Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell-Type-Specific Gene Editing

Autor: Ariana Hirsh, Robert Dullea, Meihua Tu, Spiros Liras, Joan Compte Barrón, Kris A. Borzilleri, Nannan Ma, Rima Mendonsa, Justin Bellenger, Romain Rouet, Lorena de Oñate, Xidong Feng, Jennifer A. Doudna, Nathanael G. Lintner, David M. Rubitski, Benjamin A. Thuma, Marc D. Roy, Alison H. Varghese, Kim F. McClure, Ross C. Wilson, Thomas J. McLellan, Hanna M. Wisniewska, James E. Finley, Boris A. Chrunyk, Vincent Mascitti, Kaihong Zhou, Kevin D. Hesp

Publikováno v: Journal of the American Chemical Society, vol 140, iss 21
Rouet, Romain; Thuma, Benjamin A; Roy, Marc D; Lintner, Nathanael G; Rubitski, David M; Finley, James E; et al.(2018). Receptor-Mediated Delivery of CRISPR-Cas9 Endonuclease for Cell-Type-Specific Gene Editing.. Journal of the American Chemical Society, 140(21), 6596-6603. doi: 10.1021/jacs.8b01551. UC Berkeley: Retrieved from: http://www.escholarship.org/uc/item/61k3t664

CRISPR-Cas RNA-guided endonucleases hold great promise for disrupting or correcting genomic sequences through site-specific DNA cleavage and repair. However, the lack of methods for cell- and tissue-selective delivery currently limits both research a

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9af967c5dc058dcd0e0a6cfe05eb8b81
https://doi.org/10.1021/jacs.8b01551

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Discovery of a Series of Efficient, Centrally Efficacious BACE1 Inhibitors through Structure-Based Drug Design

Autor: Christopher John Helal, Kevin Atchison, Christopher Ryan Butler, Cathleen Gonzales, Ricardo Lira, Shawn D. Doran, Feng Pan, Felix Vajdos, Alison H. Varghese, Gabriela Barreiro, Michael Aaron Brodney, Jinlong Wang, Elizabeth Mary Beck, Luis Martinez-Alsina, Yong Zhang, Kevin D. Parris, David Riddell, Joseph Michael Young, Leanne M. Buzon, Ashley Robshaw, Kevin Ogilvie, Jason K. Dutra, Brian T. O’Neill, John C. Murray, Charles E. Nolan

Publikováno v: Journal of Medicinal Chemistry. 58:2678-2702

The identification of centrally efficacious β-secretase (BACE1) inhibitors for the treatment of Alzheimer's disease (AD) has historically been thwarted by an inability to maintain alignment of potency, brain availability, and desired absorption, dis

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::455eb0d85337e81623d88cc6b235a8b0
https://doi.org/10.1021/jm501833t

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