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.

June 26, 2020
Open Access

A systems approach delivers a functional microRNA catalog and expanded targets for seizure suppression in temporal lobe epilepsy

Proceedings of the National Academy of Sciences of the United States of America
Morten T VenøDavid C Henshall
June 21, 2020
Review

Long non-coding RNA in cervical cancer: From biology to therapeutic opportunity

Biomedicine & Pharmacotherapy = Biomédecine & Pharmacothérapie
Jiaxing HeTianmin Xu
July 4, 2020
Review

The diagnostic challenges of cardiac amyloidosis: A practical approach to the two main types

Blood Reviews
Cindy VargaRaymond L Comenzo
June 24, 2020
Review

The emerging role of angiotensinogen in cardiovascular diseases

Journal of Cellular Physiology
Yinchuan XuZhaocai Zhang
June 26, 2020
Review
Open Access

The Mechanisms of Nuclear Proteotoxicity in Polyglutamine Spinocerebellar Ataxias

Frontiers in Neuroscience
Davin LeeSung Bae Lee
July 4, 2020

A role for alternative splicing in circadian control of exocytosis and glucose homeostasis

Genes & Development
Biliana MarchevaJoseph Bass
June 23, 2020

Silencing of MYH7 ameliorates disease phenotypes in human iPSC-Cardiomyocytes

Physiological Genomics
Alexandra DainisEuan A Ashley
July 2, 2020

Suppression of proteolipid protein rescues Pelizaeus-Merzbacher disease

Nature
Matthew S ElittPaul J Tesar
July 4, 2020
Preprint

CRISPR/Cas9-based silencing of the ATXN1 gene in Spinocerebellar ataxia type 1 (SCA1) fibroblasts

BioRxiv : the Preprint Server for Biology
F. SalvatoriPeggy Marconi
June 21, 2020
Review
Open Access

Therapeutic siRNA: state of the art

Signal Transduction and Targeted Therapy
Bo HuXing-Jie Liang
July 7, 2020
Open Access

CARMAL Is a Long Non-coding RNA Locus That Regulates MFGE8 Expression

Frontiers in Genetics
Sébastien SoubeyrandRuth McPherson
July 2, 2020
Open Access

SnoRNA signatures in cartilage ageing and osteoarthritis

Scientific Reports
Mandy J PeffersTim J M Welting
July 4, 2020

Quantum chemical topology at the spin-orbit configuration interaction level: Application to astatine compounds

Journal of Computational Chemistry
Cecilia Gomez PechRémi Maurice
June 27, 2020
Review

Overview of treatments used in transthyretin-related hereditary amyloidosis: a systematic review

European Journal of Hospital Pharmacy. Science and Practice
Héctor Cristóbal GutiérrezMarta Valero Domínguez
July 7, 2020

Understanding and therapeutic targeting of aberrant mRNA splicing mechanisms in oncogenesis

[Rinshō ketsueki] The Japanese journal of clinical hematology
Atsushi TanakaDaichi Inoue
June 26, 2020

Preclinical Nanomedicines for Polyglutamine-Based Neurodegenerative Diseases

Molecular Pharmaceutics
Leo Kit Cheung LeeChung Hang Jonathan Choi
July 7, 2020
Review

Prevention of heart failure with preserved ejection fraction (HFpEF): reexamining microRNA-21 inhibition in the era of oligonucleotide-based therapeutics

Cardiovascular Pathology : the Official Journal of the Society for Cardiovascular Pathology
David Ben-NunFrancisco Fuentes
June 25, 2020

TTR gene silencing therapy in post liver transplant hereditary ATTR amyloidosis patients

Amyloid : the International Journal of Experimental and Clinical Investigation : the Official Journal of the International Society of Amyloidosis
Orly Moshe-LilieChafic Karam

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