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PMP22-lowering approaches for CMT1A
Because CMT1A is caused by an extra copy of PMP22, several programmes aim to reduce PMP22 expression using gene therapy, RNA-targeted approaches or small molecules. Progress and results vary by programme.
Because CMT1A is caused by an extra copy of PMP22, several programmes aim to reduce PMP22 expression using gene therapy, RNA-targeted approaches or small molecules. Progress and results vary by programme.
Focus: CMT1A (PMP22 duplication).
Phase or type: Phase 1–3 across programmes.
Source
- Source
- Multiple academic and industry groups
- Organisation
- Multiple academic and industry groups
Curated by the Charcot-Marie-Tooth Foundation of India.
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