Literature DB >> 22516095

Virus-mediated gene delivery for human gene therapy.

Mauro Giacca1, Serena Zacchigna.   

Abstract

After over 20 years from the first application of gene transfer in humans, gene therapy is now a mature discipline, which has progressively overcome several of the hurdles that prevented clinical success in the early stages of application. So far, the vast majority of gene therapy clinical trials have exploited viral vectors as very efficient nucleic acid delivery vehicles both in vivo and ex vivo. Here we summarize the current status of viral gene transfer for clinical applications, with special emphasis on the molecular properties of the major classes of viral vectors and the information so far obtained from gene therapy clinical trials.
Copyright © 2012 Elsevier B.V. All rights reserved.

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Year:  2012        PMID: 22516095     DOI: 10.1016/j.jconrel.2012.04.008

Source DB:  PubMed          Journal:  J Control Release        ISSN: 0168-3659            Impact factor:   9.776


  84 in total

1.  Controlled Transcription of Exogenous mRNA in Platelets Using Protocells.

Authors:  Vivienne Chan; Stefanie K Novakowski; Simon Law; Christa Klein-Bosgoed; Christian J Kastrup
Journal:  Angew Chem Int Ed Engl       Date:  2015-09-14       Impact factor: 15.336

2.  Inhibition of the NF-κB pathway by R65 ribozyme gene via adeno-associated virus serotype 9 ameliorated oxidized LDL induced human umbilical vein endothelial cell injury.

Authors:  Hui Zhai; Qing-Jie Chen; Xiao-Ming Gao; Yi-Tong Ma; Bang-Dang Chen; Zi-Xiang Yu; Xiao-Mei Li; Fen Liu; Yang Xiang; Jia Xie; Yi-Ning Yang
Journal:  Int J Clin Exp Pathol       Date:  2015-09-01

Review 3.  High-Density Lipoproteins: Nature's Multifunctional Nanoparticles.

Authors:  Rui Kuai; Dan Li; Y Eugene Chen; James J Moon; Anna Schwendeman
Journal:  ACS Nano       Date:  2016-02-25       Impact factor: 15.881

4.  POxylation as an alternative stealth coating for biomedical applications.

Authors:  Herdis Bludau; Anna E Czapar; Andrzej S Pitek; Sourabh Shukla; Rainer Jordan; Nicole F Steinmetz
Journal:  Eur Polym J       Date:  2016-11-05       Impact factor: 4.598

Review 5.  Baculovirus: an insect-derived vector for diverse gene transfer applications.

Authors:  Kari J Airenne; Yu-Chen Hu; Thomas A Kost; Richard H Smith; Robert M Kotin; Chikako Ono; Yoshiharu Matsuura; Shu Wang; Seppo Ylä-Herttuala
Journal:  Mol Ther       Date:  2013-02-26       Impact factor: 11.454

6.  Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I.

Authors:  L Bočkor; G Bortolussi; A Iaconcig; G Chiaruttini; C Tiribelli; M Giacca; F Benvenuti; L Zentilin; A F Muro
Journal:  Gene Ther       Date:  2017-08-14       Impact factor: 5.250

7.  Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts.

Authors:  Jian Ding; Zhi-Qiang Lin; Jian-Ming Jiang; Christine E Seidman; Jonathan G Seidman; William T Pu; Da-Zhi Wang
Journal:  J Vis Exp       Date:  2016-12-17       Impact factor: 1.355

Review 8.  State-of-the-art human gene therapy: part I. Gene delivery technologies.

Authors:  Dan Wang; Guangping Gao
Journal:  Discov Med       Date:  2014 Jul-Aug       Impact factor: 2.970

9.  Polymyxin B treatment improves bactofection efficacy and reduces cytotoxicity.

Authors:  Charles H Jones; Snehal Rane; Emily Patt; Anitha Ravikrishnan; Chih-Kuang Chen; Chong Cheng; Blaine A Pfeifer
Journal:  Mol Pharm       Date:  2013-10-18       Impact factor: 4.939

Review 10.  Human Cardiac Gene Therapy.

Authors:  Kiyotake Ishikawa; Thomas Weber; Roger J Hajjar
Journal:  Circ Res       Date:  2018-08-17       Impact factor: 17.367

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