHANK3 gene is too large, how a mini gene is designed, and how it’s delivered  • Where genome editing (base editing / prime editing) may fit in the future  If conversations like this help, please subscribe, like, and share — it’s the best way to support GENEration Hope.  This content is for informational purposes only and is not medical advice.
Key topics
- SHANK3 biology
- brain disorders
- gene therapy
- clinical translation
Transcript
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Download transcriptRelated 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.
What AI Drug Discovery Could Mean for Rare Disease Families
AI drug discovery is entering a new phase: not just better software, but a new industrial stack linking frontier models, pharma data, robotics, and real experiments.
Why it matters: Rare disease research often starts with small datasets, limited funding, urgent timelines, and difficult trial design. AI-linked discovery systems could help researchers generate stronger candidates and better experiments faster, but families should watch for clinical validation, access, manufacturing, and clear evidence rather than hype.
Source: GENEration Hope editorial analysis
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