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MTMR5 shRNA
- Development stage
- Preclinical
- Lead developer
- Johns Hopkins University
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Parenteral
01
Overview
MTMR5 shRNA is an experimental RNA interference-based therapeutic candidate designed to enhance neuroprotective proteostasis by targeting the autophagy suppressor MTMR5 (Myotubularin-related protein 5). In neurons, MTMR5 acts as a negative regulator of the autophagy-lysosome network. By utilizing lentiviral delivery of short hairpin RNA (shRNA) to knock down MTMR5 expression, the treatment aims to promote the degradation of toxic alpha-synuclein (asyn) aggregates, which are central to the pathophysiology of Parkinson's disease (PD). Research conducted at Johns Hopkins University has demonstrated that reversing this negative regulatory mechanism can rescue neurodegeneration in various genetic contexts of PD, including those with SNCA or GBA1 mutations.
- Other names
- shRNA targeting MTMR5MTMR5 knockdownMTMR-5 knockdownMTMR 5 knockdown
02
Targets
Lipid phosphatase (Lipid phosphatase family)
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