Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies.

Détails

Ressource 1Télécharger: 26052330_BIB_FEDC491D3112.pdf (4024.38 [Ko])
Etat: Public
Version: Final published version
ID Serval
serval:BIB_FEDC491D3112
Type
Article: article d'un périodique ou d'un magazine.
Sous-type
Synthèse (review): revue aussi complète que possible des connaissances sur un sujet, rédigée à partir de l'analyse exhaustive des travaux publiés.
Collection
Publications
Institution
Titre
Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies.
Périodique
Frontiers In Immunology
Auteur(s)
Ott de Bruin L.M., Volpi S., Musunuru K.
ISSN
1664-3224 (Electronic)
ISSN-L
1664-3224
Statut éditorial
Publié
Date de publication
2015
Peer-reviewed
Oui
Volume
6
Pages
250
Langue
anglais
Notes
Publication types: Journal Article ; Review Publication Status: epublish Document Type: Review
Résumé
Severe combined immunodeficiency (SCID) and other severe non-SCID primary immunodeficiencies (non-SCID PID) can be treated by allogeneic hematopoietic stem cell (HSC) transplantation, but when histocompatibility leukocyte antigen-matched donors are lacking, this can be a high-risk procedure. Correcting the patient's own HSCs with gene therapy offers an attractive alternative. Gene therapies currently being used in clinical settings insert a functional copy of the entire gene by means of a viral vector. With this treatment, severe complications may result due to integration within oncogenes. A promising alternative is the use of endonucleases such as ZFNs, TALENs, and CRISPR/Cas9 to introduce a double-stranded break in the DNA and thus induce homology-directed repair. With these genome-editing tools a correct copy can be inserted in a precisely targeted "safe harbor." They can also be used to correct pathogenic mutations in situ and to develop cellular or animal models needed to study the pathogenic effects of specific genetic defects found in immunodeficient patients. This review discusses the advantages and disadvantages of these endonucleases in gene correction and modeling with an emphasis on CRISPR/Cas9, which offers the most promise due to its efficacy and versatility.
Pubmed
Web of science
Open Access
Oui
Création de la notice
20/06/2015 9:22
Dernière modification de la notice
20/08/2019 17:29
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