iNeu™ Motor Neurons ALS TDP43 Q331K, Disease Model

Description

Tar DNA binding protein (TDP-43) is a highly conserved ribonucleoprotein that accumulates as ubiquitinated inclusions in patients with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP-43 usually shuttles between the nucleus and cytoplasm, and plays a role in regulating RNA splicing, stability and transport. TDP-43 neuropathology is present in most familial and sporadic ALS; therefore, understanding TDP-43 dysfunction may provide a way for therapeutic intervention. Further TDP-43 mutations, including M337V and Q331K variants, account for nearly 5% of familial ALS.

In order to promote ALS/FTD research work, an isogenic model of ALS motor neuron was generated. Using CRISPR gene editing to introduce M337V or Q331K mutations into our iNeu motor neuron parental induced pluripotent stem (iPS) cell line. These gene-edited iPS cells are fully differentiated into high-purity Human cells, showing similar indicators to our apparently healthy iNeu motor neurons.

The isogenic disease model iNeu motor neuron ALS TDP43 M337V and TDP43 Q331K combined with iNeu motor neuron provides a precision medicine-based method that can promote functional genomics research, identification of drug pathways, and the development of co-culture models. They can be used to identify the functional changes and disease mechanisms associated with TDP-43, for drug discovery and the identification of ALS/FTD therapeutic targets, and the development of neuromuscular junction disease models.

Features

Highly pure
iNeu Motor Neurons ALS TDP43 are highly pure cholinergic Human motor neurons that are ≥90% β-III tubulin+/Nestin- and ≥70% Isl1/2 at 14 days in vitro. These proteins also express characteristic markers of motor neurons including Isl1, AchR, C9or72, VAChT, and FoxP1.

Homogenous and reproducible
iNeu Motor Neurons ALS TDP43 undergo rigorous quality control testing ensure the same performance with every batch of cells.

Rapid results
iNeu Motor Neurons ALS TDP43 are shipped cryopreserved with optimized media. Simply thaw and use.

Neurological Disease Models

ALS-related Cells

Cell Types

Motor Neurons

Relevant Diseases

Amyotrophic Lateral Sclerosis

Species

Human

Size

1 Vial: ≥ 3 x 10^6 cells/vial

Tissue Source

Blood

Age

50-59

Mycoplasma Testing

The cell line has been screened using the Mycoplasma Detection Kit to confirm the absence of Mycoplasma contamination.

Shipping

Dry ice.

Storage

Liquid Nitrogen

Research Use Only

For research use only

Quality Control

All cells test negative for mycoplasma, bacteria, yeast, and fungi.

Target

TDP43

Official Name

HTR1A

Alternative Names

5-HT1A receptor; ADRB2RL1; ADRBRL1; 5-HT1A; 5-hydroxytryptamine (serotonin) receptor 1A; 5HT1A; RAT5HT1A; 5-HT-1A; 5-hydroxytryptamine receptor 1A; serotonin receptor 1A; 5-HT1A receptor; Gpcr18

Gender

Male

Donor Health

Disease

Ethnicity

Caucasian