Multimodal CRISPR perturbations of GWAS loci associated with coronary artery disease in vascular endothelial cells. by Florian Wünnemann, Thierry Fotsing Tadjo, Mélissa Beaudoin, Simon Lalonde, Ken Sin Lo, Benjamin P Kleinstiver, Guillaume Lettre

Get full text

Allele-specific silencing of the gain-of-function mutation in Huntington’s disease using CRISPR/Cas9 by Jun Wan Shin, Eun Pyo Hong, Seri S. Park, Doo Eun Choi, Ihn Sik Seong, Madelynn N. Whittaker, Benjamin P. Kleinstiver, Richard Z. Chen, Jong-Min Lee

Get full text

CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells by Julie Brault, Taylor Liu, Siyuan Liu, Amanda Lawson, Uimook Choi, Nikita Kozhushko, Vera Bzhilyanskaya, Mara Pavel-Dinu, Ronald J. Meis, Michael A. Eckhaus, Sandra S. Burkett, Marita Bosticardo, Benjamin P. Kleinstiver, Benjamin P. Kleinstiver, Luigi D. Notarangelo, Cicera R. Lazzarotto, Shengdar Q. Tsai, Xiaolin Wu, Gary A. Dahl, Matthew H. Porteus, Harry L. Malech, Suk See De Ravin

Get full text

Non-invasive detection of allele-specific CRISPR-SaCas9-KKH disruption of TOR1A DYT1 allele in a xenograft mouse model by Katia E. Maalouf, Dawn Madison Frederick, Nutan Sharma, Edwina Abou Haidar, Tianhe Xiao, Justin Seungkyu Han, Mohammed S. Mahamdeh, Roy J. Soberman, David Rufino-Ramos, Benjamin P. Kleinstiver, Hyder A. Jinnah, Christine A. Vaine, D. Cristopher Bragg, Koen Breyne

Get full text

Treatment of monogenic and digenic dominant genetic hearing loss by CRISPR-Cas9 ribonucleoprotein delivery in vivo by Yong Tao, Veronica Lamas, Wan Du, Wenliang Zhu, Yiran Li, Madelynn N. Whittaker, John A. Zuris, David B. Thompson, Arun Prabhu Rameshbabu, Yilai Shu, Xue Gao, Johnny H. Hu, Charles Pei, Wei-Jia Kong, Xuezhong Liu, Hao Wu, Benjamin P. Kleinstiver, David R. Liu, Zheng-Yi Chen

Get full text

Cell-based artificial APC resistant to lentiviral transduction for efficient generation of CAR-T cells from various cell sources by Irene Scarfò, Marcela V Maus, Andrea Schmidts, Leah C Marsh, Ambike A Srivastava, Amanda A Bouffard, Angela C Boroughs, Rebecca C Larson, Felipe Bedoya, Bryan D Choi, Matthew J Frigault, Stefanie R Bailey, Mark B Leick, Sonika Vatsa, Michael C Kann, Michelle S Prew, Benjamin P Kleinstiver, J Keith Joung

Get full text

Splice modulators target PMS1 to reduce somatic expansion of the Huntington’s disease-associated CAG repeat by Zachariah L. McLean, Dadi Gao, Kevin Correia, Jennie C. L. Roy, Shota Shibata, Iris N. Farnum, Zoe Valdepenas-Mellor, Marina Kovalenko, Manasa Rapuru, Elisabetta Morini, Jayla Ruliera, Tammy Gillis, Diane Lucente, Benjamin P. Kleinstiver, Jong-Min Lee, Marcy E. MacDonald, Vanessa C. Wheeler, Ricardo Mouro Pinto, James F. Gusella

Get full text

PAM-flexible genome editing with an engineered chimeric Cas9 by Lin Zhao, Sabrina R. T. Koseki, Rachel A. Silverstein, Nadia Amrani, Christina Peng, Christian Kramme, Natasha Savic, Martin Pacesa, Tomás C. Rodríguez, Teodora Stan, Emma Tysinger, Lauren Hong, Vivian Yudistyra, Manvitha R. Ponnapati, Joseph M. Jacobson, George M. Church, Noah Jakimo, Ray Truant, Martin Jinek, Benjamin P. Kleinstiver, Erik J. Sontheimer, Pranam Chatterjee

Get full text