Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

Yin, Hao, Song, Chun-Qing, Dorkin, Joseph R, Zhu, Lihua J, Li, Yingxiang, Wu, Qiongqiong, Park, Angela, Yang, Junghoon +14 more·2016·Nature Biotechnology

DOI 10.1038/nbt.3471

Cited by (874)

Items 101–200 of 874

Application of CRISPR/Cas9-Based Gene Editing in HIV-1/AIDS Therapy
Qiaoqiao Xiao, Deyin Guo, Shuliang Chen · 2019 · Frontiers in Cellular and Infection Microbiology
Application of prime editing to the correction of mutations and phenotypes in adult mice with liver and eye diseases
Hyewon Jang, Dong Hyun Jo, Chang Sik Cho, Jeong Hong Shin, Jung Hwa Seo, Goosang Yu, Ramu Gopalappa, Daesik Kim, Sung‐Rae Cho, Jeong Hun Kim et al. · 2021 · Nature Biomedical Engineering
Biodegradable Polymers for Gene-Delivery Applications
Chih‐Kuang Chen, Ping-Kuan Huang, Wing‐Cheung Law, Chia-Hui Chu, Nai‐Tzu Chen, Leu‐Wei Lo · 2020 · International Journal of Nanomedicine
An engineered exosome for delivering sgRNA:Cas9 ribonucleoprotein complex and genome editing in recipient cells
Yangyang Ye, Xiang Zhang, Fei Xie, Bin Xu, Ping Xie, Ting Yang, Qian Shi, Chen‐Yu Zhang, Yujing Zhang, Jiangning Chen et al. · 2020 · Biomaterials Science
CRISPR-Cas9-mediated homology-directed repair for precise gene editing
Hongyu Liao, Jiahao Wu, Nathan J. VanDusen, Yifei Li, Yanjiang Zheng · 2024 · Molecular Therapy — Nucleic Acids
Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy
Mohammed Fatih Rasul, Bashdar Mahmud Hussen, Abbas Burhan Salihi, Bnar Saleh Ismael, Paywast Jamal Jalal, Anna Zanichelli, Elena Jamali, Aria Baniahmad, Soudeh Ghafouri‐Fard, Abbas Basiri et al. · 2022 · Molecular Cancer
CRISPR/Cas9-mediated genome editing induces exon skipping by alternative splicing or exon deletion
Haiwei Mou, Jordan L. Smith, Lingtao Peng, Hao Yin, Jill E. Moore, Xiao-Ou Zhang, Chun‐Qing Song, Ankur Sheel, Qiongqiong Wu, Deniz Mahmut Özata et al. · 2017 · Genome biology
CRISPR–Cas: a tool for cancer research and therapeutics
Hao Yin, Wen Xue, Daniel G. Anderson · 2019 · Nature Reviews Clinical Oncology
Gene Editing on Center Stage
Rasmus Otkjær Bak, Natalia Gomez‐Ospina, Matthew Hebden Porteus · 2018 · Trends in Genetics
Nanoscale platforms for messenger RNA delivery
Li, Bin, Zhang, Xinfu, Dong, Yizhou · 2019 · WIREs Nanomedicine and Nanobiotechnology
In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease
Lau, Cia-Hin, Suh, Yousin · 2017 · F1000Research
In Situ Gene Therapy via AAV-CRISPR-Cas9-Mediated Targeted Gene Regulation
Moreno, Ana M., Fu, Xin, Zhu, Jie, Katrekar, Dhruva, Shih, Yu-Ru V., Marlett, John, Cabotaje, Jessica, Tat, Jasmine, Naughton, John, Lisowski, Leszek et al. · 2018 · Molecular Therapy
Biomaterial-assisted biotherapy: A brief review of biomaterials used in drug delivery, vaccine development, gene therapy, and stem cell therapy
Xuejiao Han, Aqu Alu, Hongmei Liu, Yi Shi, Xiawei Wei, Lulu Cai, Yuquan Wei · 2022 · Bioactive Materials
Delivery approaches for CRISPR/Cas9 therapeutics in vivo: advances and challenges
David C. Luther, Y.W. Lee, Harini Nagaraj, Federica Scaletti, Vincent M. Rotello · 2018 · Expert Opinion on Drug Delivery
Breaking the final barrier: Evolution of cationic and ionizable lipid structure in lipid nanoparticles to escape the endosome
Kaitlin Mrksich, Marshall Scott Padilla, Michael J. Mitchell · 2024 · Advanced Drug Delivery Reviews
Delivering the right message: Challenges and opportunities in lipid nanoparticles-mediated modified mRNA therapeutics—An innate immune system standpoint
Yasmin Granot-Matok, Dan Peer · 2017 · Seminars in Immunology
Murine liver repair via transient activation of regenerative pathways in hepatocytes using lipid nanoparticle-complexed nucleoside-modified mRNA
Fatima Rizvi, Elissa Everton, Anna R. Smith, Hua Liu, Elizabeth Osota, Mitchell B. Beattie, Ying K. Tam, Norbert Pardi, Drew Weissman, Valerie Gouon–Evans · 2021 · Nature Communications
Spatial control of in vivo CRISPR–Cas9 genome editing via nanomagnets
Haibao Zhu, Linlin Zhang, Sheng Tong, Ciaran M. Lee, Harshavardhan Deshmukh, Gang Bao · 2018 · Nature Biomedical Engineering
Programmable base editing of mutated TERT promoter inhibits brain tumour growth
Xinjian Li, Xu Qian, Bin Wang, Yan Xia, Yanhua Zheng, Linyong Du, Daqian Xu, Dongming Xing, Ronald Anthony DePinho, Zhimin Lu · 2020 · Nature Cell Biology
Graphene oxide-mediated Cas9/sgRNA delivery for efficient genome editing
Yue, Huahua, Zhou, Xiaoming, Cheng, Meng, Xing, Da · 2018 · Nanoscale
Genome-wide profiling of adenine base editor specificity by EndoV-seq
Liang, Puping, Xie, Xiaowei, Zhi, Shengyao, Sun, Hongwei, Zhang, Xiya, Chen, Yu, Chen, Yuxi, Xiong, Yuanyan, Ma, Wenbin, Liu, Dan et al. · 2019 · Nature Communications
Delivery of CRISPR/Cas9 for therapeutic genome editing
Xiaojie Xu, Tao Yu Wan, Huhu Xin, Da Li, Hongming Pan, Jun Wu, Ping Yuan · 2019 · The Journal of Gene Medicine
A Potent Branched-Tail Lipid Nanoparticle Enables Multiplexed mRNA Delivery and Gene Editing In Vivo
Khalid A. Hajj, Jilian R. Melamed, Namit Chaudhary, Nicholas G. Lamson, Rebecca L. Ball, Saigopalakrishna S. Yerneni, Kathryn A. Whitehead · 2020 · Nano Letters
CRISPR-Cas9 Targeting of PCSK9 in Human Hepatocytes In Vivo—Brief Report
Xiao Wang, Avanthi Raghavan, Tao Chen, Lyon Qiao, Yongxian Zhang, Qiurong Ding, Kiran Musunuru · 2016 · Arteriosclerosis Thrombosis and Vascular Biology
Immunity to CRISPR Cas9 and Cas12a therapeutics
Wei Leong Chew · 2017 · WIREs Systems Biology and Medicine
A Proton‐Activatable DNA‐Based Nanosystem Enables Co‐Delivery of CRISPR/Cas9 and DNAzyme for Combined Gene Therapy
Feng Li, Nachuan Song, Yuhang Dong, Shuai Li, Ling‐Hui Li, Yujie Liu, Zhemian Li, Dayong Yang · 2022 · Angewandte Chemie International Edition
Immunogenicity of CRISPR therapeutics—Critical considerations for clinical translation
Radwa Ewaisha, Karen S. Anderson · 2023 · Frontiers in Bioengineering and Biotechnology
CRISPR-Cas9 for in vivo Gene Therapy: Promise and Hurdles
Wei-Jing Dai, Liyao Zhu, Zhongyi Yan, Yong Xu, Qilong Wang, Xiaojie Lu · 2016 · Molecular Therapy — Nucleic Acids
Synthetically modified guide RNA and donor DNA are a versatile platform for CRISPR-Cas9 engineering
Kunwoo Lee, Vanessa A Mackley, Anirudh Rao, Anthony T. Chong, Mark A. DeWitt, Jacob E. Corn, Niren Murthy · 2017 · eLife
Non-viral delivery of the CRISPR/Cas system: DNA versus RNA versus RNP
Yi Lin, Ernst Wagner, Ulrich Lächelt · 2022 · Biomaterials Science
Throughput-scalable manufacturing of SARS-CoV-2 mRNA lipid nanoparticle vaccines
Sarah J. Shepherd, Xuexiang Han, Alvin J. Mukalel, Rakan D. El-Mayta, Ajay S. Thatte, Jingyu Wu, Marshall Scott Padilla, Mohamad‐Gabriel Alameh, Neha Srikumar, Daeyeon Lee et al. · 2023 · Proceedings of the National Academy of Sciences
Nanoparticles for nucleic acid delivery: Applications in cancer immunotherapy
Alvin J. Mukalel, Rachel Riley, Rui Zhang, Michael J. Mitchell · 2019 · Cancer Letters
A Self-Assembled Platform Based on Branched DNA for sgRNA/Cas9/Antisense Delivery
Jianbing Liu, Tiantian Wu, Xuehe Lu, Xiaohui Wu, Shaoli Liu, Shuai Zhao, Xuehui Xu, Baoquan Ding · 2019 · Journal of the American Chemical Society
Ionizable Amino‐Polyesters Synthesized via Ring Opening Polymerization of Tertiary Amino‐Alcohols for Tissue Selective mRNA Delivery
Piotr S. Kowalski, Umberto Capasso Palmiero, Yuxuan Huang, Arnab Rudra, Róbert Langer, Daniel G. Anderson · 2018 · Advanced Materials
Long-lasting analgesia via targeted in situ repression of Na V 1.7 in mice
Ana M. Moreno, Fernando Alemán, Glaucilene Ferreira Catroli, Matthew A. Hunt, Michael Y. Hu, Amir Dailamy, Andrew Pla, Sarah A. Woller, Nathan D. Palmer, Udit Parekh et al. · 2021 · Science Translational Medicine
Advances in CRISPR/Cas-based Gene Therapy in Human Genetic Diseases
Shaoshuai Wu, Qing-Cui Li, Changqing Yin, Wen Xue, Chun‐Qing Song · 2020 · Theranostics
Delivery of Tissue-Targeted Scalpels: Opportunities and Challenges for In Vivo CRISPR/Cas-Based Genome Editing
Tuo Wei, Qiang Cheng, Lukas Farbiak, Daniel G. Anderson, Róbert Langer, Daniel J. Siegwart · 2020 · ACS Nano
Peptide-encoding mRNA barcodes for the high-throughput in vivo screening of libraries of lipid nanoparticles for mRNA delivery
Luke H. Rhym, Rajith S. Manan, Antonius A. Koller, Georgina Stephanie, Daniel G. Anderson · 2023 · Nature Biomedical Engineering
Activating PTEN Tumor Suppressor Expression with the CRISPR/dCas9 System
Colette Moses, Fiona Nugent, Charlene Babra Waryah, Benjamín García‐Bloj, Alan R. Harvey, Pilar Blancafort · 2018 · Molecular Therapy — Nucleic Acids
Chitosan in Non-Viral Gene Delivery: Role of Structure, Characterization Methods, and Insights in Cancer and Rare Diseases Therapies
Beatriz Santos-Carballal, Elena Fernández Fernández, Francisco M. Goycoolea · 2018 · Polymers
Nanoplatforms for mRNA Therapeutics
Meng, Chaoyang, Chen, Zhe, Li, Gang, Welte, Thomas, Shen, Haifa · 2021 · Advanced Therapeutics
Responsive Nanocarriers as an Emerging Platform for Cascaded Delivery of Nucleic Acids to Cancer
Yang Liu, Cong‐Fei Xu, Shoaib Iqbal, Xianzhu Yang, Jun Wang · 2017 · Advanced Drug Delivery Reviews
CRISPR/Cas9‐mediated genome editing: From basic research to translational medicine
Filipe V. Jacinto, Wolfgang Link, Bibiana I. Ferreira · 2020 · Journal of Cellular and Molecular Medicine
Clinical applications of the CRISPR/Cas9 genome-editing system: Delivery options and challenges in precision medicine
Mohadeseh Khoshandam, Hossein Soltaninejad, Marziyeh Mousazadeh, Amir Ali Hamidieh, Saman Hosseinkhani · 2023 · Genes & Diseases
Novel Drug Delivery Systems: An Important Direction for Drug Innovation Research and Development
Qian Chen, Zhen Yang, Haoyu Liu, Jingyuan Man, Ayodele Olaolu Oladejo, Sally Ibrahim, Shengyi Wang, Baocheng Hao · 2024 · Pharmaceutics
Synthetic genetic circuits for programmable biological functionalities
Peng‐Fei Xia, Hua Ling, Jee Loon Foo, Matthew Wook Chang · 2019 · Biotechnology Advances
Synthetically controlling dendrimer flexibility improves delivery of large plasmid DNA
Jessica A. Kretzmann, Diwei Ho, Cameron W. Evans, Janice H. C. Plani-Lam, Benjamín García‐Bloj, A. Elaaf Mohamed, Megan L. O’Mara, Ethan Ford, Dennis Eng Kiat Tan, Ryan Lister et al. · 2017 · Chemical Science
CRISPR/Cas9 for Cancer Therapy: Hopes and Challenges
Marta Martínez-Lage, Pilar Puig-Serra, Pablo R. Menéndez, Raúl Torres-Ruiz, Sandra Rodríguez · 2018 · Biomedicines
Scaffold-mediated CRISPR-Cas9 delivery system for acute myeloid leukemia therapy
Tzu‐Chieh Ho, Hye Sung Kim, Yumei Chen, Yamin Li, Mark W. LaMere, Caroline Chen, Hui Wang, Jing Gong, Cal D. Palumbo, John M. Ashton et al. · 2021 · Science Advances
Biomedical applications of mRNA nanomedicine
Xiong, Qingqing, Lee, Gha Young, Ding, Jianxun, Li, Wenliang, Shi, Jinjun · 2018 · Nano Research
The NIH Somatic Cell Genome Editing program
Krishanu Saha, Erik J. Sontheimer, Philip John Brooks, Melinda R. Dwinell, Charles A. Gersbach, David R. Liu, Stephen A. Murray, Shengdar Q. Tsai, Ross C. Wilson, Daniel G. Anderson et al. · 2021 · Nature
Multiplexed RNAi therapy against brain tumor-initiating cells via lipopolymeric nanoparticle infusion delays glioblastoma progression
Dou Yu, Omar F. Khan, Mario L. Suvà, Biqin Dong, Wojciech K. Panek, Ting Xiao, Meijing Wu, Yu Han, Atique U. Ahmed, Irina V. Balyasnikova et al. · 2017 · Proceedings of the National Academy of Sciences
Genome-Wide CRISPR Screen Identifies Regulators of Mitogen-Activated Protein Kinase as Suppressors of Liver Tumors in Mice
Chun‐Qing Song, Yingxiang Li, Haiwei Mou, Jill E. Moore, Angela Park, Yotsawat Pomyen, Soren H. Hough, Zachary Kennedy, Andrew H. Fischer, Hao Yin et al. · 2016 · Gastroenterology
Therapeutic gene editing: delivery and regulatory perspectives
Gayong Shim, Dongyoon Kim, Gyu Thae Park, Hyerim Jin, Soo‐Kyung Suh, Yu‐Kyoung Oh · 2017 · Acta Pharmacologica Sinica
mRNA therapeutics deliver a hopeful message
Zifu Zhong, Séan Mc Cafferty, Francis Combes, Hanne Huysmans, Joyca De Temmerman, Arlieke Gitsels, Daisy C.G. Vanrompay, João Portela Catani, Niek N. Sanders · 2018 · Nano Today
Applications of CRISPR-Cas Enzymes in Cancer Therapeutics and Detection
Chun‐Hao Huang, Ko-Chuan Lee, Jennifer A. Doudna · 2018 · Trends in cancer
Robotics, microfluidics, nanotechnology and AI in the synthesis and evaluation of liposomes and polymeric drug delivery systems
Egor Egorov, Calvin Pieters, Hila Korach-Rechtman, Jeny Shklover, Avi Schroeder · 2021 · Drug Delivery and Translational Research
Genome editing by natural and engineered CRISPR-associated nucleases
Wen Y. Wu, Joyce H.G. Lebbink, Roland Kanaar, Niels Geijsen, John van der OOST · 2018 · Nature Chemical Biology
CRISPR-Mediated Integration of Large Gene Cassettes Using AAV Donor Vectors
Rasmus Otkjær Bak, Matthew Hebden Porteus · 2017 · Cell Reports
CRISPR/Cas9 Genome-Editing System in Human Stem Cells: Current Status and Future Prospects
Zhao Zhang, Yuelin Zhang, Fei Gao, Shuo Han, Kathryn S.E. Cheah, Hung‐Fat Tse, Qizhou Lian · 2017 · Molecular Therapy — Nucleic Acids
In vivo genome editing via the HITI method as a tool for gene therapy
Keiichiro Suzuki, Juan Carlos Izpisúa Belmonte · 2017 · Journal of Human Genetics
Non-Viral Delivery To Enable Genome Editing
Yuan Rui, David R. Wilson, Jordan J. Green · 2018 · Trends in biotechnology
Zinc Fingers, TALEs, and CRISPR Systems: A Comparison of Tools for Epigenome Editing
Charlene Babra Waryah, Colette Moses, Mahira Arooj, Pilar Blancafort · 2018 · Methods in molecular biology
CRISPR-Cas12a delivery by DNA-mediated bioresponsive editing for cholesterol regulation
Wujin Sun, Jinqiang Wang, Quanyin Hu, Xingwu Zhou, Ali Khademhosseini, Zhen Gu · 2020 · Science Advances
Human genetic variation alters CRISPR-Cas9 on- and off-targeting specificity at therapeutically implicated loci
Samuel Lessard, Laurent C. Francioli, Jessica Alföldi, Jean‐Claude Tardif, Patrick T. Ellinor, Daniel G. MacArthur, Guillaume Lettre, Stuart H. Orkin, Matthew C. Canver · 2017 · Proceedings of the National Academy of Sciences
Therapeutic genome editing of triple-negative breast tumors using a noncationic and deformable nanolipogel
Peng Guo, Jiang Yang, Jing Huang, Debra T. Auguste, Marsha A. Moses · 2019 · Proceedings of the National Academy of Sciences
Non-viral delivery of genome-editing nucleases for gene therapy
M Wang, Zachary Glass, Qiaobing Xu · 2016 · Gene Therapy
Systemic delivery of CRISPR/Cas9 with PEG-PLGA nanoparticles for chronic myeloid leukemia targeted therapy
Yang Liu, Gui Zhao, Cong‐Fei Xu, Ying‐Li Luo, Zi-Dong Lu, Jun Wang · 2018 · Biomaterials Science
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo
Raed Ibraheim, Chun‐Qing Song, Aamir Mir, Nadia Amrani, Wen Xue, Erik J. Sontheimer · 2018 · Genome biology
Sharpening the Molecular Scissors: Advances in Gene-Editing Technology
Mike Broeders, Pablo Herrero-Hernandez, Martijn P. T. Ernst, Ans T. van der Ploeg, W.W.M. Pim Pijnappel · 2019 · iScience
Chemical modifications of adenine base editor mRNA and guide RNA expand its application scope
Tingting Jiang, Jordana M. Henderson, Kevin J. Coote, Yi Cheng, Hillary C. Valley, Xiao-Ou Zhang, Qin Wang, Luke H. Rhym, Yueying Cao, Gregory A. Newby et al. · 2020 · Nature Communications
Cell-type-specific genome editing with a microRNA-responsive CRISPR–Cas9 switch
Moe Hirosawa, Yoshihiko Fujita, Callum Parr, Karin Hayashi, Shunnichi Kashida, Akitsu Hotta, Knut Woltjen, Hirohide Saito · 2017 · Nucleic Acids Research
Recent advances in lipid nanoparticles for delivery of nucleic acid, mRNA, and gene editing-based therapeutics
Hidefumi Mukai, Koki Ogawa, Naoya Kato, Shigeru Kawakami · 2022 · Drug Metabolism and Pharmacokinetics
Biodegradable Amino-Ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo
Zhang, Xinfu, Li, Bin, Luo, Xiao, Zhao, Weiyu, Jiang, Justin, Zhang, Chengxiang, Gao, Min, Chen, Xiaofang, Dong, Yizhou · 2017 · ACS Applied Materials & Interfaces
Advancements and Obstacles of CRISPR-Cas9 Technology in Translational Research
Liting You, Ruizhan Tong, Mengqian Li, Yuncong Liu, Jianxin Xue, You Lü · 2019 · Molecular Therapy — Methods & Clinical Development
Friend or Foe? Evidence Indicates Endogenous Exosomes Can Deliver Functional gRNA and Cas9 Protein
Ran Chen, Hongxin Huang, Hui Liu, Jingyuan Xi, Jing Ning, Wanjia Zeng, Congle Shen, Ting Zhang, Guangxin Yu, Qiang Xu et al. · 2019 · Small
CRISPR/Cas9 for Human Genome Engineering and Disease Research
Xin Xiong, Meng Chen, Wendell A. Lim, Dehua Zhao, Lei S. Qi · 2016 · Annual Review of Genomics and Human Genetics
CRISPR technologies for stem cell engineering and regenerative medicine
Mu‐Nung Hsu, Yu‐Han Chang, Vu Anh Truong, Po‐Liang Lai, Thị Kieu Nuong Nguyen, Yu‐Chen Hu · 2019 · Biotechnology Advances
In vivo targeted delivery of nucleic acids and CRISPR genome editors enabled by GSH-responsive silica nanoparticles
Yuyuan Wang, Pawan Kumar Shahi, Xiuxiu Wang, Ruosen Xie, Yi Zhao, Min Wu, Seth Roge, Bikash Ranjan Pattnaik, Shaoqin Gong · 2021 · Journal of Controlled Release
Carrier strategies boost the application of CRISPR/Cas system in gene therapy
Zunkai Xu, Qingnan Wang, Haiping Zhong, Yaoyao Jiang, Xiaoguang Shi, Bo Yuan, Na Yu, Shubiao Zhang, Xiaoyong Yuan, Shutao Guo et al. · 2022 · Exploration
Liver Injury and Regeneration: Current Understanding, New Approaches, and Future Perspectives
Shainan Hora, Torsten Wüestefeld · 2023 · Cells
Increased and prolonged human norovirus infection in RAG2/IL2RG deficient gnotobiotic pigs with severe combined immunodeficiency
Shaohua Lei, Junghyun Ryu, Ke Wen, Erica L. Twitchell, Tammy Bui, Ashwin Kumar Ramesh, Mariah Weiss, Guohua Li, Helen Samuel, Sherrie Clark‐Deener et al. · 2016 · Scientific Reports
Nanostructured Materials for Intracellular Cargo Delivery
Andy Kah Ping Tay, Nicholas A. Melosh · 2019 · Accounts of Chemical Research
Ultrasound-Controlled CRISPR/Cas9 System Augments Sonodynamic Therapy of Hepatocellular Carcinoma
Haohao Yin, Liping Sun, Yinying Pu, Jifeng Yu, Wei Feng, Caihong Dong, Bangguo Zhou, Dou Du, Yan Zhang, Yu Chen et al. · 2021 · ACS Central Science
CRISPR-Cas9–mediated therapeutic editing of Rpe65 ameliorates the disease phenotypes in a mouse model of Leber congenital amaurosis
Dong Hyun Jo, Dong Woo Song, Chang Sik Cho, Un Gi Kim, Kyu Jun Lee, Kihwang Lee, Sung Wook Park, Jin‐Soo Kim, Jin Hyoung Kim, Jin‐Soo Kim et al. · 2019 · Science Advances
All‐In‐One Dendrimer‐Based Lipid Nanoparticles Enable Precise HDR‐Mediated Gene Editing In Vivo
Lukas Farbiak, Qiang Cheng, Tuo Wei, Ester Álvarez‐Benedicto, Lindsay T. Johnson, Sang M. Lee, Daniel J. Siegwart · 2021 · Advanced Materials
Messenger RNA Delivery for Tissue Engineering and Regenerative Medicine Applications
Siddharth Patel, Avathamsa Athirasala, Paula dos Passos Menezes, N. Ashwanikumar, Ting Zou, Gaurav Sahay, Luiz E. Bertassoni · 2018 · Tissue Engineering Part A
Lipid Nanoparticles for Delivery of Therapeutic RNA Oligonucleotides
Keith Henry Moss, Petya G. Popova, Sine Reker Hadrup, Kira Astakhova, Maria Taskova · 2019 · Molecular Pharmaceutics
Co-encapsulation of Cas9 mRNA and guide RNA in polyplex micelles enables genome editing in mouse brain
Saed Abbasi, Satoshi Uchida, Kazuko Toh, Theofilus A. Tockary, Anjaneyulu Dirisala, Kotaro Hayashi, Shigeto Fukushima, Kazunori Kataoka · 2021 · Journal of Controlled Release
Somatic genome editing with CRISPR/Cas9 generates and corrects a metabolic disease
Kelsey E. Jarrett, Ciaran M. Lee, Yi-Hsien Yeh, Rachel H. Hsu, Rajat Gupta, Min Zhang, Perla J. Rodriguez, Chang Seok Lee, Baiba Kurins Gillard, Karl‐Dimiter Bissig et al. · 2017 · Scientific Reports
In Vivo Delivery Systems for Therapeutic Genome Editing
Luyao Wang, Fangfei Li, Lei Dang, Chao Liang, Chao Wang, Bing He, Jin Liu, Defang Li, Xiaohao Wu, Xuegong Xu et al. · 2016 · International Journal of Molecular Sciences
Cell-Selective Messenger RNA Delivery and CRISPR/Cas9 Genome Editing by Modulating the Interface of Phenylboronic Acid-Derived Lipid Nanoparticles and Cellular Surface Sialic Acid
Qiao Tang, Ji Liu, Ying Yan Jiang, Meining Zhang, Lanqun Mao, Ming Wang · 2019 · ACS Applied Materials & Interfaces
CRISPR-Cas9 therapeutics in cancer: promising strategies and present challenges
Lang Yi, Jinming Li · 2016 · Biochimica et Biophysica Acta (BBA) - Reviews on Cancer
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
Sumitava Dastidar, Simon Ardui, Kshitiz Singh, Debanjana Majumdar, Nisha Nair, Yanfang Fu, Deepak Reyon, Ermira Samara-Kuko, M Gerli, Arnaud F. Klein et al. · 2018 · Nucleic Acids Research
In situ repurposing of dendritic cells with CRISPR/Cas9-based nanomedicine to induce transplant tolerance
Yue Zhang, Song Shen, Gui Zhao, Cong‐Fei Xu, Houbing Zhang, Ying‐Li Luo, Zhiting Cao, Jia Yu Shi, Zhibin Zhao, Zhe‐Xiong Lian et al. · 2019 · Biomaterials
Recent advances in chemical modifications of guide RNA, mRNA and donor template for CRISPR-mediated genome editing
Qiubing Chen, Ying Zhang, Hao Yin · 2020 · Advanced Drug Delivery Reviews
Non-viral Delivery of Zinc Finger Nuclease mRNA Enables Highly Efficient In Vivo Genome Editing of Multiple Therapeutic Gene Targets
Conway, Anthony, Mendel, Matthew, Kim, Kenneth, McGovern, Kyle, Boyko, Alisa, Zhang, Lei, Miller, Jeffrey C., DeKelver, Russell C., Paschon, David E., Mui, Barbara L. et al. · 2019 · Molecular Therapy
History of Polymeric Gene Delivery Systems
Peng Zhang, Ernst Wagner · 2017 · Topics in Current Chemistry
HPV Oncogene Manipulation Using Nonvirally Delivered CRISPR/Cas9 or Natronobacterium gregoryi Argonaute
Yeh‐Hsing Lao, Mingqiang Li, Madeleine A. Gao, Dan Shao, Chun‐Wei Chi, Dantong Huang, Syandan Chakraborty, Tzu‐Chieh Ho, Weiqian Jiang, Hongxia Wang et al. · 2018 · Advanced Science
Multiplexed in vivo homology-directed repair and tumor barcoding enables parallel quantification of Kras variant oncogenicity
Ian P. Winters, Shin‐Heng Chiou, Nicole K. Paulk, Christopher D. McFarland, Pranav V. Lalgudi, Rosanna K. Ma, Leszek Lisowski, Andrew J. Connolly, Dmitri A. Petrov, Mark A. Kay et al. · 2017 · Nature Communications