Literature DB >> 29951572

Nab Escaping AAV Mutants Isolated from Mouse Muscles.

Zheng Chai1, R Jude Samulski1,2, Chengwen Li1,3.   

Abstract

Neutralizing antibodies (Nabs) are a major challenge in clinical trials of adeno-associated virus (AAV) vector gene therapy, because Nabs are able to inhibit AAV transduction in patients. We have successfully isolated several novel Nab-escaped AAV chimeric capsids in mice by administrating a mixture of AAV shuffled library and patient serum. These AAV chimeric capsid mutants enhanced Nab evasion from patient serum with a high muscle transduction efficacy. In this protocol, we describe the procedures for selection of the Nab-escaped AAV chimeric capsid, including isolation and characterization of Nab-escaping AAV mutants in mice muscle.

Entities:  

Keywords:  AAV; Chimeric capsid; Mouse muscle; Nab-escaping AAV

Year:  2018        PMID： 29951572      PMCID： PMC6018009          DOI： 10.21769/BioProtoc.2841

Source DB:  PubMed          Journal:  Bio Protoc        ISSN： 2331-8325


  5 in total

1.  Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector.

Authors:  Dawn E Bowles; Scott W J McPhee; Chengwen Li; Steven J Gray; Jade J Samulski; Angelique S Camp; Juan Li; Bing Wang; Paul E Monahan; Joseph E Rabinowitz; Joshua C Grieger; Lakshmanan Govindasamy; Mavis Agbandje-McKenna; Xiao Xiao; R Jude Samulski
Journal:  Mol Ther       Date:  2011-11-08       Impact factor: 11.454

2.  Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus.

Authors:  X Xiao; J Li; R J Samulski
Journal:  J Virol       Date:  1998-03       Impact factor: 5.103

3.  A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection.

Authors:  Lin Yang; Jiangang Jiang; Lauren M Drouin; Mavis Agbandje-McKenna; Chunlian Chen; Chunping Qiao; Dongqiuye Pu; Xiaoyun Hu; Da-Zhi Wang; Juan Li; Xiao Xiao
Journal:  Proc Natl Acad Sci U S A       Date:  2009-02-20       Impact factor: 11.205

4.  Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer.

Authors:  Chengwen Li; Shuqing Wu; Blake Albright; Matthew Hirsch; Wuping Li; Yu-Shan Tseng; Mavis Agbandje-McKenna; Scott McPhee; Aravind Asokan; R Jude Samulski
Journal:  Mol Ther       Date:  2015-07-29       Impact factor: 11.454

5.  Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles.

Authors:  Wuping Li; Aravind Asokan; Zhijian Wu; Terry Van Dyke; Nina DiPrimio; Jarrod S Johnson; Lakshmanan Govindaswamy; Mavis Agbandje-McKenna; Stefan Leichtle; D Eugene Redmond; Thomas J McCown; Kimberly B Petermann; Norman E Sharpless; Richard J Samulski
Journal:  Mol Ther       Date:  2008-05-20       Impact factor: 11.454
  5 in total
  3 in total

Review 1.  Adeno-Associated Viruses (AAV) and Host Immunity - A Race Between the Hare and the Hedgehog.

Authors:  Kleopatra Rapti; Dirk Grimm
Journal:  Front Immunol       Date:  2021-10-29       Impact factor: 7.561

2.  Chimeric Mice Engrafted With Canine Hepatocytes Exhibits Similar AAV Transduction Efficiency to Hemophilia B Dog.

Authors:  Wenwei Shao; Junjiang Sun; Xiaojing Chen; Amanda Dobbins; Elizabeth P Merricks; R Jude Samulski; Timothy C Nichols; Chengwen Li
Journal:  Front Pharmacol       Date:  2022-01-31       Impact factor: 5.810

Review 3.  Advances of adeno-associated virus applied in gene therapy to hemophilia from bench work to the clinical use.

Authors:  Xiaolei Pei; Mingzhe Han; Lei Zhang
Journal:  Blood Sci       Date:  2019-10-21
  3 in total

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