Latest Publications

Publication Title Authors Journal Publication Date PubMed/Proj #
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9 Li A, Tanner MR, Lee CM, Hurley AE, De Giorgi M, Jarrett KE, Davis TH1, Doerfler AM, Bao G, Beeton C, Lagor WR Molecular Therapy 2020 Apr 19 32348718
Optimizing biodegradable nanoparticle size for tissue-specific delivery Mandl HK, Quijano E, Suh HW, Sparago E, Oeck S, Grun M, Glazer PM, Saltzman WM J Control Release 2019 Nov 28 31654688
Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia Krishnamurthy S, Wohlford-Lenane C, Kandimalla S, Sartre G, Meyerholz DK, Théberge V, Hallée S, Duperré AM, Del’Guidice T, Lepetit-Stoffaes JP, Barbeau X, Guay D & McCray Jr PB. Nature Communications 2019 Oct 28 31659165 s41467-019-12922-y
Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States Wei Sun, Jing Yang, Zhi Cheng,Nadia Amrani, Chao Liu, Kangkang Wang, Raed Ibraheim, Alireza Edraki, Xue Huang, Min Wang, Jiuyu Wang, Liang Liu, Gang Sheng, Yanhua Yang, Jizhong Lou, Erik J. Sontheimer, Yanli Wang. Molecular Cell 2019 Oct 24 31668930

Search-and-replace genome editing without double-strand breaks or donor DNA

Anzalone AV, Randolph PB, Davis JR, Sousa AA, Koblan LW, Levy JM, Chen PJ, Wilson C, Newby GA, Raguram A, Liu DR Nature 2019 Oct 21 316634902
A biodegradable nanocapsule delivers a Cas9 ribonucleoprotein complex for in vivo genome editing Chen G, Abdeen AA, Wang Y, Shahi PK, Robertson S, Xie R, Suzuki M, Pattnaik BR, Saha K, Gong S. Nature Nanotechnology 2019 Sept 9 31501532/
s41565-019-0539-2
Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles Liu J, Chang J, Jiang Y, Meng X, Sun T, Mao L, Xu Q, Wang M Advanced Materials 2019 Aug 31 31215123
Continuous evolution of base editors with expanded target compatibility and improved activity Thuronyi BW, Koblan LW, Levy JM, Yeh W, Zheng C, Newby GA, Wilson C, Bhaumik M, Shubina-Oleinik O, Holt JR, Lui DR. Nature Nanotechnology 2019 Jul 22 31332326/
s41587-019-0193-0
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity Xiao Y, Muhuri M, Li S, Qin W, Xu G, Luo L, Li J, Letizia AJ, Wang SK, Chan YK, Wang C, Fuchs SP, Wang D, Su Q, Nahid MA, Church GM, Farzan M, Yang L, Wei Y, Desrosiers RC, Mueller C, Tai PW, Gao G. JCI Insight 2019 Jul 11 31112525
An anionic human protein mediates cationic liposome delivery of genome editing proteins into mammalian cells Kim YB, Zhao KT, Thompson DB, Liu DR. Nature Communications 2019 Jul 2 31266953
High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs Xu, Meiyu; Li, Jia; Xie, Jun; He, Ran; Su, Qin; Gao, Guangping; Tai, Phillip W L Human Gene Therapy 2019 Jun 17 31072208/
1UG3HL147367-01
Circularly permuted and PAM-modified Cas9 variants broaden the targeting scope of base editors Huang TP, Zhao KT, Miller SM, Gaudeli NM, Oakes BL, Fellmann C, Savage DF, Liu DR Nat Biotechnol 2019 May 20 31110355
Development of hRad51-Cas9 nickase fusions that mediate HDR without double-stranded breaks. Rees HA, Yeh WH, Liu DR Nature Communications 2019 May 17 31110355
Intrathecal Adeno-Associated Viral Vector-Mediated Gene Delivery for Adrenomyeloneuropathy Gong, Yi; Berenson, Anna; Laheji, Fiza; Gao, Guangping; Wang, Dan; Ng, Carrie; Volak, Adrienn; Kok, Rene; Kreouzis, Vasileios; Dijkstra, Inge M; Kemp, Stephan; Maguire, Casey A; Eichler, Florian Human Gene Therapy 2019 May 30358470/Proj. 1UG3HL147367-01
Analysis and minimization of cellular RNA editing by DNA adenine base editors Rees HA, Wilson C, Doman JL, Liu DR Science Advances 2019 May 8 31086823
Integrating Combinatorial Lipid Nanoparticle and Chemically Modified Protein for Intracellular Delivery and Genome Editing Chang, Jin; Chen, Xianghan; Glass, Zachary; Gao, Feng, Mao, Lanqun; Wang, Ming; Xu, Qiaobing Accounts of Chemical Research 2019 Mar 19 30586281
Intracellular Delivery of His-Tagged Genome-Editing Proteins Enabled by Nitrilotriacetic Acid-Containing Lipidoid Nanoparticles. Yamin Li Alice Chukun Li Qiaobing Xu Advance Healthcare Materials 2019 Mar 8 30565897/1UG3TR002636-01
Anti-drug Antibody Responses Impair Prophylaxis Mediated by AAV-Delivered HIV-1 Broadly Neutralizing Antibodies Gardner, Matthew R; Fetzer, Ina; Kattenhorn, Lisa M; Davis-Gardner, Meredith E; Zhou, Amber S; Alfant, Barnett; Weber, Jesse A; Kondur, Hema R; Martinez-Navio, Jose M; Fuchs, Sebastian P; Desrosiers, Ronald C; Gao, Guangping; Lifson, Jeffrey D; Farzan, Michael Molecular therapy : the journal of the American Society of Gene Therapy 2019 Mar 6 PMC6403482
Simultaneous targeting of linked loci in mouse embryos using base editing Lee, Hye Kyung; Willi, Michaela; Smith, Harold E; Miller, Shannon M; Liu, David R; Liu, Chengyu; Hennighausen, Lothar Scientific Reports 2019 Feb 7 31101808
Illuminating the genome-wide activity of genome editors for safe and effective therapeutics Cheng, Yong; Tsai, Shengdar Q Genome Biology 2018 Dec 22 30577870/
Proj. U01HL145793
Targeting fidelity of adenine and cytosine base editors in mouse embryos Lee, Hye Kyung; Willi, Michaela; Miller, Shannon M; Kim, Sojung; Liu, Chengyu; Liu, David R Nature Communications 2018 Nov 15 30442934
Defining CRISPR–Cas9 genome-wide nuclease activities with CIRCLE-seq. Lazzarotto, Cicera R; Nguyen, Nhu T; Tang, Xing; Malagon-Lopez, Jose; Guo, Jimmy A; Aryee, Martin J; Joung, J Keith; Tsai, Shengdar Q Nature Protocols 2018 Nov 13 30341435
Understanding and repurposing CRISPR-mediated alternative splicing Smith, Jordan L; Mou, Haiwei; Xue, Wen Genome Biology 2018 Nov 6 30400804
Genome editing to ‘re-write’ wrongs Perry ME, Valdes KM, Austin CP, Brooks PJ Nature Reviews Drug Discovery 2018 Oct 17 29930285