Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics-Reference-Cited by-同舟云学术

Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics

Published:2023-08-01 Issue:4 Volume:33 Page:238-247
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Zhou Haiyan¹²^ORCID,Arechavala-Gomeza Virginia³⁴^ORCID,Garanto Alejandro⁵⁶^ORCID

Affiliation:

1. Genetics and Genomic Medicine Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, London, United Kingdom.

2. NIHR Great Ormond Street Hospital Biomedical Research Center, London, United Kingdom.

3. Nucleic Acid Therapeutics for Rare Disorders (NAT-RD), Biocruces Bizkaia Health Research Institute, Barakaldo, Spain.

4. Ikerbasque, Basque Foundation for Science, Bilbao, Spain.

5. Department of Pediatrics, Amalia Children's Hospital, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

6. Department of Human Genetics, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, The Netherlands.

Publisher

Mary Ann Liebert Inc

Subject

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

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2023.0001

Reference56 articles.

1. Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

2. Antisense Drugs Make Sense for Neurological Diseases

3. AON-based degradation of c.151C>T mutant COCH transcripts associated with dominantly inherited hearing impairment DFNA9

4. Splice-Modulating Oligonucleotide QR-110 Restores CEP290 mRNA and Function in Human c.2991+1655A>G LCA10 Models

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