Novel Gene Therapy Generated New Functional Neurons in Mouse Models of Huntington’s Disease (HD)

Novel Gene Therapy Generated New Functional Neurons in Mouse Models of Huntington’s Disease (HD)

Publication date: Mar 13, 2020

STATE COLLEGE, Penn. –(BUSINESS WIRE)–NeuExcell Therapeutics, Inc. , an early-stage gene therapy company focusing on neurodegenerative diseases, today announced the publication of research demonstrating proof of principle for the company’s investigational gene therapy, which generated functional new neurons in animal models of Huntington’s disease (HD).

The results, published in the current issue of Nature Communications1, show that the new gene therapy led to improvement in HD symptoms and extension of lifespan in validated mouse models, indicating a promising approach to treating this disease and other serious central nervous system (CNS) disorders.

The Nature Communications paper describes how a research team led by Gong Chen, PhD, NeuExcell’s founder and chief scientific officer, developed an adeno-associated virus-based (AAV-based) gene therapy to regenerate functional new neurons in the brains of mice with HD-like symptoms.

Dr. Chen, a former professor at Penn State University and now leading a brain repair center at Jinan University in China, pioneered this gene therapy approach to neuroregeneration, which delivers specific neural transcription factors to a targeted site in the brain or spinal cord.

Dr. Chen and colleagues reported that the gene therapy converted 80% of viral infected astrocytes in the striatum into functional GABAergic neurons, and 60% of the newly generated neurons were the right type of MSNs.

-Such high neuroregeneration efficiency explains the significant motor functional recovery after our gene therapy treatment and even more strikingly, a significant extension of life span among the treated HD mice,” Dr. Chen noted.

In addition to the successful conversion of astrocytes to neurons and the resulting improvement in HD symptoms, Dr. Chen sees this type of neuroregenerative gene therapy as potentially beneficial in other neurodegenerative conditions such as Alzheimer’s disease, Parkinson’s disease, and ALS (amyotrophic lateral sclerosis), as well as in cases of ischemic cortical stroke, traumatic brain or spinal injury.

Concepts Keywords
ALS Neurodegenerative diseases NeuExcell
Alzheimer Striatum
Amyotrophic Lateral Sclerosis Human brain
Astrocyte Astrocyte
Astrocytes Neuron
Brain Neuroscience
BUSINESS WIRE Stem cells
Central Nervous System Basal ganglia
China Branches of biology
CNS Disorders Glial cells
Cortical Addiction
GABAergic Ischemic cortical stroke
Genetic Disease Transplantation
Glia Innovative therapy
Glial Investigational therapy
Glial Cells
Huntingtin
Huntington
Immune System
Ischemic
Jinan
Medium Spiny Neurons
Memory
Mice
Motor Skills
Mutant
Mutation
Nature
Nature Communications
Nerve
Neural Tissue
Neurodegenerative
Neurodegenerative Diseases
Neurological
Neurology
Neuron
Neurons
Neuroregeneration
Parkinson
Pedro Gonzalez
Pennsylvania
PhD
Primate
Progressive
Spinal Cord
Spinal Cord Injury
Spinal Injury
Striatal
Striatum
Stroke
Transcription Factors
Traumatic Brain Injury
Viral
Virus
Vivo

Semantics

Type Source Name
disease MESH genetic disease
disease MESH neurodegenerative diseases
drug DRUGBANK Nonoxynol-9
disease MESH suffering
disease MESH stroke
disease MESH amyotrophic lateral sclerosis
drug DRUGBANK Tropicamide
pathway KEGG Huntington disease
disease MESH community
disease MESH Huntington Disease

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