Gene therapy generates new neurons to treat Huntington’s disease

Gene therapy generates new neurons to treat Huntington’s disease

Publication date: Feb 29, 2020

A research group led by Dr. Gong Chen, a former professor at Penn State University and now leading a brain repair center at Jinan University in China, has developed a novel gene therapy to regenerate functional new neurons in mouse models of HD.

We are developing a series of NeuroD1-based gene therapies to reprogram brain internal glial cells directly into functional new neurons to treat a variety of brain disorders, including Huntington’s disease, Alzheimer’s disease, stroke, ALS, and many more,” said Dr. Chen.

“Because every single neuron in our brain is surrounded by supporting glial cells, such direct glia-to-neuron conversion technology offers great advantages over stem cell transplantation therapy in terms of high efficiency of neuroregeneration and no worries about immunorejection,” Dr. Chen added.

Dr. Chen is one of the early pioneers making use of internal brain glial cells to regenerate functional new neurons by overexpressing neural transcription factors in the mouse brain.

“In order to generate GABAergic neurons, we combined NeuroD1 together with another transcription factor Dlx2, which is known to generate GABAergic neurons during early brain development, and successfully converted striatal astrocytes into GABAergic neurons in HD mice,” said the first author of this article Dr. Zheng Wu.

In this HD mouse study, Dr. Chen and colleagues reported that 80% of the AAV-infected striatal astrocytes were directly converted into GABAergic neurons and the remaining astrocytes can proliferate to replenish themselves.

Concepts Keywords
ALS Molecular Therapy
Alzheimer Brain disorders
Astrocyte Abnormal chorea
Astrocytes Striatum
Axons Mutant product
Brain Gamma-Aminobutyric acid
China Transplantation
Chorea NEUROD1
Clinical Trials Neurodegeneration
Cortical Astrocyte
Fair Dealing Neuron
FDA Addiction
Force Medical terminology
GABAergic Basal ganglia
Glia Glial cells
Glial Brain
Glial Cells Branches of biology
Global Brain
Glutamatergic
Guangdong
Huntingtin
Huntington
Jinan
Macau
Mice
Mutant
Mutation
Nature Communications
Neurodegeneration
Neurogenic
Neuron
Neurons
Neuroregeneration
Primate
Striatal
Striatum
Stroke
Synaptic Connections
Transcription Factor
Transcription Factors
Vector
Vectors
Virus

Semantics

Type Source Name
disease MESH stroke
disease MESH brain disorders
disease MESH chorea
disease MESH rare disease
disease MESH disease progression
drug DRUGBANK Guanosine

Similar

Leave a Comment

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