RNA-targeted drugs for neuromuscular diseases-Reference-Cited by-同舟云学术

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RNA-targeted drugs for neuromuscular diseases

Published:2021-01 Issue:6524 Volume:371 Page:29-31
ISSN:0036-8075
Container-title:Science
language:en
Short-container-title:Science

Author:

Ferlini Alessandra¹²,Goyenvalle Aurelie³,Muntoni Francesco²⁴

Affiliation:

1. Unit of Medical Genetics, Department of Medical Science, University of Ferrara, Ferrara, Italy.

2. Dubowitz Neuromuscular Centre, UCL Great Ormond Street Institute of Child Health, University College London, London, UK.

3. Université Paris-Saclay, UVSQ, Inserm, END-ICAP, Versailles, France.

4. NIHR Great Ormond Street Hospital Biomedical Research Centre, London, UK.

Abstract

Progress with antisense oligonucleotide therapies opens a path for future development

Publisher

American Association for the Advancement of Science (AAAS)

Subject

Multidisciplinary

Reference15 articles.

1. Impending therapies for Duchenne muscular dystrophy

2. Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy

3. A randomized placebo-controlled phase 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy

4. Golodirsen: First Approval

5. Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

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1. Levels of Exon-Skipping Are Not Artificially Overestimated Because of the Increased Affinity of Tricyclo-DNA-Modified Antisense Oligonucleotides to the Target DMD Exon;Nucleic Acid Therapeutics;2024-07-24

2. RNA structure in alternative splicing regulation: from mechanism to therapy;Acta Biochimica et Biophysica Sinica;2024-07-01

3. A review on mechanistic insights into structure and function of dystrophin protein in pathophysiology and therapeutic targeting of Duchenne muscular dystrophy;International Journal of Biological Macromolecules;2024-04

4. Reformer: Deep learning model for characterizing protein-RNA interactions from sequence at single-base resolution;2024-01-15

5. Partial restoration of brain dystrophin by tricyclo-DNA antisense oligonucleotides alleviates emotional deficits in mdx52 mice;Molecular Therapy - Nucleic Acids;2023-06

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