Search Results - "Hastings, Michelle"

Splice-switching antisense oligonucleotides as therapeutic drugs by Havens, Mallory A, Hastings, Michelle L

“…Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic acids that base-pair with a pre-mRNA and disrupt the normal splicing…”
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Selective release of microRNA species from normal and malignant mammary epithelial cells by Pigati, Lucy, Yaddanapudi, Sree C S, Iyengar, Ravi, Kim, Dong-Ja, Hearn, Steven A, Danforth, David, Hastings, Michelle L, Duelli, Dominik M

“…MicroRNAs (miRNAs) in body fluids are candidate diagnostics for a variety of conditions and diseases, including breast cancer. One premise for using…”
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RNA therapeutics by Hastings, Michelle L, Krainer, Adrian R

“…"RNA therapeutics" refers to a disease treatment or drug that utilizes RNA as a component. In this context, RNA may be the direct target of a small-molecule…”
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Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c by Pan, Bifeng, Askew, Charles, Galvin, Alice, Heman-Ackah, Selena, Asai, Yukako, Indzhykulian, Artur A, Jodelka, Francine M, Hastings, Michelle L, Lentz, Jennifer J, Vandenberghe, Luk H, Holt, Jeffrey R, Géléoc, Gwenaëlle S

“…Gene therapy in the inner ear achieves unprecedented recovery of hearing and balance behavior in Usher syndrome mice. Because there are currently no biological…”
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Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells by Michaels, Wren E, Bridges, Robert J, Hastings, Michelle L

“…Abstract Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, encoding an…”
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Induction of Antagonistic Soluble Decoy Receptor Tyrosine Kinases by Intronic PolyA Activation by Vorlová, Sandra, Rocco, Gina, LeFave, Clare V., Jodelka, Francine M., Hess, Ken, Hastings, Michelle L., Henke, Erik, Cartegni, Luca

“…Alternative intronic polyadenylation (IPA) can generate truncated protein isoforms with significantly altered functions. Here, we describe 31…”
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Biogenesis of mammalian microRNAs by a non-canonical processing pathway by Havens, Mallory A, Reich, Ashley A, Duelli, Dominik M, Hastings, Michelle L

“…Canonical microRNA biogenesis requires the Microprocessor components, Drosha and DGCR8, to generate precursor-miRNA, and Dicer to form mature miRNA. The…”
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Elevating microRNA levels by targeting biogenesis with steric-blocking antisense oligonucleotides by Havens, Mallory A., Hinrich, Anthony J., Rigo, Frank, Hastings, Michelle L.

“…MicroRNAs (miRNAs) are regulators of gene expression, and their dysregulation is linked to cancer and other diseases, making them important therapeutic…”
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Open reading frame correction using splice-switching antisense oligonucleotides for the treatment of cystic fibrosis by Michaels, Wren E, Pena-Rasgado, Cecilia, Kotaria, Rusudan, Bridges, Robert J, Hastings, Michelle L

“…gene mutations that result in the introduction of premature termination codons (PTCs) are common in cystic fibrosis (CF). This mutation type causes a severe…”
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RNA localization and co‐translational interactions control RAB13 GTPase function and cell migration by Moissoglu, Konstadinos, Stueland, Michael, Gasparski, Alexander N, Wang, Tianhong, Jenkins, Lisa M, Hastings, Michelle L, Mili, Stavroula

“…Numerous RNAs exhibit specific distribution patterns in mammalian cells. However, the functional and mechanistic consequences are relatively unknown. Here, we…”
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SCRIB and PUF60 Are Primary Drivers of the Multisystemic Phenotypes of the 8q24.3 Copy-Number Variant by Dauber, Andrew, Golzio, Christelle, Guenot, Cécile, Jodelka, Francine M., Kibaek, Maria, Kjaergaard, Susanne, Leheup, Bruno, Martinet, Danielle, Nowaczyk, Malgorzata J.M., Rosenfeld, Jill A., Zeesman, Susan, Zunich, Janice, Beckmann, Jacques S., Hirschhorn, Joel N., Hastings, Michelle L., Jacquemont, Sebastien, Katsanis, Nicholas

“…Copy-number variants (CNVs) represent a significant interpretative challenge, given that each CNV typically affects the dosage of multiple genes. Here we…”
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Fetal gene therapy and pharmacotherapy to treat congenital hearing loss and vestibular dysfunction by Hastings, Michelle L., Brigande, John V.

“…Disabling hearing loss is expected to affect over 900 million people worldwide by 2050. The World Health Organization estimates that the annual economic impact…”
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MicroRNAs are exported from malignant cells in customized particles by Palma, Jaime, Yaddanapudi, Sree C, Pigati, Lucy, Havens, Mallory A, Jeong, Sarah, Weiner, Geoffrey A, Weimer, Kristina Mary Ellen, Stern, Brittany, Hastings, Michelle L, Duelli, Dominik M

“…MicroRNAs (miRNAs) are released from cells in association with proteins or microvesicles. We previously reported that malignant transformation changes the…”
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Targeting RNA splicing for disease therapy by Havens, Mallory A., Duelli, Dominik M., Hastings, Michelle L.

“…Splicing of pre‐messenger RNA into mature messenger RNA is an essential step for the expression of most genes in higher eukaryotes. Defects in this process…”
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Targeting SR proteins improves SMN expression in spinal muscular atrophy cells by Wee, Claribel D, Havens, Mallory A, Jodelka, Francine M, Hastings, Michelle L

“…Spinal muscular atrophy (SMA) is one of the most common inherited causes of pediatric mortality. SMA is caused by deletions or mutations in the survival of…”
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Antisense Oligonucleotides for the Treatment of Inner Ear Dysfunction by Hastings, Michelle L., Jones, Timothy A.

“…Antisense oligonucleotides (ASOs) have shown potential as therapeutic molecules for the treatment of inner ear dysfunction. The peripheral sensory organs…”
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Drosha promotes splicing of a pre-microRNA-like alternative exon by Havens, Mallory A, Reich, Ashley A, Hastings, Michelle L

“…The ribonuclease III enzyme Drosha has a central role in the biogenesis of microRNA (miRNA) by binding and cleaving hairpin structures in primary RNA…”
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Mitochondrial clearance and maturation of autophagosomes are compromised in LRRK2 G2019S familial Parkinson’s disease patient fibroblasts by Korecka, Joanna A, Thomas, Ria, Christensen, Dan P, Hinrich, Anthony J, Ferrari, Eliza J, Levy, Simon A, Hastings, Michelle L, Hallett, Penelope J, Isacson, Ole

“…Abstract This study utilized human fibroblasts as a preclinical discovery and diagnostic platform for identification of cell biological signatures specific for…”
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Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness by Lentz, Jennifer J, Jodelka, Francine M, Hinrich, Anthony J, McCaffrey, Kate E, Farris, Hamilton E, Spalitta, Matthew J, Bazan, Nicolas G, Duelli, Dominik M, Rigo, Frank, Hastings, Michelle L

“…Usher syndrome is a congenital form of deafness and vestibular dysfunction characterized by mutations in the USH1C gene encoding harmonin. Michelle L. Hastings…”
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Viral-like TLR3 induction of cytokine networks and α-synuclein are reduced by complement C3 blockade in mouse brain by Thomas, Ria, Connolly, Kyle J., Brekk, Oeystein R., Hinrich, Anthony J., Hastings, Michelle L., Isacson, Ole, Hallett, Penelope J.

“…Inflammatory processes and mechanisms are of central importance in neurodegenerative diseases. In the brain, α-synucleinopathies such as Parkinson’s disease…”
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