A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

Yang, Yang, Wang, Lili, Bell, Peter, McMenamin, Deirdre, He, Zhenning, White, John, Yu, Hongwei, Xu, Chenyu, Morizono, Hiroki, Musunuru, Kiran, Batshaw, Mark L, Wilson, James M·2016·Nature Biotechnology

DOI 10.1038/nbt.3469

Cited by (564)

Items 301–400 of 564

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Alice Rovai, Bomee Chung, Qingluan Hu, Sebastian Hook, Qinggong Yuan, Tibor Kempf, Florian Schmidt, Dirk Grimm, Steven Roger Talbot, Lars Steinbrück et al. · 2022 · Nature Communications
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Yoshiki Higashijima, Seiichi Hirano, Masaomi Nangaku, Osamu Nureki · 2017 · Kidney International
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Colin T. Konishi, Chengzu Long · 2021 · Journal of Biomedical Research
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Jessica L. Schneller, Ciaran M. Lee, Leah E. Venturoni, Randy J. Chandler, Ang Li, Sangho Myung, Thomas James Cradick, Ayrea E. Hurley, William R. Lagor, Gang Bao et al. · 2021 · Molecular Therapy — Methods & Clinical Development
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GARY J. GIBSON, Maozhou Yang · 2017 · Nature Reviews Rheumatology
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Benjamin E. Epstein, David V. Schaffer · 2017 · Advances in experimental medicine and biology
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Romain Rouet, Daniel Christ · 2019 · ACS Chemical Biology
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Xiaozhen Liu, Jing Qiao, Ruixuan Jia, Fan Zhang, Xiang Meng, Yang Li, Liping Yang · 2023 · eLife
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Kobra Zakikhan, Behshad Pournasr, Vosough Massoud, Nassiri Asl Marjan · 2017 · PubMed Central
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Xiang Ling Meng, Tiangang Wu, Qiuyue Lou, Kaiyuan Niu, Lei Jiang, Qing‐zhong Xiao, Tao Xu, Lei Zhang · 2022 · Bioengineering & Translational Medicine
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James E. DiCarlo, Anurag Deeconda, Stephen H. Tsang · 2017 · Advances in experimental medicine and biology
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Rajagopal N. Aravalli, Clifford J. Steer · 2017 · Journal of Cellular Biochemistry
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Guodong Wang, Qi Li, Junjie Xu, Shuai Zhao, Rui Zhou, Zhenkang Chen, Wentong Jiang, Xue Gao, Shuang Zhou, Zhiyu Chen et al. · 2022 · Journal of Neuroscience
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Kshitiz Singh, Raffaele Fronza, Hanneke Evens, Marinee Chuah, Thierry VandenDriessche · 2024 · Molecular Therapy — Methods & Clinical Development
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Marta Trevisan, Giulia Masi, Giorgio Palù · 2020 · Translational Gastroenterology and Hepatology
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Rachel L. Mintz, John D. Loike, Ruth L. Fischbach · 2018 · Science and Engineering Ethics
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Rosemary C. Challis, Sripriya Ravindra Kumar, Ken Y. Chan, Collin Challis, Min Jee Jang, Pradeep Sundaram Rajendran, John D. Tompkins, Kalyanam Shivkumar, Benjamin E. Deverman, Viviana Gradinaru · 2018 · bioRxiv (Cold Spring Harbor Laboratory)
CRISPR Technology in Disease Management: An Updated Review of Clinical Translation and Therapeutic Potential
Bahareh Farasati Far, Marziyeh Akbari, Mohammad Amin Habibi, Morteza Katavand, Sherko Nasseri · 2025 · Cell Proliferation
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Tomoko Kato, Shuji Takada · 2016 · Briefings in Functional Genomics
CRISPR/dCas9‐mediated transposition with specificity and efficiency of site‐directed genomic insertions
Lena Goshayeshi, Sara Yousefi Taemeh, Nima Dehdilani, M Nasiri, Mohammad Mahdi Ghahramani Seno, Hesam Dehghani · 2021 · The FASEB Journal
Duchenne Muscular Dystrophy Gene Therapy
Fawzy Ali Saad, Jasen F. Saad, Gabriele Siciliano, Luciano Merlini, Corrado Italo Angelini · 2022 · Current Gene Therapy
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JANICE YANG CHOU, Brian C. Mansfield · 2023 · Frontiers in Molecular Medicine
Precision in Action: The Role of Clustered Regularly Interspaced Short Palindromic Repeats/Cas in Gene Therapies
Amrutha Banda, Olivia Impomeni, Aparana Singh, Abdul Rasheed Baloch, Wenhui Hu, Dabbu Kumar Jaijyan · 2024 · Vaccines
Gene Therapy Overexpressing Neuregulin 1 Type I in Combination With Neuregulin 1 Type III Promotes Functional Improvement in the SOD1G93A ALS Mice
Guillem Mòdol‐Caballero, Mireia Herrando‐Grabulosa, Sergi Verdés, Belén García-Lareu, Neus Hernández, Isaac Francos-Quijorna, Rubén López‐Vales, Assumpció Bosch, Xavier Navarro · 2021 · Frontiers in Neurology
Liver-directed gene-based therapies for inborn errors of metabolism
Pasquale Piccolo, Alessandro Rossi, Nicola Brunetti‐Pierri · 2020 · Expert Opinion on Biological Therapy
CrisPR/CRIS systems are highly effective and useful for genomic manipulation. Despite this, cardiac treatment remains difficult due to existing genome editing and delivery processes.
Moataz Dowaidar · 2021 · OSF Preprints (OSF Preprints)
Therapeutic genome editing with engineered nucleases
Simone Alexandra Haas, Viviane Dettmer‐Monaco, Toni Cathomen · 2017 · Hämostaseologie
CRISPR-Cas Genome Surgery in Ophthalmology
James E. DiCarlo, Jesse D. Sengillo, Sally Justus, Thiago Cabral, Stephen H. Tsang, Vinit B. Mahajan · 2017 · Translational Vision Science & Technology
In vivo application of base and prime editing to treat inherited retinal diseases
Dong Hyun Jo, Sangsu Bae, Seokjoong Kim, Jin‐Soo Kim, Jeong Hun Kim · 2022 · Progress in Retinal and Eye Research
Targeting multiple myeloma with nanobody-based heavy chain antibodies, bispecific killer cell engagers, chimeric antigen receptors, and nanobody-displaying AAV vectors
Julia Hambach, Anna Marei Mann, Peter Bannas, Friedrich Koch‐Nolte · 2022 · Frontiers in Immunology
Site-specific genome editing in treatment of inherited diseases: possibility, progress, and perspectives
Chao Huang, Qing Li, Jinsong Li · 2022 · Medical Review
Emerging and potential use of CRISPR in human liver disease
Salah Adlat, Alexandra M. Vázquez-Salgado, Markcus Lee, Dingzi Yin, Kirk J. Wangensteen · 2023 · Hepatology
Adeno-Associated Virus Serotype 8-Mediated Genetic Labeling of Cholangiocytes in the Neonatal Murine Liver
Sanghoon Lee, Ping Zhou, Senyo Whyte, Soona Shin · 2020 · Pharmaceutics
Use of Customizable Nucleases for Gene Editing and Other Novel Applications
Pradeep Reddy, Felipe Vilella, Juan Carlos Izpisúa Belmonte, Carlos Simón · 2020 · Genes
CRISPR/Cas9-mediated correction of MITF homozygous point mutation in a Waardenburg syndrome 2A pig model
Jing Tao Yao, Yu Wang, Chunwei Cao, Ruigao Song, Dengfeng Bi, Hongyong Zhang, Yongshun Li, Guosong Qin, Naipeng Hou, Jin Zhang et al. · 2021 · Molecular Therapy — Nucleic Acids
rAAV capsid mutants eliminate leaky expression from DNA donor template for homologous recombination
Chen Ling, Chenghui Yu, Cong Wang, Ming Yang, Hengbin Yang, Keying Yang, Yun He, Yajie Shen, Shiyi Tang, Xiaomin Yu et al. · 2024 · Nucleic Acids Research
CRISPR-Mediated Ophthalmic Genome Surgery
Galaxy Y. Cho, Yazeed L. Abdulla, Jesse D. Sengillo, Sally Justus, Kellie A. Schaefer, Alexander G. Bassuk, Stephen H. Tsang, Vinit B. Mahajan · 2017 · Current Ophthalmology Reports
Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler
Xiu Jin, Jing Su, Qinyu Zhao, Ruiting Li, Jianlu Xiao, Xiaomei Zhong, Li Song, Yi Liu, Kaiqin She, Hongxin Deng et al. · 2022 · Molecular Therapy — Methods & Clinical Development
CRISPR/Cas9: an overview of recent developments and applications in cancer research
Shamjetsabam Nandibala Devi, Rashmi Rana, Priyanka Malik, Nirmal Kumar Ganguly · 2024 · International Journal of Surgery
Engineering adeno-associated viral vectors for CRISPR/Cas based in vivo therapeutic genome editing
Buhle Moyo, Lucas B.C. Brown, I. Khondaker, Gang Bao · 2025 · Biomaterials
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Narmadhaa Siva, Sonal Gupta, Ayam Gupta, Jayendra Nath Shukla, Babita Malik, Nidhi Shukla · 2021 · 3 Biotech
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Ningning Liu, Jing Tian, Clifford J. Steer, Qinghua Han, Guisheng Song · 2023 · Hepatology
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Dilpreet Singh · 2024 · AAPS PharmSciTech · Retracted
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Marina Beraza-Millor, Julen Rodríguez-Castejón, Ana del Pozo‐Rodríguez, Alicia Rodríguez‐Gascón, Maria Ángelés Solinís · 2024 · BioDrugs
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In vivo adenine base editing corrects newborn murine model of Hurler syndrome
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Allison M. Sharrar, Luisa Arake de Tacca, Trevor N. Collingwood, Zuriah A. Meacham, David Rabuka, Johanna Staples-Ager, Michael W. Schelle · 2023 · The CRISPR Journal
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Gene editing in allergic diseases: Identification of novel pathways and impact of deleting allergen genes
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An Exon-Specific Small Nuclear U1 RNA (ExSpeU1) Improves Hepatic OTC Expression in a Splicing-Defective spf/ash Mouse Model of Ornithine Transcarbamylase Deficiency
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