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...

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Veröffentlicht in:Doklady. Biological sciences 2024-04, Vol.515 (1), p.15-19
Hauptverfasser: Kraskovskaya, N. A., Khotin, M. G., Tomilin, A. N., Mikhailova, N. A.
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container_title Doklady. Biological sciences
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creator Kraskovskaya, N. A.
Khotin, M. G.
Tomilin, A. N.
Mikhailova, N. A.
description 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.
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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. 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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
title Direct Reprogramming of Somatic Skin Cells from a Patient with Huntington’s Disease into Striatal Neurons to Create Models of Pathology
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