Self-Reactive CFTR T Cells in Humans: Implications for Gene Therapy-Reference-Cited by-同舟云学术

Self-Reactive CFTR T Cells in Humans: Implications for Gene Therapy

Published:2013-09 Issue:3 Volume:24 Page:108-115
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Calcedo Roberto¹,Griesenbach Uta²³,Dorgan Daniel J.⁴,Soussi Samia²³,Boyd A. Christopher³⁵,Davies Jane C.²³,Higgins Tracy E.²³,Hyde Stephen C.³⁶,Gill Deborah R.³⁶,Innes J. Alastair³⁵,Porteous David J.³⁵,Alton Eric W.²³,Wilson James M.¹,Limberis Maria P.¹

Affiliation:

1. Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA 19104.

2. Department of Gene Therapy, Imperial College London, London, SW3 6LR, United Kingdom.

3. UK Cystic Fibrosis Gene Therapy Consortium.

4. Hospital of the University of Pennsylvania, Philadelphia, PA 19104.

5. Medical Genetics Section, University of Edinburgh, Edinburgh, EH4 2XU, United Kingdom.

6. Gene Medicine Research Group, University of Oxford, Oxford, OX3 9DU, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics(clinical)

Link

http://www.liebertpub.com/doi/pdf/10.1089/humc.2012.249

Reference15 articles.

1. Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector

2. The Role of Inflammation in the Pathophysiology of CF Lung Disease

3. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for theF508del-CFTRmutation

4. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a Phosphorylation-dependent but ATP-independent Manner

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