New gene therapy for Huntington’s demonstrates success in mice

New gene therapy for Huntington’s demonstrates success in mice

Publication date: Mar 03, 2020

A new gene therapy to potentially treat Huntington’s disease has regenerated the functional neurons in mouse models of the condition.

-We are developing a series of neurogenic differentiation 1 (NeuroD1) based gene therapies to reprogramme brain internal glial cells directly into functional new neurons to treat a variety of brain disorders including Huntington’s Disease, Alzheimer’s disease, stroke, amyotrophic lateral sclerosis (ALS) and many more,” said lead researcher Dr Gong Chen.

Chen’s team has previously demonstrated that expression of NeuroD1 in the mouse brain can directly convert cortical astrocytes (one subtype of glial cells) into functional neurons.

In order to generate GABAergic neurons, the researchers combined NeuroD1 together with another transcription factor Dlx2, known to generate GABAergic neurons during early brain development and successfully converted striatal astrocytes into GABAergic neurons in mice with Huntington’s.

Concepts Keywords
ALS Mutant product
Amyotrophic Lateral Sclerosis Brain disorders
Astrocytes Branches of biology
Axons Glial cells
Brain Basal ganglia
China NEUROD1
Cortical Neuron
Force Astrocyte
GABAergic Nervous system
Glia Gamma-Aminobutyric acid
Glial Brain
Glial Cells Striatum
Global Brain Neuronal lineage marker
Glutamatergic Transplantation
Huntington
Jinan University
Mutant
Nature Communications
Neurogenic
Neuron
Neurons
Neuroregeneration
Striatal
Striatum
Stroke
Synaptic Connections
Transcription Factor
Vectors
Virus

Semantics

Type Source Name
drug DRUGBANK Spinosad
disease MESH brain disorders
disease MESH stroke
disease MESH amyotrophic lateral sclerosis
disease MESH disease progression

Similar

Original Article

Leave a Comment

Your email address will not be published. Required fields are marked *