In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis-Reference-Cited by-同舟云学术

In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis

Published:2019-03-27 Issue:485 Volume:11 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Sun Xingshen¹^ORCID,Yi Yaling¹^ORCID,Yan Ziying¹^ORCID,Rosen Bradley H.¹²^ORCID,Liang Bo¹,Winter Michael C.¹,Evans T. Idil Apak¹,Rotti Pavana G.¹,Yang Yu¹^ORCID,Gray Jaimie S.¹,Park Soo Yeun¹,Zhou Weihong¹,Zhang Yulong¹,Moll Shashanna R.¹,Woody Lisa³^ORCID,Tran Dao M.³,Jiang Licong³^ORCID,Vonk Annelotte M.⁴^ORCID,Beekman Jeffrey M.⁴,Negulescu Paul³,Van Goor Fred³,Fiorino Dennis F.³^ORCID,Gibson-Corley Katherine N.⁵,Engelhardt John F.¹²^ORCID

Affiliation:

1. Department of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.

2. Department of Internal Medicine, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.

3. Vertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.

4. Pediatric Pulmonology and Regenerative Medicine Center, University Medical Center Utrecht, Utrecht, Netherlands.

5. Department of Pathology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.

Abstract

In utero and early postnatal functional rescue of cystic fibrosis transmembrane conductance regulator improves survival in cystic fibrosis ferrets.

Funder

National Heart, Lung, and Blood Institute

National Institute of Diabetes and Digestive and Kidney Diseases

Cystic Fibrosis Foundation

Vertex Pharmaceuticals

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference35 articles.

1. Cystic fibrosis

2. A new era in the treatment of cystic fibrosis: correction of the underlying CFTR defect

3. Therapeutic approaches to CFTR dysfunction: From discovery to drug development

4. Corrector combination therapies for F508del-CFTR

5. Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

Cited by 127 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Inhalation of SP-101 Followed by Inhaled Doxorubicin Results in Robust and Durable hCFTRΔR Transgene Expression in the Airways of Wild-Type and Cystic Fibrosis Ferrets;Human Gene Therapy;2024-09-04

2. Cystic fibrosis;Nature Reviews Disease Primers;2024-08-08

3. Nutritional status in the era of highly effective CFTR modulators;Pediatric Pulmonology;2024-08-06

4. Amelioration of airway and GI disease in G551D-CF ferrets by AAV1 and AAV6;Gene Therapy;2024-07-28

5. In Utero Gene Therapy and its Application in Genetic Hearing Loss;Advanced Biology;2024-07-15