Literature DB >> 33472034

Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths Forward.

Soon H Choi1, John F Engelhardt2.   

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Year:  2021        PMID： 33472034      PMCID： PMC7854343          DOI： 10.1016/j.ymthe.2021.01.010

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   12.910


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  15 in total

1.  Directed evolution of adeno-associated virus to an infectious respiratory virus.

Authors:  Katherine J D A Excoffon; James T Koerber; David D Dickey; Matthew Murtha; Shaf Keshavjee; Brian K Kaspar; Joseph Zabner; David V Schaffer
Journal:  Proc Natl Acad Sci U S A       Date:  2009-02-23       Impact factor: 11.205

2.  Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus.

Authors:  J A Wagner; T Reynolds; M L Moran; R B Moss; J J Wine; T R Flotte; P Gardner
Journal:  Lancet       Date:  1998-06-06       Impact factor: 79.321

Review 3.  New animal models of cystic fibrosis: what are they teaching us?

Authors:  Nicholas W Keiser; John F Engelhardt
Journal:  Curr Opin Pulm Med       Date:  2011-11       Impact factor: 3.155

4.  Lung phenotype of juvenile and adult cystic fibrosis transmembrane conductance regulator-knockout ferrets.

Authors:  Xingshen Sun; Alicia K Olivier; Bo Liang; Yaling Yi; Hongshu Sui; Turan I A Evans; Yulong Zhang; Weihong Zhou; Scott R Tyler; John T Fisher; Nicholas W Keiser; Xiaoming Liu; Ziying Yan; Yi Song; J Adam Goeken; Joann M Kinyon; Danielle Fligg; Xiaoyan Wang; Weiliang Xie; Thomas J Lynch; Paul M Kaminsky; Zoe A Stewart; R Marshall Pope; Timothy Frana; David K Meyerholz; Kalpaj Parekh; John F Engelhardt
Journal:  Am J Respir Cell Mol Biol       Date:  2014-03       Impact factor: 6.914

5.  Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors.

Authors:  Ziying Yan; Roman Zak; G W Gant Luxton; Teresa C Ritchie; Ursula Bantel-Schaal; John F Engelhardt
Journal:  J Virol       Date:  2002-03       Impact factor: 5.103

6.  Optimization of Recombinant Adeno-Associated Virus-Mediated Expression for Large Transgenes, Using a Synthetic Promoter and Tandem Array Enhancers.

Authors:  Ziying Yan; Xingshen Sun; Zehua Feng; Guiying Li; John T Fisher; Zoe A Stewart; John F Engelhardt
Journal:  Hum Gene Ther       Date:  2015-04-20       Impact factor: 5.695

7.  Biological Differences in rAAV Transduction of Airway Epithelia in Humans and in Old World Non-human Primates.

Authors:  Xiaoming Liu; Meihui Luo; Cyndi Trygg; Ziying Yan; Diana C M Lei-Butters; Carolina I Smith; Anne C Fischer; Keith Munson; William B Guggino; Bruce A Bunnell; John F Engelhardt
Journal:  Mol Ther       Date:  2007-07-31       Impact factor: 11.454

8.  Repeated aerosolized AAV-CFTR for treatment of cystic fibrosis: a randomized placebo-controlled phase 2B trial.

Authors:  Richard B Moss; Carlos Milla; John Colombo; Frank Accurso; Pamela L Zeitlin; John P Clancy; L Terry Spencer; Joseph Pilewski; David A Waltz; Henry L Dorkin; Thomas Ferkol; Mark Pian; Bonnie Ramsey; Barrie J Carter; Dana B Martin; Alison E Heald
Journal:  Hum Gene Ther       Date:  2007-08       Impact factor: 5.695

9.  Infection Is Not Required for Mucoinflammatory Lung Disease in CFTR-Knockout Ferrets.

Authors:  Bradley H Rosen; T Idil Apak Evans; Shashanna R Moll; Jaimie S Gray; Bo Liang; Xingshen Sun; Yulong Zhang; Chandler W Jensen-Cody; Anthony M Swatek; Weihong Zhou; Nan He; Pavana G Rotti; Scott R Tyler; Nicholas W Keiser; Preston J Anderson; Leonard Brooks; Yalan Li; R Marshall Pope; Maheen Rajput; Eric A Hoffman; Kai Wang; J Kirk Harris; Kalpaj R Parekh; Katherine N Gibson-Corley; John F Engelhardt
Journal:  Am J Respir Crit Care Med       Date:  2018-05-15       Impact factor: 30.528

10.  Highly Efficient Transgenesis in Ferrets Using CRISPR/Cas9-Mediated Homology-Independent Insertion at the ROSA26 Locus.

Authors:  Miao Yu; Xingshen Sun; Scott R Tyler; Bo Liang; Anthony M Swatek; Thomas J Lynch; Nan He; Feng Yuan; Zehua Feng; Pavana G Rotti; Soon H Choi; Weam Shahin; Xiaoming Liu; Ziying Yan; John F Engelhardt
Journal:  Sci Rep       Date:  2019-02-13       Impact factor: 4.379
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