Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of DUX4-Reference-Cited by-同舟云学术

Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of DUX4

Published:2021-06-01 Issue:3 Volume:31 Page:201-207
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Falzarano Maria Sofia¹,Argenziano Monica²,Marsollier Anne Chalotte³,Mariot Virginie³,Rossi Davide²,Selvatici Rita¹,Dumonceaux Julie³,Cavalli Roberta²,Ferlini Alessandra¹⁴

Affiliation:

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

2. Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy.

3. NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom.

4. The Dubowitz Neuromuscular Centre, UCL Street Institute of Child Health, London, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Drug Discovery,Genetics,Molecular Biology,Molecular Medicine,Biochemistry

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2020.0862

Reference43 articles.

1. Therapies for rare diseases: therapeutic modalities, progress and challenges ahead

2. RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs

3. FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

4. The potential of antisense oligonucleotide therapies for inherited childhood lung diseases

5. Exon-skipping advances for Duchenne muscular dystrophy

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