Search Results - "WATTS, K"

The chemical evolution of oligonucleotide therapies of clinical utility by Khvorova, Anastasia, Watts, Jonathan K

“…Refinements in the chemistries employed in oligonucleotide therapeutics have galvanized clinical progress. The complex interplay between chemical modifications…”
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Silencing disease genes in the laboratory and the clinic by Watts, Jonathan K, Corey, David R

“…Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress…”
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Enhancer RNA Facilitates NELF Release from Immediate Early Genes by Schaukowitch, Katie, Joo, Jae-Yeol, Liu, Xihui, Watts, Jonathan K., Martinez, Carlos, Kim, Tae-Kyung

“…Enhancer RNAs (eRNAs) are a class of long noncoding RNAs (lncRNA) expressed from active enhancers, whose function and action mechanism are yet to be firmly…”
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Mafic Alkaline Magmatism and Rare Earth Element Mineralization in the Mojave Desert, California: The Bobcat Hills Connection to Mountain Pass by Watts, K. E., Miller, D. M., Ponce, D. A.

“…Occurrences of alkaline and carbonatite rocks with high concentrations of rare earth elements (REE) are a defining feature of Precambrian geology in the Mojave…”
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Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide by Tran, Hélène, Moazami, Michael P., Yang, Huiya, McKenna-Yasek, Diane, Douthwright, Catherine L., Pinto, Courtney, Metterville, Jake, Shin, Minwook, Sanil, Nitasha, Dooley, Craig, Puri, Ajit, Weiss, Alexandra, Wightman, Nicholas, Gray-Edwards, Heather, Marosfoi, Miklos, King, Robert M., Kenderdine, Thomas, Fabris, Daniele, Bowser, Robert, Watts, Jonathan K., Brown, Robert H.

“…Expansions of a G 4 C 2 repeat in the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD),…”
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ConfGen: A Conformational Search Method for Efficient Generation of Bioactive Conformers by Watts, K. Shawn, Dalal, Pranav, Murphy, Robert B, Sherman, Woody, Friesner, Rich A, Shelley, John C

“…We describe the methodology, parametrization, and application of a conformational search method, called ConfGen, designed to efficiently generate bioactive…”
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Oligonucleotide therapeutics: chemistry, delivery and clinical progress by Sharma, Vivek K, Watts, Jonathan K

“…Oligonucleotide therapeutics have the potential to become a third pillar of drug development after small molecules and protein therapeutics. However, the three…”
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Macrocycle Conformational Sampling with MacroModel by Watts, K. Shawn, Dalal, Pranav, Tebben, Andrew J, Cheney, Daniel L, Shelley, John C

“…Sampling low energy conformations of macrocycles is challenging due to the large size of many of these molecules and the constraints imposed by the macrocycle…”
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Waste Cooking Oil as an Alternate Feedstock for Biodiesel Production by Chhetri, Arjun B., Watts, K. Chris, Islam, M. Rafiqul

“…As crude oil price reach a new high, the need for developing alternate fuels has become acute. Alternate fuels should be economically attractive in order to…”
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Heavily and fully modified RNAs guide efficient SpyCas9-mediated genome editing by Mir, Aamir, Alterman, Julia F., Hassler, Matthew R., Debacker, Alexandre J., Hudgens, Edward, Echeverria, Dimas, Brodsky, Michael H., Khvorova, Anastasia, Watts, Jonathan K., Sontheimer, Erik J.

“…RNA-based drugs depend on chemical modifications to increase potency and to decrease immunogenicity in vivo. Chemical modification will likely improve the…”
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Non-edible plant oils as new sources for biodiesel production by Chhetri, Arjun B, Tango, Martin S, Budge, Suzanne M, Watts, K C, Islam, M R

“…Due to the concern on the availability of recoverable fossil fuel reserves and the environmental problems caused by the use those fossil fuels, considerable…”
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Surface tensions of petro-diesel, canola, jatropha and soapnut biodiesel fuels at elevated temperatures and pressures by Chhetri, A.B., Watts, K.C.

“…► Surface tensions of three biodiesel fuels and diesel were measured at elevated temperatures and pressures. ► Surface tensions were measured up to 473K and…”
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Distinct influences of affective and cognitive factors on children’s non-verbal and verbal mathematical abilities by Wu, Sarah S., Chen, Lang, Battista, Christian, Smith Watts, Ashley K., Willcutt, Erik G., Menon, Vinod

“…Individual differences in children’s math performance have been associated with math anxiety, attention problems, working memory (WM), and reading skills, but…”
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Chemically modified siRNA: tools and applications by Watts, Jonathan K., Deleavey, Glen F., Damha, Masad J.

“…Chemical modification provides solutions to many of the challenges facing siRNA therapeutics. This review examines the various siRNA modifications available,…”
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Domain-inlaid Nme2Cas9 adenine base editors with improved activity and targeting scope by Bamidele, Nathan, Zhang, Han, Dong, Xiaolong, Cheng, Haoyang, Gaston, Nicholas, Feinzig, Hailey, Cao, Hanbing, Kelly, Karen, Watts, Jonathan K., Xie, Jun, Gao, Guangping, Sontheimer, Erik J.

“…Nme2Cas9 has been established as a genome editing platform with compact size, high accuracy, and broad targeting range, including single-AAV-deliverable…”
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Genome-wide detection of CRISPR editing in vivo using GUIDE-tag by Liang, Shun-Qing, Liu, Pengpeng, Smith, Jordan L., Mintzer, Esther, Maitland, Stacy, Dong, Xiaolong, Yang, Qiyuan, Lee, Jonathan, Haynes, Cole M., Zhu, Lihua Julie, Watts, Jonathan K., Sontheimer, Erik J., Wolfe, Scot A., Xue, Wen

“…Analysis of off-target editing is an important aspect of the development of safe nuclease-based genome editing therapeutics. in vivo assessment of nuclease…”
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Poly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD by Krishnan, Gopinath, Raitcheva, Denitza, Bartlett, Daniel, Prudencio, Mercedes, McKenna-Yasek, Diane M., Douthwright, Catherine, Oskarsson, Björn E., Ladha, Shafeeq, King, Oliver D., Barmada, Sami J., Miller, Timothy M., Bowser, Robert, Watts, Jonathan K., Petrucelli, Leonard, Brown, Robert H., Kankel, Mark W., Gao, Fen-Biao

“…GGGGCC repeat expansion in C9ORF72 , which can be translated in both sense and antisense directions into five dipeptide repeat (DPR) proteins, including…”
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Structure of the catalytically active APOBEC3G bound to a DNA oligonucleotide inhibitor reveals tetrahedral geometry of the transition state by Maiti, Atanu, Hedger, Adam K., Myint, Wazo, Balachandran, Vanivilasini, Watts, Jonathan K., Schiffer, Celia A., Matsuo, Hiroshi

“…APOBEC3 proteins (A3s) are enzymes that catalyze the deamination of cytidine to uridine in single-stranded DNA (ssDNA) substrates, thus playing a key role in…”
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Sleep dysfunction and thalamic abnormalities in adolescents at ultra high-risk for psychosis by Lunsford-Avery, Jessica R, Orr, Joseph M, Gupta, Tina, Pelletier-Baldelli, Andrea, Dean, Derek J, Smith Watts, Ashley K, Bernard, Jessica, Millman, Zachary B, Mittal, Vijay A

“…Abstract Background Sleep dysfunction is a pervasive, distressing characteristic of psychosis, yet little is known regarding sleep quality prior to illness…”
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Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts by Shin, Minwook, Chan, Io Long, Cao, Yuming, Gruntman, Alisha M, Lee, Jonathan, Sousa, Jacquelyn, Rodríguez, Tomás C, Echeverria, Dimas, Devi, Gitali, Debacker, Alexandre J, Moazami, Michael P, Krishnamurthy, Pranathi Meda, Rembetsy-Brown, Julia M, Kelly, Karen, Yukselen, Onur, Donnard, Elisa, Parsons, Teagan J, Khvorova, Anastasia, Sontheimer, Erik J, Maehr, René, Garber, Manuel, Watts, Jonathan K

“…Abstract The lung is a complex organ with various cell types having distinct roles. Antisense oligonucleotides (ASOs) have been studied in the lung, but it has…”
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