RNA-Generated and Gene-Edited Induced Pluripotent Stem Cells for Disease Modeling and Therapy.
J Cell Physiol
; 232(6): 1262-1269, 2017 Jun.
Article
en En
| MEDLINE
| ID: mdl-27631155
ABSTRACT
Cellular reprogramming by epigenomic remodeling of chromatin holds great promise in the field of human regenerative medicine. As an example, human-induced Pluripotent Stem Cells (iPSCs) obtained by reprograming of patient somatic cells are sufficiently similar to embryonic stem cells (ESCs) and can generate all cell types of the human body. Clinical use of iPSCs is dependent on methods that do not utilize genome altering transgenic technologies that are potentially unsafe and ethically unacceptable. Transient delivery of exogenous RNA into cells provides a safer reprogramming system to transgenic approaches that rely on exogenous DNA or viral vectors. RNA reprogramming may prove to be more suitable for clinical applications and provide stable starting cell lines for gene-editing, isolation, and characterization of patient iPSC lines. The introduction and rapid evolution of CRISPR/Cas9 gene-editing systems has provided a readily accessible research tool to perform functional human genetic experiments. Similar to RNA reprogramming, transient delivery of mRNA encoding Cas9 in combination with guide RNA sequences to target specific points in the genome eliminates the risk of potential integration of Cas9 plasmid constructs. We present optimized RNA-based laboratory procedure for making and editing iPSCs. In the near-term these two powerful technologies are being harnessed to dissect mechanisms of human development and disease in vitro, supporting both basic, and translational research. J. Cell. Physiol. 232 1262-1269, 2017. © 2016 Wiley Periodicals, Inc.
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
ARN
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Enfermedad
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Células Madre Pluripotentes Inducidas
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Edición Génica
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Modelos Biológicos
Tipo de estudio:
Prognostic_studies
Aspecto:
Ethics
Límite:
Humans
Idioma:
En
Revista:
J Cell Physiol
Año:
2017
Tipo del documento:
Article
País de afiliación:
Italia