Retraction Note: Robust method for TALEN-edited correction of pF508del in patient-specific induced pluripotent stem cells
| Autor: | Maria V. Camarasa, Víctor Miguel Gálvez |
|---|---|
| Jazyk: | angličtina |
| Rok vydání: | 2017 |
| Předmět: |
0301 basic medicine
Genetics Induced stem cells Transcription activator-like effector nuclease lcsh:R5-920 Tetraploid complementation assay Medicine (miscellaneous) Nucleofection Cell Biology Biology Biochemistry Genetics and Molecular Biology (miscellaneous) Cell biology lcsh:Biochemistry 03 medical and health sciences 030104 developmental biology Cancer stem cell Molecular Medicine lcsh:QD415-436 Stem cell Induced pluripotent stem cell lcsh:Medicine (General) Selectable marker |
| Zdroj: | Stem Cell Research & Therapy, Vol 8, Iss 1, Pp 1-1 (2017) |
| ISSN: | 1757-6512 |
| DOI: | 10.1186/s13287-017-0591-5 |
| Popis: | Cystic fibrosis is one of the most frequent inherited rare diseases, caused by mutations in the cystic fibrosis transmembrane conductance regulator gene. Apart from symptomatic treatments, therapeutic protocols for curing the disease have not yet been established. The regeneration of genetically corrected, disease-free epithelia in cystic fibrosis patients is envisioned by designing a stem cell/genetic therapy in which patient-derived pluripotent stem cells are genetically corrected, from which target tissues are derived. In this framework, we present an efficient method for seamless correction of pF508del mutation in patient-specific induced pluripotent stem cells by gene edited homologous recombination. Gene edition has been performed by transcription activator-like effector nucleases and a homologous recombination donor vector which contains a PiggyBac transposon-based double selectable marker cassette. This new method has been designed to partially avoid xenobiotics from the culture system, improve cell culture efficiency and genome stability by using a robust culture system method, and optimize timings. Overall, once the pluripotent cells have been amplified for the first nucleofection, the procedure can be completed in 69 days, and can be easily adapted to edit and change any gene of interest. |
| Databáze: | OpenAIRE |
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