Literature DB >> 21499295

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.

Federico Mingozzi1, Katherine A High.   

Abstract

In vivo gene replacement for the treatment of inherited disease is one of the most compelling concepts in modern medicine. Adeno-associated virus (AAV) vectors have been extensively used for this purpose and have shown therapeutic efficacy in a range of animal models. Successful translation to the clinic was initially slow, but long-term expression of donated genes at therapeutic levels has now been achieved in patients with inherited retinal disorders and haemophilia B. Recent exciting results have raised hopes for the treatment of many other diseases. As we discuss here, the prospects and challenges for AAV gene therapy are to a large extent dependent on the target tissue and the specific disease.

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Year:  2011        PMID： 21499295     DOI： 10.1038/nrg2988

Source DB:  PubMed          Journal:  Nat Rev Genet        ISSN： 1471-0056            Impact factor:   53.242


  162 in total

1.  Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors.

Authors:  H Chao; Y Liu; J Rabinowitz; C Li; R J Samulski; C E Walsh
Journal:  Mol Ther       Date:  2000-12       Impact factor: 11.454

2.  Quantitative analysis of the packaging capacity of recombinant adeno-associated virus.

Authors:  J Y Dong; P D Fan; R A Frizzell
Journal:  Hum Gene Ther       Date:  1996-11-10       Impact factor: 5.695

3.  Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain.

Authors:  Cassia N Cearley; John H Wolfe
Journal:  Mol Ther       Date:  2006-01-18       Impact factor: 11.454

4.  Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector.

Authors:  Dolan Sondhi; Neil R Hackett; Daniel A Peterson; Jamie Stratton; Michael Baad; Kelly M Travis; James M Wilson; Ronald G Crystal
Journal:  Mol Ther       Date:  2006-12-19       Impact factor: 11.454

5.  Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1.

Authors:  Byron L Lam; William J Feuer; Fawzi Abukhalil; Vittorio Porciatti; William W Hauswirth; John Guy
Journal:  Arch Ophthalmol       Date:  2010-09

6.  Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia.

Authors:  Marina Cavazzana-Calvo; Emmanuel Payen; Olivier Negre; Gary Wang; Kathleen Hehir; Floriane Fusil; Julian Down; Maria Denaro; Troy Brady; Karen Westerman; Resy Cavallesco; Beatrix Gillet-Legrand; Laure Caccavelli; Riccardo Sgarra; Leila Maouche-Chrétien; Françoise Bernaudin; Robert Girot; Ronald Dorazio; Geert-Jan Mulder; Axel Polack; Arthur Bank; Jean Soulier; Jérôme Larghero; Nabil Kabbara; Bruno Dalle; Bernard Gourmel; Gérard Socie; Stany Chrétien; Nathalie Cartier; Patrick Aubourg; Alain Fischer; Kenneth Cornetta; Frédéric Galacteros; Yves Beuzard; Eliane Gluckman; Frederick Bushman; Salima Hacein-Bey-Abina; Philippe Leboulch
Journal:  Nature       Date:  2010-09-16       Impact factor: 49.962

7.  Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia.

Authors:  Corinna Lebherz; Guangping Gao; Jean-Pierre Louboutin; John Millar; Daniel Rader; James M Wilson
Journal:  J Gene Med       Date:  2004-06       Impact factor: 4.565

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.  Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease.

Authors:  Mario A Cabrera-Salazar; Eric M Roskelley; Jie Bu; Bradley L Hodges; Nelson Yew; James C Dodge; Lamya S Shihabuddin; Istvan Sohar; David E Sleat; Ronald K Scheule; Beverly L Davidson; Seng H Cheng; Peter Lobel; Marco A Passini
Journal:  Mol Ther       Date:  2007-07-17       Impact factor: 11.454

10.  Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.

Authors:  Amit C Nathwani; John T Gray; Jenny McIntosh; Catherine Y C Ng; Junfang Zhou; Yunyu Spence; Melanie Cochrane; Elaine Gray; Edward G D Tuddenham; Andrew M Davidoff
Journal:  Blood       Date:  2006-11-07       Impact factor: 22.113
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  398 in total

1.  Intranasal vaccination with AAV5 and 9 vectors against human papillomavirus type 16 in rhesus macaques.

Authors:  Karen Nieto; Christiane Stahl-Hennig; Barbara Leuchs; Martin Müller; Lutz Gissmann; Jürgen A Kleinschmidt
Journal:  Hum Gene Ther       Date:  2012-04-18       Impact factor: 5.695

2.  Molecular characterization of the newly identified human parvovirus 4 in the family Parvoviridae.

Authors:  Sai Lou; Baoyan Xu; Qinfeng Huang; Ning Zhi; Fang Cheng; Susan Wong; Kevin Brown; Eric Delwart; Zhengwen Liu; Jianming Qiu
Journal:  Virology       Date:  2011-10-30       Impact factor: 3.616

Review 3.  Cocaine hydrolase gene therapy for cocaine abuse.

Authors:  Stephen Brimijoin; Yang Gao
Journal:  Future Med Chem       Date:  2012-02       Impact factor: 3.808

4.  Incorporation of antigens into viral capsids augments immunogenicity of adeno-associated virus vector-based vaccines.

Authors:  Jan Rybniker; Angela Nowag; Hanna Janicki; Kai Demant; Pia Hartmann; Hildegard Büning
Journal:  J Virol       Date:  2012-10-03       Impact factor: 5.103

5.  Full-length dystrophin reconstitution with adeno-associated viral vectors.

Authors:  William Lostal; Kasun Kodippili; Yongping Yue; Dongsheng Duan
Journal:  Hum Gene Ther       Date:  2014-03-31       Impact factor: 5.695

6.  Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.

Authors:  Hiu Man Viecelli; Richard P Harbottle; Suet Ping Wong; Andrea Schlegel; Marinee K Chuah; Thierry VandenDriessche; Cary O Harding; Beat Thöny
Journal:  Hepatology       Date:  2014-07-29       Impact factor: 17.425

Review 7.  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

8.  Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression.

Authors:  Patrice Vidal; Serena Pagliarani; Pasqualina Colella; Helena Costa Verdera; Louisa Jauze; Monika Gjorgjieva; Francesco Puzzo; Solenne Marmier; Fanny Collaud; Marcelo Simon Sola; Severine Charles; Sabrina Lucchiari; Laetitia van Wittenberghe; Alban Vignaud; Bernard Gjata; Isabelle Richard; Pascal Laforet; Edoardo Malfatti; Gilles Mithieux; Fabienne Rajas; Giacomo Pietro Comi; Giuseppe Ronzitti; Federico Mingozzi
Journal:  Mol Ther       Date:  2017-12-28       Impact factor: 11.454

Review 9.  Adeno-associated virus as a gene therapy vector: strategies to neutralize the neutralizing antibodies.

Authors:  Majid Lotfinia; Meghdad Abdollahpour-Alitappeh; Behzad Hatami; Mohammad Reza Zali; Morteza Karimipoor
Journal:  Clin Exp Med       Date:  2019-05-03       Impact factor: 3.984

10.  CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes.

Authors:  Benjamin Steines; David D Dickey; Jamie Bergen; Katherine Jda Excoffon; John R Weinstein; Xiaopeng Li; Ziying Yan; Mahmoud H Abou Alaiwa; Viral S Shah; Drake C Bouzek; Linda S Powers; Nicholas D Gansemer; Lynda S Ostedgaard; John F Engelhardt; David A Stoltz; Michael J Welsh; Patrick L Sinn; David V Schaffer; Joseph Zabner
Journal:  JCI Insight       Date:  2016-09-08
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