Fiche publication
Date publication
mars 2020
Journal
Molecular therapy : the journal of the American Society of Gene Therapy
Auteurs
Membres identifiés du Cancéropôle Est :
Dr HERAULT Yann
Tous les auteurs :
Teboul L, Herault Y, Wells S, Qasim W, Pavlovic G
Lien Pubmed
Résumé
Genome editing tools have already revolutionized biomedical research and are also expected to have an important impact in the clinic. However, their extensive use in research has revealed much unpredictability, both off and on target, in the outcome of their application. We discuss the challenges associated with this unpredictability, both for research and in the clinic. For the former, an extensive validation of the model is essential. For the latter, potential unpredicted activity does not preclude the use of these tools but requires that molecular evidence to underpin the relevant risk:benefit evaluation is available. Safe and successful clinical application will also depend on the mode of delivery and the cellular context.
Mots clés
CRISPR/Cas9, clinical safety, functional genomics, gene delivery, gene therapy, medical research, research reproducibility
Référence
Mol. Ther.. 2020 Mar 20;:
