Drug intelligence / Profile preview

rAAV-sh-Ngn3

Development stage
Unknown
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Parenteral
01

Overview

rAAV-sh-Ngn3 is a recombinant adeno-associated virus (rAAV) vector designed to deliver short hairpin RNA (shRNA) for the targeted knockdown of Neurogenin-3 (Ngn3) expression. Ngn3 is a basic helix-loop-helix (bHLH) transcription factor that serves as a master regulator for the differentiation of endocrine progenitor cells in the pancreas and the intestine. By leveraging the RNA interference (RNAi) pathway, rAAV-sh-Ngn3 facilitates the sequence-specific degradation of Ngn3 mRNA, thereby inhibiting the production of the Ngn3 protein. This construct is extensively used as a research tool in preclinical models to investigate the developmental requirements of the endocrine pancreas, study the plasticity of pancreatic cell types, and model conditions of Ngn3 deficiency. While Ngn3 overexpression is a common strategy in regenerative medicine for diabetes, the shRNA-mediated knockdown approach is primarily utilized to elucidate gene function and regulatory networks in developmental biology.

Other names
rAAV-shRNA-Ngn3rAAV-shRNA-Ngn-3rAAV-shRNA-Ngn 3AAV-shRNA-Ngn3AAV-shRNA-Ngn-3AAV-shRNA-Ngn 3
02

Targets

NEUROG3 (Neurogenin-3)

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