Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations
Joint Authors
Source
Issue
Vol. 2016, Issue 2016 (31 Dec. 2015), pp.1-9, 9 p.
Publisher
Hindawi Publishing Corporation
Publication Date
2016-12-15
Country of Publication
Egypt
No. of Pages
9
Abstract EN
The recent advent of induced pluripotent stem cells (iPSCs) and gene therapy tools has raised the possibility of autologous cell therapy for rare genetic diseases.
However, cellular reprogramming is inefficient in certain diseases such as ataxia telangiectasia, Fanconi anemia, LIG4 syndrome, and fibrodysplasia ossificans progressiva syndrome, owing to interference of the disease-related genes.
To overcome these therapeutic limitations, it is necessary to fundamentally correct the abnormal gene during or prior to the reprogramming process.
In addition, as genetic etiology of Parkinson’s disease, it has been well known that induced neural stem cells (iNSCs) were progressively depleted by LRRK2 gene mutation, LRRK2 (G2019S).
Thus, to maintain the induced NSCs directly derived from PD patient cells harboring LRRK2 (G2019S), it would be ideal to simultaneously treat the LRRK2 (G2019S) fibroblast during the process of TD.
Therefore, simultaneous reprogramming (or TD) and gene therapy would provide the solution for therapeutic limitation caused by vulnerability of reprogramming or TD, in addition to being suitable for general application to the generation of autologous cell-therapy products for patients with genetic defects, thereby obviating the need for the arduous processes currently required.
American Psychological Association (APA)
Lee, Seo-Young& Chung, Sun-Ku. 2016. Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations. Stem Cells International،Vol. 2016, no. 2016, pp.1-9.
https://search.emarefa.net/detail/BIM-1116297
Modern Language Association (MLA)
Lee, Seo-Young& Chung, Sun-Ku. Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations. Stem Cells International Vol. 2016, no. 2016 (2015), pp.1-9.
https://search.emarefa.net/detail/BIM-1116297
American Medical Association (AMA)
Lee, Seo-Young& Chung, Sun-Ku. Integrating Gene Correction in the Reprogramming and Transdifferentiation Processes: A One-Step Strategy to Overcome Stem Cell-Based Gene Therapy Limitations. Stem Cells International. 2016. Vol. 2016, no. 2016, pp.1-9.
https://search.emarefa.net/detail/BIM-1116297
Data Type
Journal Articles
Language
English
Notes
Includes bibliographical references
Record ID
BIM-1116297