Genome editing in pluripotent stem cells: research and therapeutic applications
| Autor: | Michela Deleidi, Cong Yu |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2016 |
| Předmět: |
0301 basic medicine
Pluripotent Stem Cells Somatic cell Genetic enhancement Induced Pluripotent Stem Cells Biophysics Context (language use) Computational biology Biology Bioinformatics Biochemistry 03 medical and health sciences Genome editing methods [Genetic Therapy] ddc:570 Animals Humans metabolism [Cell Nucleus] Induced pluripotent stem cell Homologous Recombination Molecular Biology Gene Alleles Cell Nucleus Gene Editing Deoxyribonucleases Genome Human cytology [Pluripotent Stem Cells] Gene targeting Cell Biology Genetic Therapy cytology [Induced Pluripotent Stem Cells] metabolism [Deoxyribonucleases] 030104 developmental biology Mutation Human genome |
| Zdroj: | Biochemical and biophysical research communications 473(3), 665-674 (2016). doi:10.1016/j.bbrc.2016.02.113 |
| DOI: | 10.1016/j.bbrc.2016.02.113 |
| Popis: | Recent progress in human pluripotent stem cell (hPSC) and genome editing technologies has opened up new avenues for the investigation of human biology in health and disease as well as the development of therapeutic applications. Gene editing approaches with programmable nucleases have been successfully established in hPSCs and applied to study gene function, develop novel animal models and perform genetic and chemical screens. Several studies now show the successful editing of disease-linked alleles in somatic and patient-derived induced pluripotent stem cells (iPSCs) as well as in animal models. Importantly, initial clinical trials have shown the safety of programmable nucleases for ex vivo somatic gene therapy. In this context, the unlimited proliferation potential and the pluripotent properties of iPSCs may offer advantages for gene targeting approaches. However, many technical and safety issues still need to be addressed before genome-edited iPSCs are translated into the clinical setting. Here, we provide an overview of the available genome editing systems and discuss opportunities and perspectives for their application in basic research and clinical practice, with a particular focus on hPSC based research and gene therapy approaches. Finally, we discuss recent research on human germline genome editing and its social and ethical implications. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
|