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Please use this identifier to cite or link to this item: http://hdl.handle.net/10481/49163

Title: Gene Editing as an Alternative to Retroviral Vectors for Wiskott-Aldrich syndrome Gene therapy
Other Titles: Edición génica como alternativa a los vectores retrovirales para terapia génica del síndrome de Wiskott-Aldrich
Authors: Gutiérrez-Guerrero, Alejandra
Direction: Martín Molina, Francisco
Benabdellah, Karim
Collaborator: Universidad de Granada. Programa Oficial de Doctorado en: Biomedicina
Issue Date: 2018
Submitted Date: 9-Oct-2017
Abstract: One of the safest and most efficient treatments for monogenic diseases affecting the hematopoietic system is gene therapy using lentiviral vectors (LVs) and gammaretroviral vectors (GVs). However, although latest generation LVs and GVs are highly efficient and safe, both are retroviral vectors which integrate randomly in transcriptional active sites. This uncontrollable integration generates risk of insertional mutagenesis, which can produce undesirable effects on the modified cells. Gene editing has been proposed as a safer alternative, whose objective is the restoration of the endogenous levels of the wild type gene or the ectopic insertion of a therapeutic gene in the mutated locus or in a safe locus (safe harbor). The most potent gene editing strategies are based on the use of specific nucleases that produce double strand brakes (DSBs) in a specific site in the genome. Non homologous end joining (NHEJ) or homologous recombination (HR) repair of these DSBs allow editing the genome in different ways with a final objective: restore de normal function of the mutated gene. Our final aim was to develop a gene edition tool for efficient genetic rescue of human hematopoietic stem cells (hHSCs) from Wiskott-Aldrich syndrome (WAS) patients.
Sponsorship: Tesis Univ. Granada. Programa Oficial de Doctorado en: Biomedicina
Publisher: Universidad de Granada
Keywords: Síndrome de Wiskott Aldrich
Terapia génica
Vectores virales
Células madre hematopoyéticas
Sistema hematopoyético
Plásmidos
UDC: 61
575
616-085
310902
URI: http://hdl.handle.net/10481/49163
ISBN: 9788491637080
Rights : Creative Commons Attribution-NonCommercial-NoDerivs 3.0 License
Citation: Gutiérrez-Guerrero, A. Gene Editing as an Alternative to Retroviral Vectors for Wiskott-Aldrich syndrome Gene therapy. Granada: Universidad de Granada, 2018. [http://hdl.handle.net/10481/49163]
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