Engineering adeno-associated virus vectors for gene therapy.
作者： Chengwen Li , R. Jude Samulski
DOI: 10.1038/S41576-019-0205-4
关键词:
摘要: … However, current research indicates that the genetic modification of AAV vectors may further facilitate the success of AAV gene therapy. Vector engineering can increase AAV …
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Dawn E Bowles, Scott WJ 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, Phase 1 Gene Therapy for Duchenne Muscular Dystrophy Using a Translational Optimized AAV Vector Molecular Therapy. ,vol. 20, pp. 443- 455 ,(2012) , 10.1038/MT.2011.237
C. H. Srivastava, R. J. Samulski, L. Lu, S. H. Larsen, A. Srivastava, Construction of a recombinant human parvovirus B19: adeno-associated virus 2 (AAV) DNA inverted terminal repeats are functional in an AAV-B19 hybrid virus. Proceedings of the National Academy of Sciences of the United States of America. ,vol. 86, pp. 8078- 8082 ,(1989) , 10.1073/PNAS.86.20.8078
Melvin Y Rincon, Shilpita Sarcar, Dina Danso-Abeam, Marleen Keyaerts, Janka Matrai, Ermira Samara-Kuko, Abel Acosta-Sanchez, Takis Athanasopoulos, George Dickson, Tony Lahoutte, Pieter De Bleser, Thierry VandenDriessche, Marinee K Chuah, None, Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy. Molecular Therapy. ,vol. 23, pp. 43- 52 ,(2015) , 10.1038/MT.2014.178
C. Li, K. Goudy, M. Hirsch, A. Asokan, Y. Fan, J. Alexander, J. Sun, P. Monahan, D. Seiber, J. Sidney, A. Sette, R. Tisch, J. Frelinger, R. J. Samulski, Cellular immune response to cryptic epitopes during therapeutic gene transfer Proceedings of the National Academy of Sciences of the United States of America. ,vol. 106, pp. 10770- 10774 ,(2009) , 10.1073/PNAS.0902269106
Anai Gonzalez-Cordero, Emma L West, Rachael A Pearson, Yanai Duran, Livia S Carvalho, Colin J Chu, Arifa Naeem, Samuel J I Blackford, Anastasios Georgiadis, Jorn Lakowski, Mike Hubank, Alexander J Smith, James W B Bainbridge, Jane C Sowden, Robin R Ali, Photoreceptor precursors derived from three-dimensional embryonic stem cell cultures integrate and mature within adult degenerate retina Nature Biotechnology. ,vol. 31, pp. 741- 747 ,(2013) , 10.1038/NBT.2643
Eric Hastie, R. Jude Samulski, Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective. Human Gene Therapy. ,vol. 26, pp. 257- 265 ,(2015) , 10.1089/HUM.2015.025
Jennifer B Nietupski, Gregory D Hurlbut, Robin J Ziegler, Qiuming Chu, Bradley L Hodges, Karen M Ashe, Mark Bree, Seng H Cheng, Richard J Gregory, John Marshall, Ronald K Scheule, Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression Molecular Therapy. ,vol. 19, pp. 1999- 2011 ,(2011) , 10.1038/MT.2011.119
Oliver J Müller, Felix Kaul, Matthew D Weitzman, Renata Pasqualini, Wadih Arap, Jürgen A Kleinschmidt, Martin Trepel, Random peptide libraries displayed on adeno-associated virus to select for targeted gene therapy vectors Nature Biotechnology. ,vol. 21, pp. 1040- 1046 ,(2003) , 10.1038/NBT856
Richard H Smith, Justin R Levy, Robert M Kotin, A Simplified Baculovirus-AAV Expression Vector System Coupled With One-step Affinity Purification Yields High-titer rAAV Stocks From Insect Cells Molecular Therapy. ,vol. 17, pp. 1888- 1896 ,(2009) , 10.1038/MT.2009.128
Hilda Petrs-Silva, Astra Dinculescu, Qiuhong Li, Seok-Hong Min, Vince Chiodo, Ji-Jing Pang, Li Zhong, Sergei Zolotukhin, Arun Srivastava, Alfred S Lewin, William W Hauswirth, High-efficiency Transduction of the Mouse Retina by Tyrosine-mutant AAV Serotype Vectors Molecular Therapy. ,vol. 17, pp. 463- 471 ,(2009) , 10.1038/MT.2008.269