Comparison and cross-validation of long-read and short-read target-enrichment sequencing methods to assess AAV vector integration into host genome

Autor: Mark Sheehan, Steven W. Kumpf, Jessie Qian, David M. Rubitski, Elias Oziolor, Thomas A. Lanz

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 4, Pp 101352- (2024)

Evaluation of host integration profiles by adeno-associated virus (AAV) is an important component of de-risking novel AAV gene therapies. Targeted enrichment sequencing (TES) is a cost-effective and comprehensive method for assessing integration. Mos

Externí odkaz: https://doaj.org/article/654cd3cfc1ad47d7988230718f57b959

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

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

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

Publikováno v: The FASEB Journal. 32

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::f270393f8db883382554c04a0d34fe48
https://doi.org/10.1096/fasebj.2018.32.1_supplement.649.2

Discovery of Two Clinical Histamine H3 Receptor Antagonists: trans-N-Ethyl-3-fluoro-3-[3-fluoro-4-(pyrrolidinylmethyl)phenyl]cyclobutanecarboxamide (PF-03654746) and trans-3-Fluoro-3-[3-fluoro-4-(pyrrolidin-1-ylmethyl)phenyl]-N-(2-methylpropyl)cyclobutanecarboxamide (PF-03654764)

Autor: Rama Y. Chandrasekaran, Jiri Aubrecht, Frederick R. Nelson, Linda A. Chatman, Hans Rollema, Frank M. Nedza, John M. Marcek, Philip A. Iredale, Jennifer B. Munzner, Betty Pettersen, Raggon Jeffrey W, David M. Rubitski, Julie Cianfrogna, Larry C. James, Karen W. Cook, Michael J. Banker, Diane F. Wong, Anne W. Schmidt, Michael Homiski, Howard Harry R, Daniel J. Lettiere, Butler Todd W, Scot Richard Mente, Jody Freeman, Travis T. Wager, Douglas K. Spracklin

Publikováno v: Journal of Medicinal Chemistry. 54:7602-7620

The discovery of two histamine H3 antagonist clinical candidates is disclosed. The pathway to identification of the two clinical candidates, 6 (PF-03654746) and 7 (PF-03654764) required five hypothesis driven design cycles. The key to success in iden

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::7c253ef2a0b55c8bf1f6522eccc0c6ed
https://doi.org/10.1021/jm200939b

Identification of multiple 5-HT₄ partial agonist clinical candidates for the treatment of Alzheimer's disease

Autor: Michael A, Brodney, David E, Johnson, Aarti, Sawant-Basak, Karen J, Coffman, Elena M, Drummond, Emily L, Hudson, Katherine E, Fisher, Hirohide, Noguchi, Nobuaki, Waizumi, Laura L, McDowell, Alexandros, Papanikolaou, Betty A, Pettersen, Anne W, Schmidt, Elaine, Tseng, Kim, Stutzman-Engwall, David M, Rubitski, Michelle A, Vanase-Frawley, Sarah, Grimwood

Publikováno v: Journal of medicinal chemistry. 55(21)

The cognitive impairments observed in Alzheimer's disease (AD) are in part a consequence of reduced acetylcholine (ACh) levels resulting from a loss of cholinergic neurons. Preclinically, serotonin 4 receptor (5-HT(4)) agonists are reported to modula

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::992b76c5d59721c47b0dcac97b51e486
https://pubmed.ncbi.nlm.nih.gov/22974325