Michael F Naso, Brian Tomkowicz, William L Perry, William R Strohl. BioDrugs 2017
Times Cited: 418
Times Cited: 418
Times Cited
Times Co-cited
Similarity
Adeno-associated virus vector as a platform for gene therapy delivery.
Dan Wang, Phillip W L Tai, Guangping Gao. Nat Rev Drug Discov 2019
Dan Wang, Phillip W L Tai, Guangping Gao. Nat Rev Drug Discov 2019
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Emerging Issues in AAV-Mediated In Vivo Gene Therapy.
Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi. Mol Ther Methods Clin Dev 2017
Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi. Mol Ther Methods Clin Dev 2017
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Engineering adeno-associated virus vectors for gene therapy.
Chengwen Li, R Jude Samulski. Nat Rev Genet 2020
Chengwen Li, R Jude Samulski. Nat Rev Genet 2020
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Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors.
Sylvie Boutin, Virginie Monteilhet, Philippe Veron, Christian Leborgne, Olivier Benveniste, Marie Françoise Montus, Carole Masurier. Hum Gene Ther 2010
Sylvie Boutin, Virginie Monteilhet, Philippe Veron, Christian Leborgne, Olivier Benveniste, Marie Françoise Montus, Carole Masurier. Hum Gene Ther 2010
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Gene therapy using adeno-associated virus vectors.
Shyam Daya, Kenneth I Berns. Clin Microbiol Rev 2008
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Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.
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Gene therapy clinical trials worldwide to 2017: An update.
Samantha L Ginn, Anais K Amaya, Ian E Alexander, Michael Edelstein, Mohammad R Abedi. J Gene Med 2018
Samantha L Ginn, Anais K Amaya, Ian E Alexander, Michael Edelstein, Mohammad R Abedi. J Gene Med 2018
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Immune responses to AAV vectors: overcoming barriers to successful gene therapy.
Federico Mingozzi, Katherine A High. Blood 2013
Federico Mingozzi, Katherine A High. Blood 2013
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.
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AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer.
Helena Costa Verdera, Klaudia Kuranda, Federico Mingozzi. Mol Ther 2020
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Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.
Kevin D Foust, Emily Nurre, Chrystal L Montgomery, Anna Hernandez, Curtis M Chan, Brian K Kaspar. Nat Biotechnol 2009
Kevin D Foust, Emily Nurre, Chrystal L Montgomery, Anna Hernandez, Curtis M Chan, Brian K Kaspar. Nat Biotechnol 2009
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Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.
Christian Hinderer, Nathan Katz, Elizabeth L Buza, Cecilia Dyer, Tamara Goode, Peter Bell, Laura K Richman, James M Wilson. Hum Gene Ther 2018
Christian Hinderer, Nathan Katz, Elizabeth L Buza, Cecilia Dyer, Tamara Goode, Peter Bell, Laura K Richman, James M Wilson. Hum Gene Ther 2018
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Non viral vectors in gene therapy- an overview.
Murali Ramamoorth, Aparna Narvekar. J Clin Diagn Res 2015
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Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection.
Carmela Zincarelli, Stephen Soltys, Giuseppe Rengo, Joseph E Rabinowitz. Mol Ther 2008
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Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems.
Ken Y Chan, Min J Jang, Bryan B Yoo, Alon Greenbaum, Namita Ravi, Wei-Li Wu, Luis Sánchez-Guardado, Carlos Lois, Sarkis K Mazmanian, Benjamin E Deverman,[...]. Nat Neurosci 2017
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Effect of genome size on AAV vector packaging.
Zhijian Wu, Hongyan Yang, Peter Colosi. Mol Ther 2010
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Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.
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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.
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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
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The clinical landscape for AAV gene therapies.
Dmitry A Kuzmin, Maria V Shutova, Natalie R Johnston, Owen P Smith, Vasily V Fedorin, Yury S Kukushkin, Johannes C M van der Loo, Elaine C Johnstone. Nat Rev Drug Discov 2021
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Human Immune Responses to Adeno-Associated Virus (AAV) Vectors.
Giuseppe Ronzitti, David-Alexandre Gross, Federico Mingozzi. Front Immunol 2020
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Adenovirus-Mediated Gene Delivery: Potential Applications for Gene and Cell-Based Therapies in the New Era of Personalized Medicine.
Cody S Lee, Elliot S Bishop, Ruyi Zhang, Xinyi Yu, Evan M Farina, Shujuan Yan, Chen Zhao, Zongyue Zheng, Yi Shu, Xingye Wu,[...]. Genes Dis 2017
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Gene therapy comes of age.
Cynthia E Dunbar, Katherine A High, J Keith Joung, Donald B Kohn, Keiya Ozawa, Michel Sadelain. Science 2018
Cynthia E Dunbar, Katherine A High, J Keith Joung, Donald B Kohn, Keiya Ozawa, Michel Sadelain. Science 2018
7
Multiyear Follow-up of AAV5-hFVIII-SQ Gene Therapy for Hemophilia A.
K John Pasi, Savita Rangarajan, Nina Mitchell, Will Lester, Emily Symington, Bella Madan, Michael Laffan, Chris B Russell, Mingjin Li, Glenn F Pierce,[...]. N Engl J Med 2020
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6
Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors.
Steven J Gray, Stacey B Foti, Joel W Schwartz, Lavanya Bachaboina, Bonnie Taylor-Blake, Jennifer Coleman, Michael D Ehlers, Mark J Zylka, Thomas J McCown, R Jude Samulski. Hum Gene Ther 2011
Steven J Gray, Stacey B Foti, Joel W Schwartz, Lavanya Bachaboina, Bonnie Taylor-Blake, Jennifer Coleman, Michael D Ehlers, Mark J Zylka, Thomas J McCown, R Jude Samulski. Hum Gene Ther 2011
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Non-viral vectors for gene-based therapy.
Hao Yin, Rosemary L Kanasty, Ahmed A Eltoukhy, Arturo J Vegas, J Robert Dorkin, Daniel G Anderson. Nat Rev Genet 2014
Hao Yin, Rosemary L Kanasty, Ahmed A Eltoukhy, Arturo J Vegas, J Robert Dorkin, Daniel G Anderson. Nat Rev Genet 2014
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Lentiviral vectors in gene therapy: their current status and future potential.
David Escors, Karine Breckpot. Arch Immunol Ther Exp (Warsz) 2010
David Escors, Karine Breckpot. Arch Immunol Ther Exp (Warsz) 2010
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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain.
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Benjamin E Deverman, Piers L Pravdo, Bryan P Simpson, Sripriya Ravindra Kumar, Ken Y Chan, Abhik Banerjee, Wei-Li Wu, Bin Yang, Nina Huber, Sergiu P Pasca,[...]. Nat Biotechnol 2016
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Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.
Michaël Hocquemiller, Laura Giersch, Mickael Audrain, Samantha Parker, Nathalie Cartier. Hum Gene Ther 2016
Michaël Hocquemiller, Laura Giersch, Mickael Audrain, Samantha Parker, Nathalie Cartier. Hum Gene Ther 2016
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Dystrophin immunity in Duchenne's muscular dystrophy.
Jerry R Mendell, Katherine Campbell, Louise Rodino-Klapac, Zarife Sahenk, Chris Shilling, Sarah Lewis, Dawn Bowles, Steven Gray, Chengwen Li, Gloria Galloway,[...]. N Engl J Med 2010
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Adeno-associated viral vector-mediated immune responses: Understanding barriers to gene delivery.
Natalie F Nidetz, Michael C McGee, Longping V Tse, Chengwen Li, Le Cong, Yunxing Li, Weishan Huang. Pharmacol Ther 2020
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Gene Therapy Leaves a Vicious Cycle.
Reena Goswami, Gayatri Subramanian, Liliya Silayeva, Isabelle Newkirk, Deborah Doctor, Karan Chawla, Saurabh Chattopadhyay, Dhyan Chandra, Nageswararao Chilukuri, Venkaiah Betapudi. Front Oncol 2019
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6
Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates.
Steven J Gray, Valerie Matagne, Lavanya Bachaboina, Swati Yadav, Sergio R Ojeda, R Jude Samulski. Mol Ther 2011
Steven J Gray, Valerie Matagne, Lavanya Bachaboina, Swati Yadav, Sergio R Ojeda, R Jude Samulski. Mol Ther 2011
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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.
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,[...]. Lancet 2016
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Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity.
F Ann Ran, Patrick D Hsu, Chie-Yu Lin, Jonathan S Gootenberg, Silvana Konermann, Alexandro E Trevino, David A Scott, Azusa Inoue, Shogo Matoba, Yi Zhang,[...]. Cell 2013
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5
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
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AAV vector integration sites in mouse hepatocellular carcinoma.
Anthony Donsante, Daniel G Miller, Yi Li, Carole Vogler, Elizabeth M Brunt, David W Russell, Mark S Sands. Science 2007
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5
Search-and-replace genome editing without double-strand breaks or donor DNA.
Andrew V Anzalone, Peyton B Randolph, Jessie R Davis, Alexander A Sousa, Luke W Koblan, Jonathan M Levy, Peter J Chen, Christopher Wilson, Gregory A Newby, Aditya Raguram,[...]. Nature 2019
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Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage.
Nicole M Gaudelli, Alexis C Komor, Holly A Rees, Michael S Packer, Ahmed H Badran, David I Bryson, David R Liu. Nature 2017
Nicole M Gaudelli, Alexis C Komor, Holly A Rees, Michael S Packer, Ahmed H Badran, David I Bryson, David R Liu. Nature 2017
5
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Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye.
Gerard A Rodrigues, Evgenyi Shalaev, Thomas K Karami, James Cunningham, Nigel K H Slater, Hongwen M Rivers. Pharm Res 2018
Gerard A Rodrigues, Evgenyi Shalaev, Thomas K Karami, James Cunningham, Nigel K H Slater, Hongwen M Rivers. Pharm Res 2018
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AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial.
Peter A LeWitt, Ali R Rezai, Maureen A Leehey, Steven G Ojemann, Alice W Flaherty, Emad N Eskandar, Sandra K Kostyk, Karen Thomas, Atom Sarkar, Mustafa S Siddiqui,[...]. Lancet Neurol 2011
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Gene therapy for neurological disorders: progress and prospects.
Benjamin E Deverman, Bernard M Ravina, Krystof S Bankiewicz, Steven M Paul, Dinah W Y Sah. Nat Rev Drug Discov 2018
Benjamin E Deverman, Bernard M Ravina, Krystof S Bankiewicz, Steven M Paul, Dinah W Y Sah. Nat Rev Drug Discov 2018
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AAV-Mediated Gene Therapy for Research and Therapeutic Purposes.
R Jude Samulski, Nicholas Muzyczka. Annu Rev Virol 2014
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Viral and nonviral delivery systems for gene delivery.
Nouri Nayerossadat, Talebi Maedeh, Palizban Abas Ali. Adv Biomed Res 2012
Nouri Nayerossadat, Talebi Maedeh, Palizban Abas Ali. Adv Biomed Res 2012
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Overcoming preexisting humoral immunity to AAV using capsid decoys.
Federico Mingozzi, Xavier M Anguela, Giulia Pavani, Yifeng Chen, Robert J Davidson, Daniel J Hui, Mustafa Yazicioglu, Liron Elkouby, Christian J Hinderer, Armida Faella,[...]. Sci Transl Med 2013
Federico Mingozzi, Xavier M Anguela, Giulia Pavani, Yifeng Chen, Robert J Davidson, Daniel J Hui, Mustafa Yazicioglu, Liron Elkouby, Christian J Hinderer, Armida Faella,[...]. Sci Transl Med 2013
5
CD8(+) T-cell responses to adeno-associated virus capsid in humans.
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,[...]. Nat Med 2007
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5
Co-cited is the co-citation frequency, indicating how many articles cite the article together with the query article. Similarity is the co-citation as percentage of the times cited of the query article or the article in the search results, whichever is the lowest. These numbers are calculated for the last 100 citations when articles are cited more than 100 times.