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Chroniques génomiques - Thérapie génique germinale, le retour ?
(Éditions EDK, Groupe EDP Sciences, 2015)
The recent development of a powerful and flexible genome editing technique (the CRISP-cas9 method) accelerates tremendously the production of animal models and will significantly enhance the perspectives of (somatic) gene ...
Chroniques génomiques - CRISPR-Cas9, une nouvelle donne pour la thérapie génique
(Éditions EDK, Groupe EDP Sciences, 2015)
Targeted modification of genes (“gene editing”) is made much easier by the recently developed CRISPR-Cas9 system. This has raised alarm about possible uses of this technology for germline modification of the human genome; ...
Chroniques génomiques - Modification ciblée du génome : une nouvelle approche prometteuse
(Éditions EDK, Groupe EDP Sciences, 2017)
A new approach, based on the use of peptide nucleic acid molecules to form triple helices and promote locus-specific recombination, demonstrates potential for in vivo gene correction at clinically significant levels and ...
Hémophilie : la thérapie génique, enfin… : Chroniques Génomiques
(EDP Sciences, 2018)
Recent efforts at gene therapy for haemophilia A and B using AAV-derived vectors show durable expression of coagulation factors at significant levels, resulting in almost complete correction of the phenotype. This is the ...