Frank Rigo

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Frank Rigo

Publications by authors named "Frank Rigo"

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66Publications

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Targeting Amyloid-β Precursor Protein, APP, Splicing with Antisense Oligonucleotides Reduces Toxic Amyloid-β Production.

Mol Ther 2018 Jun 6;26(6):1539-1551. Epub 2018 Mar 6.

Department of Cell Biology and Anatomy, Center for Genetic Diseases, Chicago Medical School and School of Graduate and Postdoctoral Studies, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, USA. Electronic address:

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June 2018

Stress Granule Assembly Disrupts Nucleocytoplasmic Transport.

Cell 2018 May 5;173(4):958-971.e17. Epub 2018 Apr 5.

Department of Neurology, School of Medicine, Johns Hopkins University, Baltimore, MD 21205, USA; Cellular and Molecular Medicine Program, School of Medicine, Johns Hopkins University, Baltimore, MD 21205, USA; Solomon H. Snyder Department of Neuroscience, School of Medicine, Johns Hopkins University, Baltimore, MD 21205, USA. Electronic address:

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May 2018

Murine knockin model for progranulin-deficient frontotemporal dementia with nonsense-mediated mRNA decay.

Proc Natl Acad Sci U S A 2018 Mar 6;115(12):E2849-E2858. Epub 2018 Mar 6.

Department of Genetics and Complex Diseases, Harvard T. H. Chan School of Public Health, Boston, MA 02115;

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March 2018

Rescue of Outer Hair Cells with Antisense Oligonucleotides in Usher Mice Is Dependent on Age of Treatment.

J Assoc Res Otolaryngol 2018 Feb 12;19(1):1-16. Epub 2017 Oct 12.

Neuroscience Center of Excellence, Louisiana State University Health Sciences Center, 2020 Gravier Street, 8th Floor, New Orleans, LA, 70112, USA.

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February 2018

Downregulation of Survivin contributes to cell-cycle arrest during postnatal cardiac development in a severe spinal muscular atrophy mouse model.

Hum Mol Genet 2018 Feb;27(3):486-498

Jiangsu Key Laboratory of Neuropsychiatric Diseases, The Second Affiliated Hospital of Soochow University, Suzhou, Jiangsu 215004, China.

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February 2018

Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat.

Nucleic Acid Ther 2018 Feb 17;28(1):23-33. Epub 2018 Jan 17.

1 Department of Pharmacology, UT Southwestern Medical Center at Dallas , Dallas, Texas.

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February 2018

Rescue of peripheral vestibular function in Usher syndrome mice using a splice-switching antisense oligonucleotide.

Hum Mol Genet 2017 09;26(18):3482-3494

Department of Cell Biology and Anatomy, The Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, USA.

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September 2017

Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice.

Mol Ther Nucleic Acids 2017 Sep 29;8:232-242. Epub 2017 Jun 29.

Department of Human Genetics, Leiden University Medical Center, Albinusdreef 2, 2333ZA Leiden, the Netherlands. Electronic address:

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September 2017

Recognition of c9orf72 Mutant RNA by Single-Stranded Silencing RNAs.

Nucleic Acid Ther 2017 04 22;27(2):87-94. Epub 2016 Dec 22.

1 Department of Pharmacology, UT Southwestern Medical Center at Dallas , Dallas, Texas.

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April 2017

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.

Nature 2017 04 12;544(7650):362-366. Epub 2017 Apr 12.

Department of Neurology, University of Utah, 175 North Medical Drive East, 5th Floor, Salt Lake City, Utah 84132, USA.

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April 2017

Disrupted neuronal maturation in Angelman syndrome-derived induced pluripotent stem cells.

Nat Commun 2017 Apr 24;8:15038. Epub 2017 Apr 24.

Department of Neuroscience, University of Connecticut School of Medicine, 263 Farmington Avenue, Farmington, Connecticut 06030, USA.

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April 2017

The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy.

Neuron 2017 Jan 22;93(1):66-79. Epub 2016 Dec 22.

Department of Neurology, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA; Department of Neuroscience, Johns Hopkins University School of Medicine, 855 North Wolfe Street, Baltimore, MD 21205, USA. Electronic address:

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January 2017

RNA-binding protein CPEB1 remodels host and viral RNA landscapes.

Nat Struct Mol Biol 2016 Dec 24;23(12):1101-1110. Epub 2016 Oct 24.

Department of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, California, USA.

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December 2016

Antisense oligonucleotides delivered to the amniotic cavity in utero modulate gene expression in the postnatal mouse.

Nucleic Acids Res 2016 Nov 28;44(20):9519-9529. Epub 2016 Sep 28.

Department of Cell Biology and Anatomy, Chicago Medical School, Rosalind Franklin University of Medicine and Science, North Chicago, IL 60064, USA

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November 2016

Protein-RNA Networks Regulated by Normal and ALS-Associated Mutant HNRNPA2B1 in the Nervous System.

Neuron 2016 Nov 20;92(4):780-795. Epub 2016 Oct 20.

Department of Cellular and Molecular Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Stem Cell Program and Institute for Genomic Medicine, University of California, San Diego, La Jolla, CA 92093, USA; Department of Bioinformatics and Systems Biology, University of California, San Diego, La Jolla, CA 92093, USA; Department of Physiology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 117597, Singapore; Molecular Engineering Laboratory, A(∗)STAR, Singapore 138673, Singapore. Electronic address:

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November 2016

The Power of Human Protective Modifiers: PLS3 and CORO1C Unravel Impaired Endocytosis in Spinal Muscular Atrophy and Rescue SMA Phenotype.

Am J Hum Genet 2016 Sep 4;99(3):647-665. Epub 2016 Aug 4.

Institute of Human Genetics, University of Cologne, 50931 Cologne Germany; Institute for Genetics, University of Cologne, 50674 Cologne, Germany; Center for Molecular Medicine Cologne, University of Cologne, 50931 Cologne, Germany. Electronic address:

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September 2016

Mammary Tumor-Associated RNAs Impact Tumor Cell Proliferation, Invasion, and Migration.

Cell Rep 2016 09;17(1):261-274

Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724, USA; Molecular and Cellular Biology Program, Stony Brook University, Stony Brook, NY 11794, USA. Electronic address:

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September 2016

Increased 4R-Tau Induces Pathological Changes in a Human-Tau Mouse Model.

Neuron 2016 06 19;90(5):941-7. Epub 2016 May 19.

Department of Neurology, Hope Center for Neurological Disorders, Washington University in St. Louis, St. Louis, MO 63110. Electronic address:

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June 2016

Antisense oligonucleotide-directed inhibition of nonsense-mediated mRNA decay.

Nat Biotechnol 2016 Feb 14;34(2):164-6. Epub 2015 Dec 14.

Cold Spring Harbor Laboratory, Cold Spring Harbor, New York, USA.

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February 2016

Reversal of phenotypes in MECP2 duplication mice using genetic rescue or antisense oligonucleotides.

Nature 2015 Dec 25;528(7580):123-6. Epub 2015 Nov 25.

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, Texas 77030, USA.

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December 2015

DICER/AGO-dependent epigenetic silencing of D4Z4 repeats enhanced by exogenous siRNA suggests mechanisms and therapies for FSHD.

Hum Mol Genet 2015 Sep 3;24(17):4817-28. Epub 2015 Jun 3.

Division of Human Biology, Fred Hutchinson Cancer Research Center, Seattle, WA 98109, USA,

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September 2015

Regulation of the ESC transcriptome by nuclear long noncoding RNAs.

Genome Res 2015 Sep 5;25(9):1336-46. Epub 2015 Jun 5.

Cold Spring Harbor Laboratory, Cold Spring Harbor, New York 11724, USA;

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September 2015

Poly(A) Signal-Dependent Transcription Termination Occurs through a Conformational Change Mechanism that Does Not Require Cleavage at the Poly(A) Site.

Mol Cell 2015 Aug 9;59(3):437-48. Epub 2015 Jul 9.

Department of Chemistry and Biochemistry and the Molecular Biology Institute, University of California, Los Angeles, Los Angeles, CA 90095, USA. Electronic address:

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August 2015

Effects on murine behavior and lifespan of selectively decreasing expression of mutant huntingtin allele by supt4h knockdown.

PLoS Genet 2015 Mar 11;11(3):e1005043. Epub 2015 Mar 11.

Institute of Biochemistry and Molecular Biology, National Yang-Ming University, Taipei, Taiwan, Republic of China; Brain Research Center, National Yang-Ming University, Taipei, Taiwan, Republic of China.

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March 2015

Towards a therapy for Angelman syndrome by targeting a long non-coding RNA.

Nature 2015 Feb 1;518(7539):409-12. Epub 2014 Dec 1.

Department of Core Antisense Research, Isis Pharmaceuticals, Carlsbad, California 92010, USA.

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February 2015

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models.

Genes Dev 2015 Feb 12;29(3):288-97. Epub 2015 Jan 12.

Cold Spring Harbor Laboratory, Cold Spring Harbor, New York 11724, USA;

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February 2015

Antisense oligonucleotide-based therapies for diseases caused by pre-mRNA processing defects.

Adv Exp Med Biol 2014 ;825:303-52

Isis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA, USA,

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December 2014

Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.

J Pharmacol Exp Ther 2014 Jul 30;350(1):46-55. Epub 2014 Apr 30.

Isis Pharmaceuticals, Carlsbad, California (F.R., S.J.C., D.A.N., G.H., S.L., J.M., R.A.F., H.G., J.S.G., S.P.H., C.F.B.); and Cold Spring Harbor Laboratory, Cold Spring Harbor, New York (Y.H., A.R.K.).

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July 2014

Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides.

Nucleic Acids Res 2014 May 3;42(9):5871-9. Epub 2014 Mar 3.

Department of Core Antisense Research, ISIS Pharmaceuticals, Inc., Carlsbad, CA 92010, USA

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May 2014

RNA toxicity from the ALS/FTD C9ORF72 expansion is mitigated by antisense intervention.

Neuron 2013 Oct;80(2):415-28

Department of Neurology, Johns Hopkins University, 855 N Wolfe Street, Rangos 2-270, Baltimore, MD 21205, USA; Brain Science Institute, Johns Hopkins University, 855 N Wolfe Street, Rangos 2-270, Baltimore, MD 21205, USA.

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October 2013

Antisense reduction of tau in adult mice protects against seizures.

J Neurosci 2013 Jul;33(31):12887-97

Department of Neurology, Hope Center for Neurological Disorders, Washington University, St Louis, Missouri 63110, USA.

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July 2013

Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness.

Nat Med 2013 Mar 4;19(3):345-50. Epub 2013 Feb 4.

Neuroscience Center and Department of Otorhinolaryngology & Biocommunications, Louisiana State University Health Sciences Center (LSUHSC), New Orleans, Louisiana, USA.

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March 2013

Antisense-based therapy for the treatment of spinal muscular atrophy.

J Cell Biol 2012 Oct;199(1):21-5

Isis Pharmaceuticals, Carlsbad, CA 92010, USA.

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October 2012

Manipulation of PK-M mutually exclusive alternative splicing by antisense oligonucleotides.

Open Biol 2012 Oct;2(10):120133

Cold Spring Harbor Laboratory, Cold Spring Harbor, NY 11724 , USA; Watson School of Biological Sciences, Cold Spring Harbor, NY 11724, USA.

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October 2012

Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing.

Nat Chem Biol 2012 Apr 15;8(6):555-61. Epub 2012 Apr 15.

Isis Pharmaceuticals, Carlsbad, California, USA.

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April 2012

Peripheral SMN restoration is essential for long-term rescue of a severe spinal muscular atrophy mouse model.

Nature 2011 Oct 5;478(7367):123-6. Epub 2011 Oct 5.

Cold Spring Harbor Laboratory, PO Box 100, Cold Spring Harbor, New York 11724, USA.

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October 2011

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model.

Genes Dev 2010 Aug 12;24(15):1634-44. Epub 2010 Jul 12.

Cold Spring Harbor Laboratory, Cold Spring Harbor, New York 11724, USA.

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August 2010

Polyadenylation releases mRNA from RNA polymerase II in a process that is licensed by splicing.

RNA 2009 May 20;15(5):823-36. Epub 2009 Mar 20.

Department of Chemistry and Biochemistry, University of California at Los Angeles, Los Angeles, California 90095-1569, USA.

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May 2009

Functional coupling of last-intron splicing and 3'-end processing to transcription in vitro: the poly(A) signal couples to splicing before committing to cleavage.

Mol Cell Biol 2008 Jan 29;28(2):849-62. Epub 2007 Oct 29.

Department of Chemistry and Biochemistry, University of California at Los Angeles, Los Angeles, CA 90095-1569, USA.

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January 2008

The RNA tether from the poly(A) signal to the polymerase mediates coupling of transcription to cleavage and polyadenylation.

Mol Cell 2005 Dec;20(5):733-45

Department of Chemistry and Biochemistry and The Molecular Biology Institute, University of California, Los Angeles, 90095, USA.

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December 2005