Literature DB >> 31283897

Antisense Oligonucleotide Therapies for Neurodegenerative Diseases.

C Frank Bennett1, Adrian R Krainer2, Don W Cleveland3.   

Abstract

Antisense oligonucleotides represent a novel therapeutic platform for the discovery of medicines that have the potential to treat most neurodegenerative diseases. Antisense drugs are currently in development for the treatment of amyotrophic lateral sclerosis, Huntington's disease, and Alzheimer's disease, and multiple research programs are underway for additional neurodegenerative diseases. One antisense drug, nusinersen, has been approved for the treatment of spinal muscular atrophy. Importantly, nusinersen improves disease symptoms when administered to symptomatic patients rather than just slowing the progression of the disease. In addition to the benefit to spinal muscular atrophy patients, there are discoveries from nusinersen that can be applied to other neurological diseases, including method of delivery, doses, tolerability of intrathecally delivered antisense drugs, and the biodistribution of intrathecal dosed antisense drugs. Based in part on the early success of nusinersen, antisense drugs hold great promise as a therapeutic platform for the treatment of neurological diseases.

Entities:  

Keywords:  Huntington's disease; RNA; amyotrophic lateral sclerosis; antisense oligonucleotides; spinal muscular atrophy

Mesh：

Substances：

Year:  2019        PMID： 31283897      PMCID： PMC7427431          DOI： 10.1146/annurev-neuro-070918-050501

Source DB:  PubMed          Journal:  Annu Rev Neurosci        ISSN： 0147-006X            Impact factor:   12.449


  133 in total

1.  Associations between biomarkers and age in the presenilin 1 E280A autosomal dominant Alzheimer disease kindred: a cross-sectional study.

Authors:  Adam S Fleisher; Kewei Chen; Yakeel T Quiroz; Laura J Jakimovich; Madelyn Gutierrez Gomez; Carolyn M Langois; Jessica B S Langbaum; Auttawut Roontiva; Pradeep Thiyyagura; Wendy Lee; Napatkamon Ayutyanont; Liliana Lopez; Sonia Moreno; Claudia Muñoz; Victoria Tirado; Natalia Acosta-Baena; Anne M Fagan; Margarita Giraldo; Gloria Garcia; Matthew J Huentelman; Pierre N Tariot; Francisco Lopera; Eric M Reiman
Journal:  JAMA Neurol       Date:  2015-03       Impact factor: 18.302

2.  Tau Prion Strains Dictate Patterns of Cell Pathology, Progression Rate, and Regional Vulnerability In Vivo.

Authors:  Sarah K Kaufman; David W Sanders; Talitha L Thomas; Allison J Ruchinskas; Jaime Vaquer-Alicea; Apurwa M Sharma; Timothy M Miller; Marc I Diamond
Journal:  Neuron       Date:  2016-10-27       Impact factor: 17.173

3.  Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS.

Authors:  Cédric Raoul; Toufik Abbas-Terki; Jean-Charles Bensadoun; Sandrine Guillot; Georg Haase; Jolanta Szulc; Christopher E Henderson; Patrick Aebischer
Journal:  Nat Med       Date:  2005-03-13       Impact factor: 53.440

4.  Determinants of exon 7 splicing in the spinal muscular atrophy genes, SMN1 and SMN2.

Authors:  Luca Cartegni; Michelle L Hastings; John A Calarco; Elisa de Stanchina; Adrian R Krainer
Journal:  Am J Hum Genet       Date:  2005-11-16       Impact factor: 11.025

5.  Cortical efferents lacking mutant huntingtin improve striatal neuronal activity and behavior in a conditional mouse model of Huntington's disease.

Authors:  Ana María Estrada-Sánchez; Courtney L Burroughs; Stephen Cavaliere; Scott J Barton; Shirley Chen; X William Yang; George V Rebec
Journal:  J Neurosci       Date:  2015-03-11       Impact factor: 6.167

6.  A negative element in SMN2 exon 7 inhibits splicing in spinal muscular atrophy.

Authors:  Tsuyoshi Kashima; James L Manley
Journal:  Nat Genet       Date:  2003-08       Impact factor: 38.330

7.  Prospective cohort study of spinal muscular atrophy types 2 and 3.

Authors:  Petra Kaufmann; Michael P McDermott; Basil T Darras; Richard S Finkel; Douglas M Sproule; Peter B Kang; Maryam Oskoui; Andrei Constantinescu; Clifton L Gooch; A Reghan Foley; Michele L Yang; Rabi Tawil; Wendy K Chung; William B Martens; Jacqueline Montes; Vanessa Battista; Jessica O'Hagen; Sally Dunaway; Jean Flickinger; Janet Quigley; Susan Riley; Allan M Glanzman; Maryjane Benton; Patricia A Ryan; Mark Punyanitya; Megan J Montgomery; Jonathan Marra; Benjamin Koo; Darryl C De Vivo
Journal:  Neurology       Date:  2012-10-17       Impact factor: 9.910

8.  Quantitative analysis of survival motor neuron copies: identification of subtle SMN1 mutations in patients with spinal muscular atrophy, genotype-phenotype correlation, and implications for genetic counseling.

Authors:  B Wirth; M Herz; A Wetter; S Moskau; E Hahnen; S Rudnik-Schöneborn; T Wienker; K Zerres
Journal:  Am J Hum Genet       Date:  1999-05       Impact factor: 11.025

Review 9.  Amyotrophic lateral sclerosis.

Authors:  Orla Hardiman; Ammar Al-Chalabi; Adriano Chio; Emma M Corr; Giancarlo Logroscino; Wim Robberecht; Pamela J Shaw; Zachary Simmons; Leonard H van den Berg
Journal:  Nat Rev Dis Primers       Date:  2017-10-05       Impact factor: 52.329

10.  A hexanucleotide repeat expansion in C9ORF72 is the cause of chromosome 9p21-linked ALS-FTD.

Authors:  Alan E Renton; Elisa Majounie; Adrian Waite; Javier Simón-Sánchez; Sara Rollinson; J Raphael Gibbs; Jennifer C Schymick; Hannu Laaksovirta; John C van Swieten; Liisa Myllykangas; Hannu Kalimo; Anders Paetau; Yevgeniya Abramzon; Anne M Remes; Alice Kaganovich; Sonja W Scholz; Jamie Duckworth; Jinhui Ding; Daniel W Harmer; Dena G Hernandez; Janel O Johnson; Kin Mok; Mina Ryten; Danyah Trabzuni; Rita J Guerreiro; Richard W Orrell; James Neal; Alex Murray; Justin Pearson; Iris E Jansen; David Sondervan; Harro Seelaar; Derek Blake; Kate Young; Nicola Halliwell; Janis Bennion Callister; Greg Toulson; Anna Richardson; Alex Gerhard; Julie Snowden; David Mann; David Neary; Michael A Nalls; Terhi Peuralinna; Lilja Jansson; Veli-Matti Isoviita; Anna-Lotta Kaivorinne; Maarit Hölttä-Vuori; Elina Ikonen; Raimo Sulkava; Michael Benatar; Joanne Wuu; Adriano Chiò; Gabriella Restagno; Giuseppe Borghero; Mario Sabatelli; David Heckerman; Ekaterina Rogaeva; Lorne Zinman; Jeffrey D Rothstein; Michael Sendtner; Carsten Drepper; Evan E Eichler; Can Alkan; Ziedulla Abdullaev; Svetlana D Pack; Amalia Dutra; Evgenia Pak; John Hardy; Andrew Singleton; Nigel M Williams; Peter Heutink; Stuart Pickering-Brown; Huw R Morris; Pentti J Tienari; Bryan J Traynor
Journal:  Neuron       Date:  2011-09-21       Impact factor: 17.173
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  74 in total

Review 1.  Antisense technology: an overview and prospectus.

Authors:  Stanley T Crooke; Brenda F Baker; Rosanne M Crooke; Xue-Hai Liang
Journal:  Nat Rev Drug Discov       Date:  2021-03-24       Impact factor: 84.694

2.  Arginine-rich dipeptide-repeat proteins as phase disruptors in C9-ALS/FTD.

Authors:  Hana M Odeh; James Shorter
Journal:  Emerg Top Life Sci       Date:  2020-12-11

3.  Progress towards drug discovery for Friedreich's Ataxia: Identifying synthetic oligonucleotides that more potently activate expression of human frataxin protein.

Authors:  Xiulong Shen; Johnathan Wong; Thahza P Prakash; Frank Rigo; Yanjie Li; Marek Napierala; David R Corey
Journal:  Bioorg Med Chem       Date:  2020-04-05       Impact factor: 3.641

Review 4.  RNA-mediated toxicity in C9orf72 ALS and FTD.

Authors:  Zachary T McEachin; Janani Parameswaran; Nisha Raj; Gary J Bassell; Jie Jiang
Journal:  Neurobiol Dis       Date:  2020-08-21       Impact factor: 5.996

Review 5.  A survey of transcripts generated by spinal muscular atrophy genes.

Authors:  Natalia N Singh; Eric W Ottesen; Ravindra N Singh
Journal:  Biochim Biophys Acta Gene Regul Mech       Date:  2020-05-06       Impact factor: 4.490

6.  Aβ Plaques.

Authors:  Lary C Walker
Journal:  Free Neuropathol       Date:  2020-10-30

7.  Development of a novel severe mouse model of spinal muscular atrophy with respiratory distress type 1: FVB-nmd.

Authors:  Monir Shababi; Caley E Smith; Mona Kacher; Zayd Alrawi; Eric Villalón; Daniel Davis; Elizabeth C Bryda; Christian L Lorson
Journal:  Biochem Biophys Res Commun       Date:  2019-10-08       Impact factor: 3.575

Review 8.  Antisense technology: A review.

Authors:  Stanley T Crooke; Xue-Hai Liang; Brenda F Baker; Rosanne M Crooke
Journal:  J Biol Chem       Date:  2021-02-16       Impact factor: 5.157

Review 9.  Current Status of Antisense Oligonucleotide-Based Therapy in Neuromuscular Disorders.

Authors:  Flavien Bizot; Adeline Vulin; Aurélie Goyenvalle
Journal:  Drugs       Date:  2020-09       Impact factor: 9.546

10.  Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB.

Authors:  Roy Maimon; Carlos Chillon-Marinas; Cedric E Snethlage; Sarthak M Singhal; Melissa McAlonis-Downes; Karen Ling; Frank Rigo; C Frank Bennett; Sandrine Da Cruz; Thomas S Hnasko; Alysson R Muotri; Don W Cleveland
Journal:  Nat Neurosci       Date:  2021-06-03       Impact factor: 24.884
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