Email this article Patient-Specific Induced Pluripotent Stem Cells for SOD1-Associated Amyotrophic Lateral Sclerosis Pathogenesis Studies
- Authors: Chestkov I.V.1, Vasilieva E.A.1, Illarioshkin S.N.2, Lagarkova M.A.1, Kiselev S.L.1
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Affiliations:
- Vavilov Institute of General Genetics RAS
- Research Center of Neurology RAMS
- Issue: Vol 6, No 1 (2014)
- Pages: 54-60
- Section: Research Articles
- Submitted: 17.01.2020
- Published: 15.03.2014
- URL: https://actanaturae.ru/2075-8251/article/view/10559
- DOI: https://doi.org/10.32607/20758251-2014-6-1-54-60
- ID: 10559
Cite item
Abstract
The genetic reprogramming technology allows one to generate pluripotent stem cells for individual patients. These cells, called induced pluripotent stem cells (iPSCs), can be an unlimited source of specialized cell types for the body. Thus, autologous somatic cell replacement therapy becomes possible, as well as the generation of in vitro cell models for studying the mechanisms of disease pathogenesis and drug discovery. Amyotrophic lateral sclerosis (ALS) is an incurable neurodegenerative disorder that leads to a loss of upper and lower motor neurons. About 10% of cases are genetically inherited, and the most common familial form of ALS is associated with mutations in the SOD1 gene. We used the reprogramming technology to generate induced pluripotent stem cells with patients with familial ALS. Patient-specific iPS cells were obtained by both integration and transgene-free delivery methods of reprogramming transcription factors. These iPS cells have the properties of pluripotent cells and are capable of direct differentiation into motor neurons.
About the authors
I. V. Chestkov
Vavilov Institute of General Genetics RAS
Author for correspondence.
Email: ichestkov@vigg.ru
Russian Federation
E. A. Vasilieva
Vavilov Institute of General Genetics RAS
Email: ichestkov@vigg.ru
Russian Federation
S. N. Illarioshkin
Research Center of Neurology RAMS
Email: ichestkov@vigg.ru
Russian Federation
M. A. Lagarkova
Vavilov Institute of General Genetics RAS
Email: ichestkov@vigg.ru
Russian Federation
S. L. Kiselev
Vavilov Institute of General Genetics RAS
Email: ichestkov@vigg.ru
Russian Federation
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