Emerging antisense oligonucleotide and viral therapies for amyotrophic lateral sclerosis

Current Opinion in Neurology
Cindy V Ly, Timothy M Miller

Abstract

Amyotrophic lateral sclerosis (ALS) is a rapidly fatal disease for which there is currently no effective therapy. The present review describes the current progress of existing molecular therapies in the clinical trial pipeline and highlights promising future antisense oligonucleotide (ASO) and viral therapeutic strategies for treating ALS. The immense progress in the design of clinical trials and generation of ASO therapies directed towards superoxide dismutase-1 (SOD1) and chromosome 9 open reading frame 72 (C9orf72) repeat expansion related disease have been propelled by fundamental work to identify the genetic underpinnings of familial ALS and develop relevant disease models. Preclinical studies have also identified promising targets for sporadic ALS (sALS). Moreover, encouraging results in adeno-associated virus (AAV)-based therapies for spinal muscular atrophy (SMA) provide a roadmap for continued improvement in delivery and design of molecular therapies for ALS. Advances in preclinical and clinical studies of ASO and viral directed approaches to neuromuscular disease, particularly ALS, indicate that these approaches have high specificity and are relatively well tolerated.

References

Apr 27, 2005·Annals of Neurology·Timothy M MillerDon W Cleveland
Nov 19, 2005·Biochemical and Biophysical Research Communications·Tim-Rasmus KiehlStefan-M Pulst
Aug 1, 2006·The Journal of Clinical Investigation·Richard A SmithDon W Cleveland
Aug 23, 2006·Nature Reviews. Neuroscience·Piera Pasinelli, Robert H Brown
Mar 1, 2008·Science·Jemeen SreedharanChristopher E Shaw
Dec 23, 2008·Nature Biotechnology·Kevin D FoustBrian K Kaspar
Dec 31, 2009·The Journal of Biological Chemistry·Chantelle F SephtonGang Yu
Feb 6, 2010·Proceedings of the National Academy of Sciences of the United States of America·Hans WilsSamir Kumar-Singh
Aug 25, 2012·Neurology Research International·Dan KrakoraMasatoshi Suzuki
Aug 13, 2013·Neuron·Shuo-Chien LingDon W Cleveland
Sep 7, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Kevin D FoustBrian K Kaspar
Oct 22, 2013·Neuron·Christopher J DonnellyJeffrey D Rothstein
Oct 25, 2013·Science Translational Medicine·Dhruv SareenRobert H Baloh
Oct 31, 2013·Proceedings of the National Academy of Sciences of the United States of America·Clotilde Lagier-TourenneJohn Ravits
Oct 7, 2014·Frontiers in Molecular Neuroscience·Giridhar MurlidharanAravind Asokan
Jun 16, 2015·The Journal of Clinical Investigation·Matthew J CrispTimothy M Miller
Dec 30, 2015·Human Gene Therapy·Florie BorelChristian Mueller
Jun 5, 2016·Journal of Neurology, Neurosurgery, and Psychiatry·Taha BaliTimothy M Miller
Jul 13, 2016·Acta Neuropathologica Communications·Vincent Picher-MartelNicolas Dupré
Jul 15, 2016·Science Translational Medicine·Aaron BurberryKevin Eggan
Sep 7, 2016·Current Opinion in Virology·Arun Srivastava
Dec 28, 2016·Neurochemical Research·Wan WangChunyan Li
Feb 9, 2017·Annals of Clinical and Translational Neurology·Ashley E FrakesBrian K Kaspar
Mar 31, 2017·Science Translational Medicine·Tania F GendronLeonard Petrucelli
May 12, 2017·EMBO Molecular Medicine·Sadanori MiyoshiYuji Yamanashi
Jul 1, 2017·Molecular Therapy : the Journal of the American Society of Gene Therapy·Maria Grazia BiferiMartine Barkats
Nov 2, 2017·The New England Journal of Medicine·Jerry R MendellBrian K Kaspar
Dec 23, 2017·Frontiers in Molecular Neuroscience·Andrew P Tosolini, James N Sleigh
Apr 10, 2018·Cell·Ke ZhangThomas E Lloyd
May 2, 2018·Annual Review of Neuroscience·Claire N BedbrookViviana Gradinaru

❮ Previous
Next ❯

Citations

Jan 16, 2019·Neurotherapeutics : the Journal of the American Society for Experimental NeuroTherapeutics·Anna M Krichevsky, Erik J Uhlmann
Mar 26, 2019·Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration·Ashley CrookDominic B Rowe
May 24, 2019·Neurology. Genetics·Daniel R ScolesStefan M Pulst
Nov 2, 2019·Alzheimer's & Dementia : the Journal of the Alzheimer's Association·Adam L BoxerNadine Tatton
Feb 23, 2020·Frontiers in Neuroscience·Frances TheunissenPatrick Anthony Akkari
Oct 8, 2019·The Journal of Clinical Investigation·Daniel M RamosCharlotte J Sumner
Dec 16, 2020·Gene Therapy·Burak BerberCihan Tastan
Jan 9, 2021·European Journal of Human Genetics : EJHG·Rebecca BorgRuben J Cauchi
Feb 8, 2021·Biochimica Et Biophysica Acta. Molecular Cell Research·April L Darling, James Shorter
Jul 29, 2020·Clinics in Laboratory Medicine·Jennifer Roggenbuck, Jamie C Fong
Mar 15, 2019·Journal of the Neurological Sciences·Stéphane MathisGwendal Le Masson
Apr 12, 2021·Molecular Therapy : the Journal of the American Society of Gene Therapy·Defne A Amado, Beverly L Davidson
Apr 27, 2021·Annual Review of Chemical and Biomolecular Engineering·Walter ThavarajahJulius B Lucks
Jul 6, 2021·Frontiers in Genetics·Eleanor G SeabyAnne O'Donnell-Luria
Aug 6, 2021·Frontiers in Cellular Neuroscience·Benjamin L Zaepfel, Jeffrey D Rothstein
Jul 29, 2021·Annals of Clinical and Translational Neurology·Fabiola PuentesAndrea Malaspina
Jan 1, 2020·Molecular Therapy. Methods & Clinical Development·David L HaggertyBrady Atwood

❮ Previous
Next ❯

Related Concepts

Related Feeds

Cell-Type Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Cell-Type-Specific Viral Vectors (ASM)

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Antisense Oligonucleotides: ND

This feed focuses on antisense oligonucleotide therapies such as Inotersen, Nusinursen, and Patisiran, in neurodegenerative diseases including amyotrophic lateral sclerosis.

ALS: Genetics

Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder characterized by muscle weakness. Here is the latest research investigating genetic alterations in this genetically heterogeneous disorder.

ALS: Genetics

Amyotrophic Lateral Sclerosis (ALS) is a progressive neurodegenerative disorder characterized by muscle weakness. ALS is a genetically heterogeneous disorder with several causative genes. Here are the latest discoveries pertaining to the genetics of this disease.

Cell-Type-Specific Viral Vectors

Viral vectors are used in biological research and therapy to deliver genetic material into cells. However, the efficiency of viral vectors varies depending on the cell type. Here is the latest research on cell-type-specific viral vectors.

Amyloid Lateral Sclerosis

Amyotrophic Lateral Sclerosis (ALS) is a progressive nervous system disease associated with the death of neurons that control voluntary muscles. Discover the latest research on ALS here.