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.
|disease||MESH||amyotrophic lateral sclerosis|
- Gene therapy generates new neurons to treat Huntington’s disease
- 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.