Consensus Guidelines for the Design and <i>In Vitro</i> Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides-Reference-Cited by-同舟云学术

Consensus Guidelines for the Design and In Vitro Preclinical Efficacy Testing N-of-1 Exon Skipping Antisense Oligonucleotides

Published:2023-02-01 Issue:1 Volume:33 Page:17-25
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Aartsma-Rus Annemieke¹²³^ORCID,Garanto Alejandro¹⁴^ORCID,van Roon-Mom Willeke¹²^ORCID,McConnell Erin M.³,Suslovitch Victoria³⁵,Yan Winston X.³,Watts Jonathan K.⁶,Yu Timothy W.³⁵⁷,

Affiliation:

1. Dutch Center for RNA Therapeutics, the Netherlands.

2. Department of Human Genetics, Leiden University Medical Center, Leiden, the Netherlands.

3. N = 1 Collaborative.

4. Department of Pediatrics and Department of Human Genetics, Radboud Institute for Molecular Life Sciences, Radboud University Medical Center, Nijmegen, the Netherlands.

5. Department of Genetics and Genomics, Boston Children's Hospital, Boston, Massachusetts, USA.

6. RNA Therapeutics Institute, UMass Chan Medical School, Worcester, Massachusetts, USA.

7. Harvard Medical School, Division of Genetics and Genomics, Boston, Massachusetts, USA.

Publisher

Mary Ann Liebert Inc

Subject

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

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2022.0060

Reference30 articles.

1. Antisense technology: an overview and prospectus

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

3. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

4. Addressing the Needs of Patients with Ultra-Rare Mutations One Patient at a Time: The n-Lorem Approach

5. ‘N of 1’ therapies need a better model

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