Direct Reprogramming of Somatic Skin Cells from a Patient with Huntington’s Disease into Striatal Neurons to Create Models of Pathology

A new in vitro model of Huntington’s disease (HD) was developed via a direct reprogramming of dermal fibroblasts from HD patients into striatal neurons. A reprogramming into induced pluripotent stem (iPS) cells is obviated in the case of direct reprogramming, which thus yields neurons that preserve...

Full description

Saved in:
Bibliographic Details
Published in:Doklady. Biological sciences 2024-04, Vol.515 (1), p.15-19
Main Authors: Kraskovskaya, N. A., Khotin, M. G., Tomilin, A. N., Mikhailova, N. A.
Format: Article
Language:English
Subjects:
Animals
Biomedical and Life Sciences
Corpus Striatum - pathology
Disease Models, Animal
Epigenetics
Evolutionary Biology
Fibroblasts
Humans
Huntingtin
Huntington Disease - genetics
Huntington Disease - pathology
Huntington's disease
Huntingtons disease
Induced Pluripotent Stem Cells - pathology
Life Sciences
Neostriatum
Neurons
Phenotypes
Plant Sciences
Pluripotency
Citations: Items that this one cites
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary:A new in vitro model of Huntington’s disease (HD) was developed via a direct reprogramming of dermal fibroblasts from HD patients into striatal neurons. A reprogramming into induced pluripotent stem (iPS) cells is obviated in the case of direct reprogramming, which thus yields neurons that preserve the epigenetic information inherent in cells of a particular donor and, consequently, the age-associated disease phenotype. A main histopathological feature of HD was reproduced in the new model; i.e., aggregates of mutant huntingtin accumulated in striatal neurons derived from a patient’s fibroblasts. Experiments with cultured neurons obtained via direct reprogramming make it possible to individually assess the progression of neuropathology and to implement a personalized approach to choosing the treatment strategy and drugs for therapy. The in vitro model of HD can be used in preclinical drug studies.
ISSN:0012-4966
1608-3105
DOI:10.1134/S0012496623700849