Search Results - "Bennett, C"

Therapeutic Antisense Oligonucleotides Are Coming of Age by Bennett, C. Frank

“…The first published description of therapeutic applications of antisense oligonucleotide (ASO) technology occurred in the late 1970s and was followed by the…”
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Antisense Oligonucleotide Therapies for Neurodegenerative Diseases by Bennett, C. Frank, Krainer, Adrian R, Cleveland, Don W

“…Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative…”
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RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform by BENNETT, C. Frank, SWAYZE, Eric E

“…Dramatic advances in understanding of the roles RNA plays in normal health and disease have greatly expanded over the past 10 years and have made it clear that…”
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Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides by Geary, Richard S., Norris, Daniel, Yu, Rosie, Bennett, C. Frank

“…Pharmacokinetic properties of oligonucleotides are largely driven by chemistry of the backbone and thus are sequence independent within a chemical class…”
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Selective tissue targeting of synthetic nucleic acid drugs by Seth, Punit P, Tanowitz, Michael, Bennett, C Frank

“…Antisense oligonucleotides (ASOs) are chemically synthesized nucleic acid analogs designed to bind to RNA by Watson-Crick base pairing. Following binding to…”
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RNA-Targeted Therapeutics by Crooke, Stanley T., Witztum, Joseph L., Bennett, C. Frank, Baker, Brenda F.

“…RNA-targeted therapies represent a platform for drug discovery involving chemically modified oligonucleotides, a wide range of cellular RNAs, and a novel…”
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Pharmacology of Antisense Drugs by Bennett, C. Frank, Baker, Brenda F, Pham, Nguyen, Swayze, Eric, Geary, Richard S

“…Recent studies have led to a greater appreciation of the diverse roles RNAs play in maintaining normal cellular function and how they contribute to disease…”
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THE 1% CONCORDANCE HUBBLE CONSTANT by Bennett, C L, Larson, D, Weiland, J L, Hinshaw, G

“…The determination of the Hubble constant has been a central goal in observational astrophysics for nearly a hundred years. Extraordinary progress has occurred…”
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Antisense Drugs Make Sense for Neurological Diseases by Bennett, C. Frank, Kordasiewicz, Holly B, Cleveland, Don W

“…The genetic basis for most inherited neurodegenerative diseases has been identified, yet there are limited disease-modifying therapies for these patients. A…”
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Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration by Melamed, Ze’ev, López-Erauskin, Jone, Baughn, Michael W., Zhang, Ouyang, Drenner, Kevin, Sun, Ying, Freyermuth, Fernande, McMahon, Moira A., Beccari, Melinda S., Artates, Jon W., Ohkubo, Takuya, Rodriguez, Maria, Lin, Nianwei, Wu, Dongmei, Bennett, C. Frank, Rigo, Frank, Da Cruz, Sandrine, Ravits, John, Lagier-Tourenne, Clotilde, Cleveland, Don W.

“…Amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) are associated with loss of nuclear transactive response DNA-binding protein 43 (TDP-43)…”
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Hybridization-mediated off-target effects of splice-switching antisense oligonucleotides by Scharner, Juergen, Ma, Wai Kit, Zhang, Qian, Lin, Kuan-Ting, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R

“…Abstract Splice-switching antisense oligonucleotides (ASOs), which bind specific RNA-target sequences and modulate pre-mRNA splicing by sterically blocking the…”
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Targeting Huntingtin Expression in Patients with Huntington’s Disease by Tabrizi, Sarah J, Leavitt, Blair R, Landwehrmeyer, G. Bernhard, Wild, Edward J, Saft, Carsten, Barker, Roger A, Blair, Nick F, Craufurd, David, Priller, Josef, Rickards, Hugh, Rosser, Anne, Kordasiewicz, Holly B, Czech, Christian, Swayze, Eric E, Norris, Daniel A, Baumann, Tiffany, Gerlach, Irene, Schobel, Scott A, Paz, Erika, Smith, Anne V, Bennett, C. Frank, Lane, Roger M

“…Mutated HTT , resulting in mutant huntingtin, causes Huntington’s disease. A phase 1–2a trial of intrathecal delivery of an antisense oligonucleotide targeting…”
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Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model by Hua, Yimin, Sahashi, Kentaro, Rigo, Frank, Hung, Gene, Horev, Guy, Bennett, C. Frank, Krainer, Adrian R.

“…Talking antisense: rescue of SMN2 in motor neurone disease Spinal muscular atrophy (SMA) is a motor neurone disease caused by a mutation in a gene called SMN1…”
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Towards a therapy for Angelman syndrome by targeting a long non-coding RNA by Meng, Linyan, Ward, Amanda J., Chun, Seung, Bennett, C. Frank, Beaudet, Arthur L., Rigo, Frank

“…Angelman syndrome is a neurodevelopmental disorder caused by disrupted function of the maternal copy of the imprinted UBE3A gene; here, targeting a long…”
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Biomedical Science Ph.D. Career Interest Patterns by Race/Ethnicity and Gender by Gibbs, Jr, Kenneth D, McGready, John, Bennett, Jessica C, Griffin, Kimberly

“…Increasing biomedical workforce diversity remains a persistent challenge. Recent reports have shown that biomedical sciences (BMS) graduate students become…”
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Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS by Miller, Timothy, Cudkowicz, Merit, Shaw, Pamela J, Andersen, Peter M, Atassi, Nazem, Bucelli, Robert C, Genge, Angela, Glass, Jonathan, Ladha, Shafeeq, Ludolph, Albert L, Maragakis, Nicholas J, McDermott, Christopher J, Pestronk, Alan, Ravits, John, Salachas, François, Trudell, Randall, Van Damme, Philip, Zinman, Lorne, Bennett, C. Frank, Lane, Roger, Sandrock, Alfred, Runz, Heiko, Graham, Danielle, Houshyar, Hani, McCampbell, Alexander, Nestorov, Ivan, Chang, Ih, McNeill, Manjit, Fanning, Laura, Fradette, Stephanie, Ferguson, Toby A

“…In a phase 1–2 dose-escalation trial involving adults with ALS due to SOD1 mutations who received intrathecal tofersen (an antisense oligonucleotide) or…”
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Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model by Sheng, Lei, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R, Hua, Yimin

“…Abstract Spinal muscular atrophy (SMA) is a motor neuron disease. Nusinersen, a splice-switching antisense oligonucleotide (ASO), was the first approved drug…”
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Artificial intelligence framework for simulating clinical decision-making: A Markov decision process approach by Bennett, Casey C, Hauser, Kris

“…Abstract Objective In the modern healthcare system, rapidly expanding costs/complexity, the growing myriad of treatment options, and exploding information…”
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Targeting RNA foci in iPSC-derived motor neurons from ALS patients with a C9ORF72 repeat expansion by Sareen, Dhruv, O'Rourke, Jacqueline G, Meera, Pratap, Muhammad, A K M G, Grant, Sharday, Simpkinson, Megan, Bell, Shaughn, Carmona, Sharon, Ornelas, Loren, Sahabian, Anais, Gendron, Tania, Petrucelli, Leonard, Baughn, Michael, Ravits, John, Harms, Matthew B, Rigo, Frank, Bennett, C Frank, Otis, Thomas S, Svendsen, Clive N, Baloh, Robert H

“…Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative condition characterized by loss of motor neurons in the brain and spinal cord. Expansions of…”
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Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models by Hua, Yimin, Liu, Ying Hsiu, Sahashi, Kentaro, Rigo, Frank, Bennett, C Frank, Krainer, Adrian R

“…Survival of motor neuron (SMN) deficiency causes spinal muscular atrophy (SMA), but the pathogenesis mechanisms remain elusive. Restoring SMN in motor neurons…”
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