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AAV9-KDAR
- Development stage
- Preclinical
- Lead developer
- Nationwide Children's Hospital
- Modality
- Gene Therapies
- Administration
- Intrathecal
01
Overview
AAV9-KDAR is an investigational adeno-associated virus serotype 9 (AAV9)-mediated knockdown-and-replace (KDAR) gene therapy designed for the treatment of Charcot-Marie-Tooth disease type 1B (CMT1B). Developed by researchers at Nationwide Children's Hospital and Ohio State University, the therapy utilizes engineered microRNAs (miMPZ) to suppress the expression of endogenous mutant Myelin Protein Zero (MPZ) while simultaneously delivering an RNAi-resistant healthy copy of MPZ (rMPZ) to Schwann cells. This dual mechanism aims to alleviate endoplasmic reticulum (ER) stress and restore normal myelination and peripheral nerve function.
- Other names
- AAV9-miMPZ-rMPZAAV-9-miMPZ-rMPZAAV 9-miMPZ-rMPZ
02
Targets
AGO2 (Argonaute RISC catalytic component 2)MPZ (Myelin protein zero)
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