Zobrazeno 1 - 10
of 136
pro vyhledávání: '"Benjamin P Kleinstiver"'
Autor:
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
Publikováno v:
Nature Communications, Vol 15, Iss 1, Pp 1-17 (2024)
Abstract Huntington’s disease (HD) is a dominant neurological disorder caused by an expanded HTT exon 1 CAG repeat that lengthens huntingtin’s polyglutamine tract. Lowering mutant huntingtin has been proposed for treating HD, but genetic modifier
Externí odkaz:
https://doaj.org/article/04c90e61e38c4922b3fb3425418eb127
Autor:
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
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-8 (2023)
Abstract CRISPR enzymes require a defined protospacer adjacent motif (PAM) flanking a guide RNA-programmed target site, limiting their sequence accessibility for robust genome editing applications. In this study, we recombine the PAM-interacting doma
Externí odkaz:
https://doaj.org/article/c6eaa1afc2b34657a91e037a58ba1052
Autor:
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
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-15 (2023)
Abstract Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using a mouse model Atp2b2 Obl/+, with a dominant hearing loss mutation (Oblivion), we show that liposome-mediated in vivo delivery of CRISPR-Cas9 ribonucle
Externí odkaz:
https://doaj.org/article/a29936b8a683497cbbd1b953799336a4
Autor:
Lisa Nieland, Thomas S. van Solinge, Pike See Cheah, Liza M. Morsett, Joseph El Khoury, Joseph I. Rissman, Benjamin P. Kleinstiver, Marike L.D. Broekman, Xandra O. Breakefield, Erik R. Abels
Publikováno v:
Molecular Therapy: Oncolytics, Vol 25, Iss , Pp 121-136 (2022)
Non-coding RNAs, including microRNAs (miRNAs), support the progression of glioma. miR-21 is a small, non-coding transcript involved in regulating gene expression in multiple cellular pathways, including the regulation of proliferation. High expressio
Externí odkaz:
https://doaj.org/article/e7f869deba594a7dad62253d7bf25498
Autor:
Jeremy Vicencio, Carlos Sánchez-Bolaños, Ismael Moreno-Sánchez, David Brena, Charles E. Vejnar, Dmytro Kukhtar, Miguel Ruiz-López, Mariona Cots-Ponjoan, Alejandro Rubio, Natalia Rodrigo Melero, Jesús Crespo-Cuadrado, Carlo Carolis, Antonio J. Pérez-Pulido, Antonio J. Giráldez, Benjamin P. Kleinstiver, Julián Cerón, Miguel A. Moreno-Mateos
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-13 (2022)
PAM requirement is a constraint for genome editing but this has been circumvented by engineered Cas9 nucleases as SpG and SpRY recognizing minimal PAM sequences. Here, the authors validate and optimize SpG and SpRY in vivo expanding the targeting lan
Externí odkaz:
https://doaj.org/article/65af9233becc4657b1eecfc5e6d8262e
Autor:
Florian Wünnemann, Thierry Fotsing Tadjo, Mélissa Beaudoin, Simon Lalonde, Ken Sin Lo, Benjamin P Kleinstiver, Guillaume Lettre
Publikováno v:
PLoS Genetics, Vol 19, Iss 3, p e1010680 (2023)
Genome-wide association studies have identified >250 genetic variants associated with coronary artery disease (CAD), but the causal variants, genes and molecular mechanisms remain unknown at most loci. We performed pooled CRISPR screens to test the i
Externí odkaz:
https://doaj.org/article/7b4b43e0998f413aa9650954810b1239
Autor:
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, Luigi D. Notarangelo, Cicera R. Lazzarotto, Shengdar Q. Tsai, Xiaolin Wu, Gary A. Dahl, Matthew H. Porteus, Harry L. Malech, Suk See De Ravin
Publikováno v:
Frontiers in Immunology, Vol 13 (2023)
IntroductionEx vivo gene therapy for treatment of Inborn errors of Immunity (IEIs) have demonstrated significant clinical benefit in multiple Phase I/II clinical trials. Current approaches rely on engineered retroviral vectors to randomly integrate c
Externí odkaz:
https://doaj.org/article/55c1f88429d84741ac3174cb89df6a2b
Allele-specific silencing of the gain-of-function mutation in Huntington’s disease using CRISPR/Cas9
Autor:
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
Publikováno v:
JCI Insight, Vol 7, Iss 19 (2022)
Dominant gain-of-function mechanisms in Huntington’s disease (HD) suggest that selective silencing of mutant HTT produces robust therapeutic benefits. Here, capitalizing on exonic protospacer adjacent motif–altering (PAM-altering) SNP (PAS), we d
Externí odkaz:
https://doaj.org/article/8c4d07b7f240401da0278594ea55cf8a
Autor:
Lilian Cruz, Bence György, Pike See Cheah, Benjamin P. Kleinstiver, William A. Eimer, Sara P. Garcia, Nutan Sharma, Laurie J. Ozelius, D. Cristopher Bragg, J. Keith Joung, Osmar Norberto de Souza, Luis Fernando Saraiva Macedo Timmers, Xandra O. Breakefield
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 21, Iss , Pp 1-12 (2020)
Most individuals affected with DYT1 dystonia have a heterozygous 3-bp deletion in the TOR1A gene (c.907_909delGAG). The mutation appears to act through a dominant-negative mechanism compromising normal torsinA function, and it is proposed that reduci
Externí odkaz:
https://doaj.org/article/7258ef6927e544c29b5663dbd5f40e37
Autor:
Killian S. Hanlon, Benjamin P. Kleinstiver, Sara P. Garcia, Mikołaj P. Zaborowski, Adrienn Volak, Stefan E. Spirig, Alissa Muller, Alexander A. Sousa, Shengdar Q. Tsai, Niclas E. Bengtsson, Camilla Lööv, Martin Ingelsson, Jeffrey S. Chamberlain, David P. Corey, Martin J. Aryee, J. Keith Joung, Xandra O. Breakefield, Casey A. Maguire, Bence György
Publikováno v:
Nature Communications, Vol 10, Iss 1, Pp 1-11 (2019)
In-depth characterization of adeno-associated virus (AAV)-mediated CRISPR delivery is still lacking. Here, the authors show high levels of integration into Cas9-induced double-strand breaks (DSBs) in therapeutically relevant genes in vivo.
Externí odkaz:
https://doaj.org/article/d463e02a3bdc40c4aca343f4b409877a