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.
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- Gene therapy generates new neurons to treat Huntington’s disease
- New gene therapy for Huntington’s demonstrates success in mice
- Gene therapy conversion of striatal astrocytes into GABAergic neurons in mouse models of Huntington’s disease.
- Innate Immunity: A Common Denominator between Neurodegenerative and Neuropsychiatric Diseases.