Article
rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice.
American journal of respiratory and critical care medicine - 1 Feb 2016
Vidović Dragana, Carlon Marianne S, da Cunha Mélanie F, Dekkers Johanna F, Hollenhorst Monika I, Bijvelds Marcel J C, Ramalho Anabela S, Van den Haute Chris, Ferrante Marc, Baekelandt Veerle, Janssens Hettie M, De Boeck Kris, Sermet-Gaudelus Isabelle, de Jonge Hugo R, Gijsbers Rik, Beekman Jeffrey M, Edelman Aleksander, Debyser Zeger
Abstract excerpt
RATIONALE: Gene therapy holds promise for a curative mutation-independent treatment applicable to all patients with cystic fibrosis (CF). The various viral vector-based clinical trials conducted in the past have demonstrated safety and tolerance of different vectors, but none have led to a clear and persistent clinical benefit. Recent clinical breakthroughs in recombinant adeno-associated viral vector...
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