Phase I Trial of Intranasal and Endobronchial Administration of a Recombinant Adeno-Associated Virus Serotype 2 (rAAV2)-CFTR Vector in Adult Cystic Fibrosis Patients: A Two-Part Clinical Study-Reference-Cited by-同舟云学术

Phase I Trial of Intranasal and Endobronchial Administration of a Recombinant Adeno-Associated Virus Serotype 2 (rAAV2)-CFTR Vector in Adult Cystic Fibrosis Patients: A Two-Part Clinical Study

Published:2003-07-20 Issue:11 Volume:14 Page:1079-1088
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Flotte Terence R.¹,Zeitlin Pamela L.²,Reynolds Thomas C.³,Heald Alison E.⁴,Pedersen Patty⁴,Beck Suzanne⁵,Conrad Carol K.³,Brass-Ernst Lois²,Humphries Margaret⁶,Sullivan Kevin⁶,Wetzel Randall⁷,Taylor George²,Carter Barrie J.⁴,Guggino William B.²

Affiliation:

1. Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610-0296; Department of Pediatrics and Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21205

2. Department of Pediatrics and Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21205

3. Department of Pediatrics and Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21205; Stanford University, Pediatric Allergy and Pulmonology, Stanford, CA 94305

4. Targeted Genetics Corporation, Seattle, WA 98101

5. Department of Pediatrics and Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21205; St. Christopher's Hospital for Children, Philadelphia, PA 19102

6. Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610-0296

7. Department of Pediatrics and Eudowood Division of Pediatric Respiratory Sciences, Johns Hopkins University School of Medicine, Baltimore, MD 21205; Children's Hospital, USC School of Medicine, Los Angeles, CA 90089

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/104303403322124792

Reference24 articles.

1. In vivo model of adeno-associated virus vector persistence and rescue

2. A Phase I Study of Aerosolized Administration of tgAAVCF to Cystic Fibrosis Subjects with Mild Lung Disease

3. Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus

4. Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR

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