Drug intelligence / Profile preview

AAV-shGrin2a

Development stage
Preclinical
Modality
Viral Vectors → Gene Addition/Replacement → Gene Therapies, Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Intracerebral (experimental; Direct Injection To Hippocampus Or Brain Region)
01

Overview

AAV-shGrin2a is an **experimental gene therapy vector** that uses an **adeno-associated virus (AAV)** to deliver a **short hairpin RNA (shRNA)** targeting the Grin2a gene, which encodes the GluN2A subunit of the N-methyl-D-aspartate receptor (NMDAR), specifically within astrocytes[1]. Grin2a knockdown by AAV-shGrin2a results in reduced expression of astrocytic GluN2A. Preclinical studies have utilized this vector for **astrocyte-specific Grin2a knockdown in the rat hippocampus**, exploring its role in cognitive and synaptic impairments in models of Alzheimer's disease[1]. Mechanistically, AAV-shGrin2a impairs synaptic function and spatial memory, interacting with amyloid-β (Aβ) pathology, and modulating neurotrophin levels (notably nerve growth factor, NGF) and associated pathways (NF-κB, Furin, VAMP3)[1].

Other names
adeno-associated virus–short hairpin RNA Grin2aAAV-Grin2a-shRNAAAV-Grin-2a-shRNAAAV-Grin 2a-shRNA
02

Targets

GRIN2A (NMDA receptor subunit 2A)

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