Literature DB >> 25238276

Phase I/II study of intrapleural administration of a serotype rh.10 replication-deficient adeno-associated virus gene transfer vector expressing the human α1-antitrypsin cDNA to individuals with α1-antitrypsin deficiency.

Maria J Chiuchiolo1, Stephen M Kaminsky, Dolan Sondhi, Denesy Mancenido, Charleen Hollmann, Ronald G Crystal.   

Abstract

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Year:  2014        PMID： 25238276     DOI： 10.1089/humc.2014.2513

Source DB:  PubMed          Journal:  Hum Gene Ther Clin Dev        ISSN： 2324-8637            Impact factor:   5.032


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  10 in total

Review 1.  Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases.

Authors:  Itishri Sahu; A K M Ashiqul Haque; Brian Weidensee; Petra Weinmann; Michael S D Kormann
Journal:  Mol Ther       Date:  2019-03-06       Impact factor: 11.454

2.  Intrapleural Gene Therapy for Alpha-1 Antitrypsin Deficiency-Related Lung Disease.

Authors:  Katie M Stiles; Dolan Sondhi; Stephen M Kaminsky; Bishnu P De; Jonathan B Rosenberg; Ronald G Crystal
Journal:  Chronic Obstr Pulm Dis       Date:  2018-08-17

Review 3.  Gene Therapy for Alpha-1 Antitrypsin Deficiency Lung Disease.

Authors:  Maria J Chiuchiolo; Ronald G Crystal
Journal:  Ann Am Thorac Soc       Date:  2016-08

Review 4.  New Directions in Pulmonary Gene Therapy.

Authors:  Amber Vu; Paul B McCray
Journal:  Hum Gene Ther       Date:  2020-09       Impact factor: 4.793

Review 5.  Manufacturing of recombinant adeno-associated viral vectors for clinical trials.

Authors:  Nathalie Clément; Joshua C Grieger
Journal:  Mol Ther Methods Clin Dev       Date:  2016-03-16       Impact factor: 6.698

6.  Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

Authors:  Florie Borel; Qiushi Tang; Gwladys Gernoux; Cynthia Greer; Ziqiong Wang; Adi Barzel; Mark A Kay; Leonard D Shultz; Dale L Greiner; Terence R Flotte; Michael A Brehm; Christian Mueller
Journal:  Mol Ther       Date:  2017-09-25       Impact factor: 11.454

Review 7.  Lessons learned from lung and liver in-vivo gene therapy: implications for the future.

Authors:  Joost van Haasteren; Stephen C Hyde; Deborah R Gill
Journal:  Expert Opin Biol Ther       Date:  2018-08-10       Impact factor: 4.388

8.  Optogenetic activation of the diaphragm.

Authors:  Ethan S Benevides; Michael D Sunshine; Sabhya Rana; David D Fuller
Journal:  Sci Rep       Date:  2022-04-20       Impact factor: 4.996

Review 9.  Pulmonary transplantation of alpha-1 antitrypsin (AAT)-transgenic macrophages provides a source of functional human AAT in vivo.

Authors:  Ewa Janosz; Miriam Hetzel; Hanna Spielmann; Srinu Tumpara; Charlotte Rossdam; Marc Schwabbauer; Doreen Kloos; Constantin von Kaisenberg; Axel Schambach; Falk F R Buettner; Sabina Janciauskiene; Nico Lachmann; Thomas Moritz
Journal:  Gene Ther       Date:  2021-07-19       Impact factor: 5.250

Review 10.  Novel Insights into the Therapeutic Potential of Lung-Targeted Gene Transfer in the Most Common Respiratory Diseases.

Authors:  Malik Bisserier; Xiao-Qing Sun; Shahood Fazal; Irene C Turnbull; Sébastien Bonnet; Lahouaria Hadri
Journal:  Cells       Date:  2022-03-12       Impact factor: 7.666
  10 in total

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