(Q38949076)

7 September 2017

title

rAAV-CFTRΔR Rescues the Cystic Fibrosis Phenotype in Human Intestinal Organoids and Cystic Fibrosis Mice (English)

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7 September 2017

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18

Zeger Debyser

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Veerle Baekelandt

10

object named as

Veerle Baekelandt

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7 September 2017

17

Aleksander Edelman

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7 September 2017

Isabelle Sermet-Gaudelus

13

object named as

Isabelle Sermet-Gaudelus

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7 September 2017

Dragana Vidović

1

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7 September 2017

Marianne S Carlon

2

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7 September 2017

Mélanie F da Cunha

3

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7 September 2017

Johanna F Dekkers

4

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7 September 2017

Monika I Hollenhorst

5

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7 September 2017

Marcel J C Bijvelds

6

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7 September 2017

Anabela S Ramalho

7

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7 September 2017

Chris Van den Haute

8

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7 September 2017

Marc Ferrante

9

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7 September 2017

Hettie M Janssens

11

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7 September 2017

Kris De Boeck

12

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7 September 2017

Hugo R de Jonge

14

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7 September 2017

Rik Gijsbers

15

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7 September 2017

Jeffrey M Beekman

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7 September 2017

language of work or name

English

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publication date

28 October 2015

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American Journal of Respiratory and Critical Care Medicine

7 September 2017

published in

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7 September 2017

volume

193

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7 September 2017

issue

3

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7 September 2017

page(s)

288-298

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Lumacaftor-Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR.

7 September 2017

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CFTR with a partially deleted R domain corrects the cystic fibrosis chloride transport defect in human airway epithelia in vitro and in mouse nasal mucosa in vivo

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Association between Hsp90 and the ClC-2 chloride channel upregulates channel function

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Characterization of nasal potential difference in cftr knockout and F508del-CFTR mice

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Transcomplementation by a truncation mutant of cystic fibrosis transmembrane conductance regulator (CFTR) enhances ΔF508 processing through a biomolecular interaction

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Cystic fibrosis transmembrane regulator inhibitors CFTR(inh)-172 and GlyH-101 target mitochondrial functions, independently of chloride channel inhibition

21 January 2018

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Mouse models of cystic fibrosis: phenotypic analysis and research applications

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New clinical diagnostic procedures for cystic fibrosis in Europe

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Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA

21 January 2018

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Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial.

21 January 2018

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Toward gene therapy for cystic fibrosis using a lentivirus pseudotyped with Sendai virus envelopes.

21 January 2018

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Long-term persistence of gene expression from adeno-associated virus serotype 5 in the mouse airways

21 January 2018

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Polarity influences the efficiency of recombinant adenoassociated virus infection in differentiated airway epithelia.

21 January 2018

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Dystrophin immunity in Duchenne's muscular dystrophy.

21 January 2018

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Cystic fibrosis transmembrane regulator missing the first four transmembrane segments increases wild type and DeltaF508 processing

21 January 2018

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Gene transfer of CFTR to airway epithelia: low levels of expression are sufficient to correct Cl- transport and overexpression can generate basolateral CFTR.

21 January 2018

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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21 January 2018

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Characterization of defects in ion transport and tissue development in cystic fibrosis transmembrane conductance regulator (CFTR)-knockout rats

21 January 2018

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Sequential targeting of CFTR by BAC vectors generates a novel pig model of cystic fibrosis

21 January 2018

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Repeated Aerosolized AAV-CFTR for Treatment of Cystic Fibrosis: A Randomized Placebo-Controlled Phase 2B Trial

21 January 2018

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10.1164/RCCM.201505-0914OC

21 January 2018

Identifiers

DOI

1 reference

7 September 2017

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