SMN1 shRNA Plasmid Expression Vector Set

Name: SMN1 shRNA Plasmid Expression Vector Set
SKU: NRYF-0925-XY346
Rating: 5 (1 reviews)

[CAT#: NRYF-0925-XY346]

Target:: SMN1

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Product Overview

Description

A targeted research kit for the specific knockdown of the Survival Motor Neuron 1 (SMN1) gene, whose loss causes SMA. The set includes three sequence-verified shRNA plasmid vectors targeting different regions of the SMN1 mRNA, plus a non-targeting scrambled shRNA plasmid as a negative control. It is a valuable tool for comparative studies and modeling the molecular consequences of SMN1 absence.

Properties

Size

5-50 μg

Shipping

Ambient

Storage

4°C

Research Use Only

Target Details

Target

SMN1

Full Name

Protein C9orf72

Gene ID

203228

Uniprot ID

Q96LT7

Target Overview

The C9orf72 gene is most notable for a specific pathogenic mutation: a GGGGCC hexanucleotide repeat expansion located in a non-coding region of the gene. This expansion is the most common genetic cause of both Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD). The mutation is believed to cause disease through multiple mechanisms, including a partial loss of the normal C9orf72 protein's function, the formation of toxic RNA aggregates (foci) that sequester other proteins, and the production of toxic dipeptide repeat proteins. The normal function of the C9orf72 protein is thought to be involved in regulating cellular trafficking and autophagy.

Publications