Search Results - "Watts, Jonathan 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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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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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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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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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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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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Quantifying the activity profile of ASO and siRNA conjugates in glioblastoma xenograft tumors in vivo by Sarli, Samantha L, Fakih, Hassan H, Kelly, Karen, Devi, Gitali, Rembetsy-Brown, Julia M, McEachern, Holly R, Ferguson, Chantal M, Echeverria, Dimas, Lee, Jonathan, Sousa, Jacquelyn, Sleiman, Hanadi F, Khvorova, Anastasia, Watts, Jonathan K

“…Glioblastoma multiforme is a universally lethal brain tumor that largely resists current surgical and drug interventions. Despite important advancements in…”
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Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo by Zhang, Han, Kelly, Karen, Lee, Jonathan, Echeverria, Dimas, Cooper, David, Panwala, Rebecca, Amrani, Nadia, Chen, Zexiang, Gaston, Nicholas, Wagh, Atish, Newby, Gregory A, Xie, Jun, Liu, David R, Gao, Guangping, Wolfe, Scot A, Khvorova, Anastasia, Watts, Jonathan K, Sontheimer, Erik J

“…Abstract Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add challenges for delivery. Chemical modification, which has been key to the…”
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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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G-rich motifs within phosphorothioate-based antisense oligonucleotides (ASOs) drive activation of FXN expression through indirect effects by Wang, Feng, Calvo-Roitberg, Ezequiel, Rembetsy-Brown, Julia M, Fang, Minggang, Sousa, Jacquelyn, Kartje, Zachary J, Krishnamurthy, Pranathi Meda, Lee, Jonathan, Green, Michael R, Pai, Athma A, Watts, Jonathan K

“…Friedreich's ataxia is an incurable disease caused by frataxin (FXN) protein deficiency, which is mostly induced by GAA repeat expansion in intron 1 of the FXN…”
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EZH2 inhibition reactivates epigenetically silenced FMR1 and normalizes molecular and electrophysiological abnormalities in fragile X syndrome neurons by Fang, Minggang, Deibler, Sara K, Krishnamurthy, Pranathi Meda, Wang, Feng, Rodriguez, Paola, Banday, Shahid, Virbasius, Ching-Man, Sena-Esteves, Miguel, Watts, Jonathan K, Green, Michael R

“…Fragile X Syndrome (FXS) is a neurological disorder caused by epigenetic silencing of the gene. Reactivation of is a potential therapeutic approach for FXS…”
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Novel Cluster and Monomer-Based GalNAc Structures Induce Effective Uptake of siRNAs in Vitro and in Vivo by Sharma, Vivek K, Osborn, Maire F, Hassler, Matthew R, Echeverria, Dimas, Ly, Socheata, Ulashchik, Egor A, Martynenko-Makaev, Yury V, Shmanai, Vadim V, Zatsepin, Timofei S, Khvorova, Anastasia, Watts, Jonathan K

“…GalNAc conjugation is emerging as a dominant strategy for delivery of therapeutic oligonucleotides to hepatocytes. The structure and valency of the GalNAc…”
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5'-Modifications improve potency and efficacy of DNA donors for precision genome editing by Ghanta, Krishna S, Chen, Zexiang, Mir, Aamir, Dokshin, Gregoriy A, Krishnamurthy, Pranathi M, Yoon, Yeonsoo, Gallant, Judith, Xu, Ping, Zhang, Xiao-Ou, Ozturk, Ahmet Rasit, Shin, Masahiro, Idrizi, Feston, Liu, Pengpeng, Gneid, Hassan, Edraki, Alireza, Lawson, Nathan D, Rivera-Pérez, Jaime A, Sontheimer, Erik J, Watts, Jonathan K, Mello, Craig C

“…Nuclease-directed genome editing is a powerful tool for investigating physiology and has great promise as a therapeutic approach to correct mutations that…”
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Next-Generation Peptide Nucleic Acid Chimeras Exhibit High Affinity and Potent Gene Silencing by Debacker, Alexandre J, Sharma, Vivek K, Meda Krishnamurthy, Pranathi, O’Reilly, Daniel, Greenhill, Rachel, Watts, Jonathan K

“…We present a new design of mixed-backbone antisense oligonucleotides (ASOs) containing both DNA and peptide nucleic acid (PNA). Previous generations of PNA–DNA…”
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RNA therapeutics on the rise by Wang, Feng, Zuroske, Travis, Watts, Jonathan K.

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Dendritic amphiphilic siRNA: Selective albumin binding, in vivo efficacy, and low toxicity by Fakih, Hassan H, Tang, Qi, Summers, Ashley, Shin, Minwook, Buchwald, Julianna E, Gagnon, Rosemary, Hariharan, Vignesh N, Echeverria, Dimas, Cooper, David A, Watts, Jonathan K, Khvorova, Anastasia, Sleiman, Hanadi F

“…Although an increasing number of small interfering RNA (siRNA) therapies are reaching the market, the challenge of efficient extra-hepatic delivery continues…”
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