Gene Therapy
A plain-English guide to gene therapy in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
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Genetic Medicine
Gene therapy, RNA medicine, newborn genome sequencing, and the treatment platforms changing the future of rare disease.
Treatment platforms and care pathways
Each topic can connect to explainers, interviews, news updates, and future CMS entries.
RNA Therapy
A plain-English guide to rna therapy in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
ASO Therapy
A plain-English guide to aso therapy in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Exon Skipping
A plain-English guide to exon skipping in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Base Editing
A plain-English guide to base editing in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Prime Editing
A plain-English guide to prime editing in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Cell Therapy
A plain-English guide to cell therapy in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Newborn Genome Sequencing
A plain-English guide to newborn genome sequencing in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Clinical Trials
A plain-English guide to clinical trials in rare disease: what the platform is trying to solve, why families are watching it closely, and how it connects to interviews, explainers, trials, and care decisions.
Related interviews
Interview
Phelan-McDermidSHANK3Gene Therapy
Dr. Guoping Feng (MIT) on SHANK3 Gene Therapy, Brain Disorders, and What’s Coming Next
Dr. Guoping Feng
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. 
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Interview
Phelan-McDermidCommunityFoundation Building
How a Parent-Led Movement Grows: Sue Lomas (Phelan-McDermid Syndrome Foundation)
Sue Lomas
Sue Lomas helped build the Phelan-McDermid Syndrome community in the earliest days—when families were scattered, information was scarce, and finding “your people” took persistence. In this conversation, Sue and I talk about how rare-dise...
Watch InterviewRelated explainers
Genetic Medicine
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.
Gene TherapyAAVRare Disease
Genetic Medicine
What Is ASO Therapy?
ASO therapy uses short pieces of synthetic genetic material to change how RNA is read, processed, or reduced inside cells.
ASORNA Therapy