Literature DB >> 28054581

Do we need marker gene studies in humans to improve clinical AAV gene therapy?

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Year:  2017        PMID： 28054581      PMCID： PMC5862146          DOI： 10.1038/gt.2016.84

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


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  9 in total

1.  Fixing the genes.

Authors:  Leon Jaroff
Journal:  Time       Date:  1999-01-11

2.  Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union.

Authors:  Seppo Ylä-Herttuala
Journal:  Mol Ther       Date:  2012-10       Impact factor: 11.454

3.  Adeno-associated virus liver transduction efficiency measured by in vivo [18F]FHBG positron emission tomography imaging in rodents and nonhuman primates.

Authors:  Astrid Pañeda; Maria Collantes; Stuart G Beattie; Itzia Otano; Jolanda Snapper; Eric Timmermans; Laura Guembe; Harald Petry; Jose Luis Lanciego; Alberto Benito; Jesus Prieto; Maria Sol Rodriguez-Pena; Iván Peñuelas; Gloria Gonzalez-Aseguinolaza
Journal:  Hum Gene Ther       Date:  2011-04-06       Impact factor: 5.695

4.  Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Authors:  Jean Bennett; Jennifer Wellman; Kathleen A Marshall; Sarah McCague; Manzar Ashtari; Julie DiStefano-Pappas; Okan U Elci; Daniel C Chung; Junwei Sun; J Fraser Wright; Dominique R Cross; Puya Aravand; Laura L Cyckowski; Jeannette L Bennicelli; Federico Mingozzi; Alberto Auricchio; Eric A Pierce; Jason Ruggiero; Bart P Leroy; Francesca Simonelli; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2016-06-30       Impact factor: 79.321

5.  Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

Authors:  Amit C Nathwani; John T Gray; Catherine Y C Ng; Junfang Zhou; Yunyu Spence; Simon N Waddington; Edward G D Tuddenham; Geoffrey Kemball-Cook; Jenny McIntosh; Mariette Boon-Spijker; Koen Mertens; Andrew M Davidoff
Journal:  Blood       Date:  2005-12-01       Impact factor: 22.113

6.  Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Authors:  Amit C Nathwani; Edward G D Tuddenham; Savita Rangarajan; Cecilia Rosales; Jenny McIntosh; David C Linch; Pratima Chowdary; Anne Riddell; Arnulfo Jaquilmac Pie; Chris Harrington; James O'Beirne; Keith Smith; John Pasi; Bertil Glader; Pradip Rustagi; Catherine Y C Ng; Mark A Kay; Junfang Zhou; Yunyu Spence; Christopher L Morton; James Allay; John Coleman; Susan Sleep; John M Cunningham; Deokumar Srivastava; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; John T Gray; Ulrike M Reiss; Arthur W Nienhuis; Andrew M Davidoff
Journal:  N Engl J Med       Date:  2011-12-10       Impact factor: 176.079

7.  Selection and evaluation of clinically relevant AAV variants in a xenograft liver model.

Authors:  Leszek Lisowski; Allison P Dane; Kirk Chu; Yue Zhang; Sharon C Cunningham; Elizabeth M Wilson; Sean Nygaard; Markus Grompe; Ian E Alexander; Mark A Kay
Journal:  Nature       Date:  2013-12-25       Impact factor: 49.962

8.  Calcium upregulation by percutaneous administration of gene therapy in patients with cardiac disease (CUPID 2): a randomised, multinational, double-blind, placebo-controlled, phase 2b trial.

Authors:  Barry Greenberg; Javed Butler; G Michael Felker; Piotr Ponikowski; Adriaan A Voors; Akshay S Desai; Denise Barnard; Alain Bouchard; Brian Jaski; Alexander R Lyon; Janice M Pogoda; Jeffrey J Rudy; Krisztina M Zsebo
Journal:  Lancet       Date:  2016-01-21       Impact factor: 79.321

9.  Biological Heart Rate Reduction Through Genetic Suppression of Gα(s) Protein in the Sinoatrial Node.

Authors:  Patrick Lugenbiel; Alexander Bauer; Kamilla Kelemen; Patrick A Schweizer; Rüdiger Becker; Hugo A Katus; Dierk Thomas
Journal:  J Am Heart Assoc       Date:  2012-04-24       Impact factor: 5.501
  9 in total
  2 in total

1.  Gene Therapy with an Adeno-Associated Viral Vector Expressing Human Interleukin-2 Alters Immune System Homeostasis in Humanized Mice.

Authors:  Philip A Durost; Ken-Edwin Aryee; Fatima Manzoor; Roland M Tisch; Christian Mueller; Agata Jurczyk; Leonard D Shultz; Michael A Brehm
Journal:  Hum Gene Ther       Date:  2017-10-03       Impact factor: 5.695

2.  Hydroxylation of N-acetylneuraminic Acid Influences the in vivo Tropism of N-linked Sialic Acid-Binding Adeno-Associated Viruses AAV1, AAV5, and AAV6.

Authors:  Estrella Lopez-Gordo; Alejandro Orlowski; Arthur Wang; Alan Weinberg; Susmita Sahoo; Thomas Weber
Journal:  Front Med (Lausanne)       Date:  2021-12-21
  2 in total

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