Literature DB >> 33570612

No CpGs for AAVs?

Lindsey A George1.   

Abstract

Year:  2021        PMID: 33570612      PMCID: PMC7885819          DOI: 10.1182/blood.2020009285

Source DB:  PubMed          Journal:  Blood        ISSN: 0006-4971            Impact factor:   22.113


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  10 in total

1.  BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression.

Authors:  Barbara A Konkle; Christopher E Walsh; Miguel A Escobar; Neil C Josephson; Guy Young; Annette von Drygalski; Scott W J McPhee; R Jude Samulski; Ivan Bilic; Maurus de la Rosa; Birgit M Reipert; Hanspeter Rottensteiner; Friedrich Scheiflinger; John C Chapin; Bruce Ewenstein; Paul E Monahan
Journal:  Blood       Date:  2021-02-11       Impact factor: 22.113

2.  Subacute Liver Failure Following Gene Replacement Therapy for Spinal Muscular Atrophy Type 1.

Authors:  Amy G Feldman; Julie A Parsons; Cullen M Dutmer; Aravindhan Veerapandiyan; Einar Hafberg; Nolan Maloney; Cara L Mack
Journal:  J Pediatr       Date:  2020-05-28       Impact factor: 4.406

3.  Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors:  Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal:  N Engl J Med       Date:  2017-11-02       Impact factor: 91.245

4.  Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

Authors:  Lindsey A George; Spencer K Sullivan; Adam Giermasz; John E J Rasko; Benjamin J Samelson-Jones; Jonathan Ducore; Adam Cuker; Lisa M Sullivan; Suvankar Majumdar; Jerome Teitel; Catherine E McGuinn; Margaret V Ragni; Alvin Y Luk; Daniel Hui; J Fraser Wright; Yifeng Chen; Yun Liu; Katie Wachtel; Angela Winters; Stefan Tiefenbacher; Valder R Arruda; Johannes C M van der Loo; Olga Zelenaia; Daniel Takefman; Marcus E Carr; Linda B Couto; Xavier M Anguela; Katherine A High
Journal:  N Engl J Med       Date:  2017-12-07       Impact factor: 91.245

5.  CpG-depleted adeno-associated virus vectors evade immune detection.

Authors:  Susan M Faust; Peter Bell; Benjamin J Cutler; Scott N Ashley; Yanqing Zhu; Joseph E Rabinowitz; James M Wilson
Journal:  J Clin Invest       Date:  2013-06-17       Impact factor: 14.808

6.  X-linked thrombophilia with a mutant factor IX (factor IX Padua).

Authors:  Paolo Simioni; Daniela Tormene; Giulio Tognin; Sabrina Gavasso; Cristiana Bulato; Nicholas P Iacobelli; Jonathan D Finn; Luca Spiezia; Claudia Radu; Valder R Arruda
Journal:  N Engl J Med       Date:  2009-10-22       Impact factor: 91.245

7.  CD8(+) T-cell responses to adeno-associated virus capsid in humans.

Authors:  Federico Mingozzi; Marcela V Maus; Daniel J Hui; Denise E Sabatino; Samuel L Murphy; John E J Rasko; Margaret V Ragni; Catherine S Manno; Jurg Sommer; Haiyan Jiang; Glenn F Pierce; Hildegund C J Ertl; Katherine A High
Journal:  Nat Med       Date:  2007-03-18       Impact factor: 53.440

8.  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

9.  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

Review 10.  Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy.

Authors:  Benjamin J Samelson-Jones; Valder R Arruda
Journal:  Mol Ther Methods Clin Dev       Date:  2018-12-31       Impact factor: 6.698

  10 in total
  2 in total

Review 1.  Durability of transgene expression after rAAV gene therapy.

Authors:  Manish Muhuri; Daniel I Levy; Martin Schulz; Douglas McCarty; Guangping Gao
Journal:  Mol Ther       Date:  2022-03-10       Impact factor: 12.910

2.  Gene therapy - are we ready now?

Authors:  Radoslaw Kaczmarek
Journal:  Haemophilia       Date:  2022-05       Impact factor: 4.263

  2 in total

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