O rkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue-Reference-Cited by-同舟云学术

O rkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue

Published:2017-06-30 Issue:9 Volume:9 Page:1224-1243
ISSN:1757-4676
Container-title:EMBO Molecular Medicine
language:en
Short-container-title:EMBO Mol Med

Author:

Molinski Steven V¹²^ORCID,Ahmadi Saumel²³^ORCID,Ip Wan⁴,Ouyang Hong⁴,Villella Adriana⁵,Miller John P⁵^ORCID,Lee Po‐Shun⁵,Kulleperuma Kethika¹²,Du Kai²,Di Paola Michelle¹²,Eckford Paul DW²,Laselva Onofrio²^ORCID,Huan Ling Jun²,Wellhauser Leigh²^ORCID,Li Ellen²,Ray Peter N⁶,Pomès Régis¹²,Moraes Theo J⁴⁷^ORCID,Gonska Tanja⁴⁷^ORCID,Ratjen Felix⁸^ORCID,Bear Christine E¹²³^ORCID

Affiliation:

1. Department of Biochemistry University of Toronto Toronto ON Canada

2. Programme in Molecular Medicine Research Institute Hospital for Sick Children Toronto ON Canada

3. Department of Physiology University of Toronto Toronto ON Canada

4. Programme in Translational Medicine Research Institute Hospital for Sick Children Toronto ON Canada

5. Proteostasis Therapeutics Cambridge MA USA

6. Division of Molecular Genetics Hospital for Sick Children Toronto ON Canada

7. Department of Paediatrics University of Toronto Toronto ON Canada

8. Division of Respiratory Medicine Hospital for Sick Children Toronto ON Canada

Funder

Cystic Fibrosis Canada

Hospital for Sick Children