HML-2 env knockdown by AAV9-mediated miRNAs attenuates amyotrophic lateral sclerosis-like manifestations in mice - PubMed
7 hours ago
- #gene therapy
- #ALS
- #neurodegeneration
- Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease with no cure, and HERV-K, specifically HML-2, is implicated in its pathogenesis.
- AAV9-mediated artificial microRNAs (amiRNAs) targeting HML-2 env gene were developed and tested in an ALS mouse model.
- Three selected amiRNAs were inserted into an AAV9 vector, showing robust silencing of HML-2 env gene and protein expression.
- In vivo, a single AAV9-amiRNA injection in neonatal mice reduced HML-2 Env expression in the brain and spinal cord, improving motor neuron survival and function.
- The study supports the therapeutic potential of AAV9-delivered amiRNAs for ALS linked to HML-2.