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adeno-associated virus expressing short hairpin RNA targeting SHMT2
- Development stage
- Preclinical
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intracerebral, Intravenous
01
Overview
Adeno-associated virus expressing short hairpin RNA targeting SHMT2 (AAV-shSHMT2) is an experimental gene therapy construct designed to silence the expression of the **Serine Hydroxymethyltransferase 2 (SHMT2)** gene. SHMT2 is a key mitochondrial enzyme in the one-carbon (1C) metabolism pathway. This construct utilizes an adeno-associated virus (AAV) vector to deliver a short hairpin RNA (shRNA) sequence that triggers the RNA interference (RNAi) pathway, leading to the degradation of SHMT2 mRNA and a subsequent reduction in SHMT2 protein levels. In the context of neurodegenerative research, specifically **Huntington's disease (HD)**, this agent is used as a tool to model SHMT2 deficiency. Research indicates that knocking down SHMT2 leads to the accumulation of homocysteine (HCY), which suppresses histone lactylation and disrupts transcriptional regulation, thereby exacerbating mutant huntingtin (mHTT) aggregation and medium spiny neuron (MSN) degeneration.
- Other names
- AAV-shRNA-SHMT2AAV-shRNA-SHMT-2AAV-shRNA-SHMT 2AAV-shRNA-targeting-SHMT2AAV-shRNA-targeting-SHMT-2AAV-shRNA-targeting-SHMT 2
02
Targets
SHMT2 (Serine hydroxymethyltransferase 2)
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