Search Results - "Bennett, C. Frank"

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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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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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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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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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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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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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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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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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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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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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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Systematic screening identifies therapeutic antisense oligonucleotides for Hutchinson–Gilford progeria syndrome by Puttaraju, Madaiah, Jackson, Michaela, Klein, Stephanie, Shilo, Asaf, Bennett, C. Frank, Gordon, Leslie, Rigo, Frank, Misteli, Tom

“…Hutchinson–Gilford progeria syndrome (HGPS) is a rare, invariably fatal childhood premature aging disorder caused by a pre-messenger RNA (mRNA) splicing defect…”
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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study by Finkel, Richard S, Dr, Chiriboga, Claudia A, MD, Vajsar, Jiri, MD, Day, John W, MD, Montes, Jacqueline, EdD, De Vivo, Darryl C, MD, Yamashita, Mason, MD, Rigo, Frank, PhD, Hung, Gene, MD, Schneider, Eugene, MD, Norris, Daniel A, PhD, Xia, Shuting, MS, Bennett, C Frank, PhD, Bishop, Kathie M, PhD

“…Summary Background Nusinersen is a 2′- O -methoxyethyl phosphorothioate-modified antisense drug being developed to treat spinal muscular atrophy. Nusinersen is…”
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Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis by Kordasiewicz, Holly B., Stanek, Lisa M., Wancewicz, Edward V., Mazur, Curt, McAlonis, Melissa M., Pytel, Kimberly A., Artates, Jonathan W., Weiss, Andreas, Cheng, Seng H., Shihabuddin, Lamya S., Hung, Gene, Bennett, C. Frank, Cleveland, Don W.

“…The primary cause of Huntington's disease (HD) is expression of huntingtin with a polyglutamine expansion. Despite an absence of consensus on the mechanism(s)…”
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MaTAR25 lncRNA regulates the Tensin1 gene to impact breast cancer progression by Chang, Kung-Chi, Diermeier, Sarah D., Yu, Allen T., Brine, Lily D., Russo, Suzanne, Bhatia, Sonam, Alsudani, Habeeb, Kostroff, Karen, Bhuiya, Tawfiqul, Brogi, Edi, Pappin, Darryl J., Bennett, C. Frank, Rigo, Frank, Spector, David L.

“…Misregulation of long non-coding RNA (lncRNA) genes has been linked to a wide variety of cancer types. Here we report on Mammary Tumor Associated RNA 25 (…”
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Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration by Lagier-Tourenne, Clotilde, Baughn, Michael, Rigo, Frank, Sun, Shuying, Liu, Patrick, Li, Hai-Ri, Jiang, Jie, Watt, Andrew T, Chun, Seung, Katz, Melanie, Qiu, Jinsong, Sun, Ying, Ling, Shuo-Chien, Zhu, Qiang, Polymenidou, Magdalini, Drenner, Kevin, Artates, Jonathan W, McAlonis-Downes, Melissa, Markmiller, Sebastian, Hutt, Kasey R, Pizzo, Donald P, Cady, Janet, Harms, Matthew B, Baloh, Robert H, Vandenberg, Scott R, Yeo, Gene W, Fu, Xiang-Dong, Bennett, C Frank, Cleveland, Don W, Ravits, John

“…Expanded hexanucleotide repeats in the chromosome 9 open reading frame 72 (C9orf72) gene are the most common genetic cause of ALS and frontotemporal…”
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Antisense-based therapy for the treatment of spinal muscular atrophy by Rigo, Frank, Hua, Yimin, Krainer, Adrian R, Bennett, C Frank

“…One of the greatest thrills a biomedical researcher may experience is seeing the product of many years of dedicated effort finally make its way to the patient…”
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