Publication Title Authors Journal Publication Date PubMed Project

Gene Delivery to Nonhuman Primate Preimplantation Embryos Using Recombinant Adeno‐Associated Virus

 Wang, Dan; Niu, Yuyu; Ren, Lingzhi; Kang, Yu; Tai, Phillip W L; Si, Chenyang; Mendonca, Craig A; Ma, Hong; Gao, Guangping; Ji, Weizhi Advanced Science
2019 Nov 6
 31728271 1UG3HL147367-01

Engineered amphiphilic peptides enable delivery of proteins and CRISPR-associated nucleases to airway epithelia

 Sateesh Krishnamurthy, Christine Wohlford-Lenane, Suhas Kandimalla, Gilles Sartre, David K. Meyerholz, Vanessa Théberge, Stéphanie Hallée, Anne-Marie Duperré, Thomas Del’Guidice, Jean-Pascal Lepetit-Stoffaes, Xavier Barbeau, David Guay & Paul B. McCray Jr. Nature Communications
2019 Oct 28
 31659165 s41467-019-12922-y
Structures of Neisseria meningitidis Cas9 Complexes in Catalytically Poised and Anti-CRISPR-Inhibited States Sun W, Yang J, Cheng Z, Amrani N, Liu C, Wang K, Ibraheim R, Edraki A, Huang X, Wang M, Wang J, Liu L, Sheng G, Yang Y, Lou J, Sontheimer EJ, Wang Y Molecular Cell
2019 Oct 22
 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
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo. Ibraheim, Raed; Song, Chun-Qing; Mir, Aamir; Amrani, Nadia; Xue, Wen; Sontheimer, Erik J Genome Biology
2019 Sept 19
 30231914 1UG3HL147367-01
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 1UG3HL147367-01
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 Aug 30
 31072208 1UG3HL147367-01
Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins Lee, Jooyoung; Mou, Haiwei; Ibraheim, Raed; Liang, Shun-Qing; Liu, Pengpeng; Xue, Wen; Sontheimer, Erik J Cold Spring Harb Protoc
2019 Aug 22
 31439808 1UG3HL147367-01
Measuring the Infectious Titer of Recombinant Adenovirus Using Tissue Culture Infection Dose 50% (TCID50) End-Point Dilution and Quantitative Polymerase Chain Reaction (qPCR) Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harb Protoc
2019 Aug 1
 31371467 1UG3HL147367-01
Preparation of a DNA Standard for Quantitative Polymerase Chain Reaction (qPCR). Lock, Martin; Korn, Michael; Wilson, James; Sena-Esteves, Miguel; Gao, Guangping Cold Spring Harb Protoc
2019 Aug 1
 31371468 1UG3HL147367-01
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, Liu DR. Nature Biotechnology
2019 Jul 22
 31332326 s41587-019-0193-0
Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis. Yang, Yeon-Suk; Xie, Jun; Wang, Dan; Kim, Jung-Min; Tai, Phillip W L; Gravallese, Ellen; Gao, Guangping; Shim, Jae-Hyuck Nature Communications
2019 Jul 4
 31273195 1UG3HL147367-01
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
Depletion of TRRAP Induces p53-Independent Senescence in Liver Cancer by Down-Regulating Mitotic Genes. Kwan, Suet-Yan; Sheel, Ankur; Song, Chun-Qing; Zhang, Xiao-Ou; Jiang, Tingting; Dang, Hien; Cao, Yueying; Ozata, Deniz M; Mou, Haiwei; Yin, Hao; Weng, Zhiping; Wang, Xin Wei; Xue, Wen Hepatology
2019 Jun 12
 31188495 1UG3HL147367-01
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity Xiao, Yuanyuan; Muhuri, Manish; Li, Shaoyong; Qin, Wanru; Xu, Guangchao; Luo, Li; Li, Jia; Letizia, Alexander J; Wang, Sean K; Chan, Ying Kai; Wang, Chunmei; Fuchs, Sebastian P; Wang, Dan; Su, Qin; Nahid, M Abu; Church, George M; Farzan, Michael; Yang, Li; Wei, Yuquan; Desrosiers, Ronald C; Mueller, Christian; Tai, Phillip Wl; Gao, Guangping JCI Insight
2019 May 21
 31112525 1UG3HL147367-01
Adeno-associated virus vector as a platform for gene therapy delivery. 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 Nature Reviews Drug Discovery
2019 May 18
 30710128 1UG3HL147367-01
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 15
 30358470 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 1UG3HL147367-01
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 1UG3HL147367-01
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
 30704961 1UG3HL147367-01
A Compact, High-Accuracy Cas9 with a Dinucleotide PAM for In Vivo Genome Editing Edraki, Alireza; Mir, Aamir; Ibraheim, Raed; Gainetdinov, Ildar; Yoon, Yeonsoo; Song, Chun-Qing; Cao, Yueying; Gallant, Judith; Xue, Wen; Rivera-Pérez, Jaime A; Sontheimer, Erik J Molecular Cell
2019 Feb 21
 30581144 1UG3HL147367-01
Quantitative and Digital Droplet-Based AAV Genome Titration Sanmiguel, Julio; Gao, Guangping; Vandenberghe, Luk H Methods in Mol Biol
2019 Feb 20
 30783968 1UG3HL147367-01