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mHTT siRNA
- Development stage
- Preclinical
- Lead developer
- Nanjing University
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
mHTT siRNA is a small interfering RNA (siRNA) designed to specifically silence the expression of the mutant huntingtin (mHTT) gene, which is the root cause of Huntington's disease. This therapeutic approach utilizes a synthetic biology strategy where genetic circuits reprogram host liver cells to produce and self-assemble the mHTT siRNA into neuron-targeting exosomes. These exosomes then facilitate the delivery of the siRNA across the blood-brain barrier to the cortex and striatum, aiming to reduce the levels of mHTT protein and toxic aggregates, thereby ameliorating behavioral deficits and neuropathologies associated with Huntington's disease.
- Other names
- mHTT-silencing siRNA
02
Targets
HTT mRNA (HTT / Huntingtin gene)
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