Literature DB >> 26689120

Selecting the Best AAV Capsid for Human Studies.

Mark A Kay1.   

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Year:  2015        PMID: 26689120      PMCID: PMC4700120          DOI: 10.1038/mt.2015.206

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   11.454


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  7 in total

1.  Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.

Authors:  Shaoyong Li; Chen Ling; Li Zhong; Mengxin Li; Qin Su; Ran He; Qiushi Tang; Dale L Greiner; Leonard D Shultz; Michael A Brehm; Terence R Flotte; Christian Mueller; Arun Srivastava; Guangping Gao
Journal:  Mol Ther       Date:  2015-09-25       Impact factor: 11.454

2.  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

3.  Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.

Authors:  Catherine S Manno; Glenn F Pierce; Valder R Arruda; Bertil Glader; Margaret Ragni; John J Rasko; John Rasko; Margareth C Ozelo; Keith Hoots; Philip Blatt; Barbara Konkle; Michael Dake; Robin Kaye; Mahmood Razavi; Albert Zajko; James Zehnder; Pradip K Rustagi; Hiroyuki Nakai; Amy Chew; Debra Leonard; J Fraser Wright; Ruth R Lessard; Jürg M Sommer; Michael Tigges; Denise Sabatino; Alvin Luk; Haiyan Jiang; Federico Mingozzi; Linda Couto; Hildegund C Ertl; Katherine A High; Mark A Kay
Journal:  Nat Med       Date:  2006-02-12       Impact factor: 53.440

4.  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

5.  Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Authors:  Amit C Nathwani; Ulreke M Reiss; Edward G D Tuddenham; Cecilia Rosales; Pratima Chowdary; Jenny McIntosh; Marco Della Peruta; Elsa Lheriteau; Nishal Patel; Deepak Raj; Anne Riddell; Jun Pie; Savita Rangarajan; David Bevan; Michael Recht; Yu-Min Shen; Kathleen G Halka; Etiena Basner-Tschakarjan; Federico Mingozzi; Katherine A High; James Allay; Mark A Kay; Catherine Y C Ng; Junfang Zhou; Maria Cancio; Christopher L Morton; John T Gray; Deokumar Srivastava; Arthur W Nienhuis; Andrew M Davidoff
Journal:  N Engl J Med       Date:  2014-11-20       Impact factor: 176.079

6.  Development and rescue of human familial hypercholesterolaemia in a xenograft mouse model.

Authors:  Beatrice Bissig-Choisat; Lili Wang; Xavier Legras; Pradip K Saha; Leon Chen; Peter Bell; Francis P Pankowicz; Matthew C Hill; Mercedes Barzi; Claudia Kettlun Leyton; Hon-Chiu Eastwood Leung; Robert L Kruse; Ryan W Himes; John A Goss; James M Wilson; Lawrence Chan; William R Lagor; Karl-Dimiter Bissig
Journal:  Nat Commun       Date:  2015-06-17       Impact factor: 14.919

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

  7 in total
  7 in total

Review 1.  Outlook and future of inner ear therapy.

Authors:  Jenna Devare; Samuel Gubbels; Yehoash Raphael
Journal:  Hear Res       Date:  2018-05-17       Impact factor: 3.208

2.  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

3.  Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity.

Authors:  Nicole K Paulk; Katja Pekrun; Erhua Zhu; Sean Nygaard; Bin Li; Jianpeng Xu; Kirk Chu; Christian Leborgne; Allison P Dane; Annelise Haft; Yue Zhang; Feijie Zhang; Chris Morton; Marcus B Valentine; Andrew M Davidoff; Amit C Nathwani; Federico Mingozzi; Markus Grompe; Ian E Alexander; Leszek Lisowski; Mark A Kay
Journal:  Mol Ther       Date:  2017-09-25       Impact factor: 11.454

4.  Blood-brain barrier shuttle peptides enhance AAV transduction in the brain after systemic administration.

Authors:  Xintao Zhang; Ting He; Zheng Chai; R Jude Samulski; Chengwen Li
Journal:  Biomaterials       Date:  2018-05-26       Impact factor: 12.479

5.  Long-Term Sustained Effect of Liver-Targeted Adeno-Associated Virus Gene Therapy for Mitochondrial Neurogastrointestinal Encephalomyopathy.

Authors:  Javier Torres-Torronteras; Raquel Cabrera-Pérez; Ferran Vila-Julià; Carlo Viscomi; Yolanda Cámara; Michio Hirano; Massimo Zeviani; Ramon Martí
Journal:  Hum Gene Ther       Date:  2018-02-26       Impact factor: 4.793

6.  An RNA Interference/Adeno-Associated Virus Vector-Based Combinatorial Gene Therapy Approach Against Hepatitis E Virus.

Authors:  Viet Loan Dao Thi; Dirk Grimm; Cindy Zhang; Andrew Freistaedter; Carolin Schmelas; Manuel Gunkel
Journal:  Hepatol Commun       Date:  2021-10-31

Review 7.  Engineering adeno-associated virus vectors for gene therapy.

Authors:  Chengwen Li; R Jude Samulski
Journal:  Nat Rev Genet       Date:  2020-02-10       Impact factor: 59.581

  7 in total

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