AAV9-sh-HOTAIR

AAV9-sh-HOTAIR is an investigational gene therapy utilizing an adeno-associated virus serotype 9 (AAV9) vector to deliver a short hairpin RNA (shRNA) targeting the lncRNA HOTAIR (HOX transcript antisense intergenic RNA), which is implicated in cancer progression. The AAV9 capsid enables efficient gene delivery to target tissues, while the shRNA component mediates RNA interference, leading to post-transcriptional silencing of HOTAIR. Targeting HOTAIR can reduce tumorigenic properties in cancer cells, as HOTAIR regulates chromatin state and gene expression affecting metastasis and proliferation. This strategy exemplifies a new generation of gene therapies for oncology, combining targeted RNA interference against oncogenic lncRNAs with AAV-mediated delivery. Although similar approaches have been tested in preclinical cancer models (e.g., using AAV-shRNA against oncogenic pathways), there is no public record of an approved or late-stage clinical program specifically under the name "AAV9-sh-HOTAIR"[2][4].