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dc.contributor.advisorMartín Molina, Franciscoes_ES
dc.contributor.advisorBenabdellah, Karimes_ES
dc.contributor.authorGutiérrez-Guerrero, Alejandraes_ES
dc.contributor.otherUniversidad de Granada. Programa Oficial de Doctorado en: Biomedicinaes_ES
dc.identifier.citationGutiérrez-Guerrero, A. Gene Editing as an Alternative to Retroviral Vectors for Wiskott-Aldrich syndrome Gene therapy. Granada: Universidad de Granada, 2018. []es_ES
dc.description.abstractOne 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.en_EN
dc.description.sponsorshipTesis Univ. Granada. Programa Oficial de Doctorado en: Biomedicinaes_ES
dc.publisherUniversidad de Granadaes_ES
dc.rightsCreative Commons Attribution-NonCommercial-NoDerivs 3.0 Licenseen_US
dc.subjectSíndrome de Wiskott Aldriches_ES
dc.subjectTerapia génicaes_ES
dc.subjectVectores viraleses_ES
dc.subjectCélulas madre hematopoyéticases_ES
dc.subjectSistema hematopoyéticoes_ES
dc.titleGene Editing as an Alternative to Retroviral Vectors for Wiskott-Aldrich syndrome Gene therapyen_EN
dc.title.alternativeEdición génica como alternativa a los vectores retrovirales para terapia génica del síndrome de Wiskott-Aldriches_ES
europeana.dataProviderUniversidad de Granada. España.es_ES

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Creative Commons Attribution-NonCommercial-NoDerivs 3.0 License
Except where otherwise noted, this item's license is described as Creative Commons Attribution-NonCommercial-NoDerivs 3.0 License