Literature DB >> 28079048

Delivering efficient liver-directed AAV-mediated gene therapy.

J Baruteau1,2,3, S N Waddington3,4, I E Alexander5,6, P Gissen1,2,7.   

Abstract

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Year:  2017        PMID: 28079048     DOI: 10.1038/gt.2016.90

Source DB:  PubMed          Journal:  Gene Ther        ISSN: 0969-7128            Impact factor:   5.250


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  18 in total

1.  Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice.

Authors:  Carmen Unzu; Ana Sampedro; Itsaso Mauleón; Manuel Alegre; Stuart G Beattie; Rafael Enríquez de Salamanca; Jolanda Snapper; Jaap Twisk; Harald Petry; Gloria González-Aseguinolaza; Julio Artieda; María Sol Rodríguez-Pena; Jesús Prieto; Antonio Fontanellas
Journal:  Mol Ther       Date:  2010-09-28       Impact factor: 11.454

2.  The use of stable isotopes in biological and medical research.

Authors:  I M LONDON
Journal:  J Clin Invest       Date:  1949-11       Impact factor: 14.808

3.  Early clinical data raise the bar for hemophilia gene therapies.

Authors:  Elie Dolgin
Journal:  Nat Biotechnol       Date:  2016-10-11       Impact factor: 54.908

4.  Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.

Authors:  Koen Vercauteren; Brad E Hoffman; Irene Zolotukhin; Geoffrey D Keeler; Jing W Xiao; Etiena Basner-Tschakarjan; Katherine A High; Hildegund Cj Ertl; Charles M Rice; Arun Srivastava; Ype P de Jong; Roland W Herzog
Journal:  Mol Ther       Date:  2016-03-29       Impact factor: 11.454

5.  Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria.

Authors:  Astrid Pañeda; Esperanza Lopez-Franco; Christine Kaeppel; Carmen Unzu; Ana Gloria Gil-Royo; Delia D'Avola; Stuart G Beattie; Cristina Olagüe; Roberto Ferrero; Ana Sampedro; Itsaso Mauleon; Stephan Hermening; Florence Salmon; Alberto Benito; Juan Jose Gavira; María Eugenia Cornet; María del Mar Municio; Christof von Kalle; Harald Petry; Jesus Prieto; Manfred Schmidt; Antonio Fontanellas; Gloria González-Aseguinolaza
Journal:  Hum Gene Ther       Date:  2013-12       Impact factor: 5.695

6.  Induction and prevention of severe hyperammonemia in the spfash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery.

Authors:  Sharon C Cunningham; Cindy Y Kok; Allison P Dane; Kevin Carpenter; Eddy Kizana; Philip W Kuchel; Ian E Alexander
Journal:  Mol Ther       Date:  2011-03-08       Impact factor: 11.454

7.  In vivo monitoring of urea cycle activity with (13)C-acetate as a tracer of ureagenesis.

Authors:  Thomas Opladen; Martin Lindner; Anibh M Das; Thorsten Marquardt; Aneal Khan; Sukru H Emre; Barbara K Burton; Bruce A Barshop; Thea Böhm; Jochen Meyburg; Kathrin Zangerl; Sebene Mayorandan; Peter Burgard; Ulrich H N Dürr; Bernd Rosenkranz; Jörg Rennecke; Jens Derbinski; Marc Yudkoff; Georg F Hoffmann
Journal:  Mol Genet Metab       Date:  2015-11-14       Impact factor: 4.797

8.  Comparative Study of Liver Gene Transfer With AAV Vectors Based on Natural and Engineered AAV Capsids.

Authors:  Lili Wang; Peter Bell; Suryanarayan Somanathan; Qiang Wang; Zhenning He; Hongwei Yu; Deirdre McMenamin; Tamara Goode; Roberto Calcedo; James M Wilson
Journal:  Mol Ther       Date:  2015-09-28       Impact factor: 11.454

9.  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

10.  Adeno-associated viral vector serotype 5 poorly transduces liver in rat models.

Authors:  Paula S Montenegro-Miranda; Astrid Pañeda; Lysbeth ten Bloemendaal; Suzanne Duijst; Dirk R de Waart; Gloria Gonzalez- Aseguinolaza; Gloria Gonzalez Aseguinolaza; Piter J Bosma
Journal:  PLoS One       Date:  2013-12-27       Impact factor: 3.240

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  9 in total

1.  Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates.

Authors:  Douglas J Ballon; Jonathan B Rosenberg; Edward K Fung; Anastasia Nikolopoulou; Paresh Kothari; Bishnu P De; Bin He; Alvin Chen; Linda A Heier; Dolan Sondhi; Stephen M Kaminsky; Paul David Mozley; John W Babich; Ronald G Crystal
Journal:  Hum Gene Ther       Date:  2020-12       Impact factor: 5.695

Review 2.  Machine perfusion of the liver: applications in transplantation and beyond.

Authors:  Carlo D L Ceresa; David Nasralla; Joerg-Matthias Pollok; Peter J Friend
Journal:  Nat Rev Gastroenterol Hepatol       Date:  2022-01-07       Impact factor: 46.802

3.  Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.

Authors:  Julien Baruteau; Dany P Perocheau; Joanna Hanley; Maëlle Lorvellec; Eridan Rocha-Ferreira; Rajvinder Karda; Joanne Ng; Natalie Suff; Juan Antinao Diaz; Ahad A Rahim; Michael P Hughes; Blerida Banushi; Helen Prunty; Mariya Hristova; Deborah A Ridout; Alex Virasami; Simon Heales; Stewen J Howe; Suzanne M K Buckley; Philippa B Mills; Paul Gissen; Simon N Waddington
Journal:  Nat Commun       Date:  2018-08-29       Impact factor: 14.919

4.  Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.

Authors:  Marti Cabanes-Creus; Samantha L Ginn; Anais K Amaya; Sophia H Y Liao; Adrian Westhaus; Claus V Hallwirth; Patrick Wilmott; Jason Ward; Kimberley L Dilworth; Giorgia Santilli; Arkadiusz Rybicki; Hiroyuki Nakai; Adrian J Thrasher; Adrian C Filip; Ian E Alexander; Leszek Lisowski
Journal:  Mol Ther Methods Clin Dev       Date:  2018-11-01       Impact factor: 6.698

5.  Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.

Authors:  Dany P Perocheau; Sharon Cunningham; Juhee Lee; Juan Antinao Diaz; Simon N Waddington; Kimberly Gilmour; Simon Eaglestone; Leszek Lisowski; Adrian J Thrasher; Ian E Alexander; Paul Gissen; Julien Baruteau
Journal:  Hum Gene Ther       Date:  2018-10-19       Impact factor: 5.695

Review 6.  Gene therapy for inherited metabolic diseases.

Authors:  Berna Seker Yilmaz; Sonam Gurung; Dany Perocheau; John Counsell; Julien Baruteau
Journal:  J Mother Child       Date:  2020-11-10

Review 7.  Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.

Authors:  Julien Baruteau; Simon N Waddington; Ian E Alexander; Paul Gissen
Journal:  J Inherit Metab Dis       Date:  2017-05-31       Impact factor: 4.982

Review 8.  Gene Therapy for Chronic HBV-Can We Eliminate cccDNA?

Authors:  Kristie Bloom; Mohube Betty Maepa; Abdullah Ely; Patrick Arbuthnot
Journal:  Genes (Basel)       Date:  2018-04-12       Impact factor: 4.096

Review 9.  Succinic Semialdehyde Dehydrogenase Deficiency: An Update.

Authors:  Miroslava Didiášová; Antje Banning; Heiko Brennenstuhl; Sabine Jung-Klawitter; Claudio Cinquemani; Thomas Opladen; Ritva Tikkanen
Journal:  Cells       Date:  2020-02-19       Impact factor: 6.600

  9 in total

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