Drug intelligence / Profile preview

rAAV-sh-NeuroD1

Development stage
Unknown
Lead developer
The Pennsylvania State University
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Intrathecal, Intralesional
01

Overview

rAAV-sh-NeuroD1 is a recombinant adeno-associated virus (rAAV) vector designed to deliver a short hairpin RNA (shRNA) targeting the Neurogenic differentiation 1 (NeuroD1) gene. It is primarily utilized as a research tool in neuroscience to investigate the functional role of NeuroD1 in processes such as adult neurogenesis, neuronal differentiation, and survival. By leveraging the RNA interference (RNAi) pathway, the vector mediates the knockdown of NeuroD1 expression in specific brain regions, such as the dentate gyrus of the hippocampus. While NeuroD1 overexpression is being explored therapeutically for in vivo cell reprogramming (e.g., converting astrocytes into neurons), the shRNA-mediated knockdown version is typically employed in loss-of-function studies to elucidate the transcription factor's necessity in neural development and maintenance.

Other names
rAAV-shRNA-NeuroD1rAAV-shRNA-NeuroD-1rAAV-shRNA-NeuroD 1AAV-shRNA-NeuroD1AAV-shRNA-NeuroD-1AAV-shRNA-NeuroD 1
02

Targets

ADRB2 (β2)

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