Antisense Oligonucleotide - Therapies For ALS

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

September 9, 2020

Identification of antisense lncRNAs targeting GSK3β as a regulator in major depressive disorder

Epigenomics
Zhifen LiuKerang Zhang
September 1, 2020

Degradation of Toxic RNA in Myotonic Dystrophy Using Gapmer Antisense Oligonucleotides

Methods in Molecular Biology
Quynh Nguyen, Toshifumi Yokota
September 2, 2020
Review

The implication of long non-coding RNAs in the diagnosis, pathogenesis and drug resistance of pancreatic ductal adenocarcinoma and their possible therapeutic potential

Biochimica Et Biophysica Acta. Reviews on Cancer
Gouri PandyaManoj Garg
September 13, 2020

Synthesis of new riboflavin modified ODNs: Effect of riboflavin moiety on the G-quadruplex arrangement and stability

Bioorganic Chemistry
Valeria RomanucciGiovanni Di Fabio
September 2, 2020
Open Access

Functional skeletal muscle model derived from SOD1-mutant ALS patient iPSCs recapitulates hallmarks of disease progression

Scientific Reports
Agnes Badu-MensahJames J Hickman
September 18, 2020

Status Update in the Use of Cell Penetrating Peptides for the Delivery of Macromolecular Therapeutics

Expert Opinion on Biological Therapy
Kaido KurrikoffÜlo Langel
September 11, 2020

DNA triplex with conformationally locked sugar disintegrates to duplex: Insights from molecular simulations

Biochemical and Biophysical Research Communications
Pradeep Pant, Maria Fisher
September 18, 2020
Review

Cholesteryl ester transfer protein inhibitors in precision medicine

Clinica Chimica Acta; International Journal of Clinical Chemistry
Xin SuDong Chang
September 16, 2020
Open Access

Aggregate-selective antibody attenuates seeded aggregation but not spontaneously evolving disease in SOD1 ALS model mice

Acta Neuropathologica Communications
Manuela LehmannUlrika Nordström
September 17, 2020
Open Access

Mouse Ataxin-2 Expansion Downregulates CamKII and Other Calcium Signaling Factors, Impairing Granule-Purkinje Neuron Synaptic Strength

International Journal of Molecular Sciences
Aleksandar ArsovićGeorg Auburger
September 5, 2020

DNA Analogues Modified at the Nonlinking Positions of Phosphorus

Accounts of Chemical Research
Pawan Kumar, Marvin H Caruthers
September 1, 2020

Development of Antisense Oligonucleotide Gapmers for the Treatment of Huntington's Disease

Methods in Molecular Biology
Tejal Aslesh, Toshifumi Yokota
September 11, 2020

Emerging Therapies in Friedreich's Ataxia

Expert Review of Neurotherapeutics
Theresa A ZesiewiczJoshua Vega
September 4, 2020
Open Access

C9orf72 poly(GR) aggregation induces TDP-43 proteinopathy

Science Translational Medicine
Casey N CookLeonard Petrucelli
September 11, 2020
Open Access

Natural display of nuclear-encoded RNA on the cell surface and its impact on cell interaction

Genome Biology
Norman HuangSheng Zhong
September 2, 2020
Open Access

A clinical report of the massive CAG repeat expansion in spinocerebellar ataxia type 2: Severe onset in a Mexican child and review previous cases

Genetics and Molecular Biology
José Sergio Alberto Ramirez-GarciaDiana García-Cruz
September 5, 2020
Review

Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy

Pediatric Pulmonology
Nicolas J Abreu, Megan A Waldrop

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