ABCMdb

PMID: 26509335

Vidovic D, Carlon MS, F da Cunha M, Dekkers JF, Hollenhorst MI, Bijvelds MJ, Ramalho AS, Van den Haute C, Ferrante M, Baekelandt V, Janssens HM, De Boeck K, Sermet-Gaudelus I, de Jonge HR, Gijsbers R, Beekman JM, Edelman A, Debyser Z
rAAV-CFTRDeltaR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and CF Mice.
Am J Respir Crit Care Med. 2015 Oct 28., [PubMed]

Sentences

No. Mutations Sentence Comment

91 ABCC7 p.Glu60* 3 4 CFTR࢞R restores chloride and fluid secretion in intestinal organoids derived from cystic 5 fibrosis subjects 6 After showing functionality in a HeLa-based cell culture model, we set out to evaluate the 7 potential of CFTR࢞R to rescue the CF phenotype in human CFTR-deficient organoids 8 (E60X/4014delATTT; referred to as CF) and to compare activity to full length 3F-CFTR. Login to comment 108 ABCC7 p.Glu60* Next, 2 rAAV-CFTR࢞R was functionally validated in human CFTR-deficient organoids 3 (E60X/4014delATTT; referred to as CF) (Figure 3E-I). Login to comment 461 ABCC7 p.Glu60* All 10 results are representative of at least three independent experiments and are presented as mean &#b1; 11 SEM. CF organoids = E60X/4015ATTTdel. Login to comment 476 ABCC7 p.Glu60* CF organoids = E60X/4015ATTTdel. Login to comment 598 ABCC7 p.Glu60* Organoids 17 originate from a CF subject with two class I mutations (E60X/4015ATTTdel). Login to comment