Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice-Reference-Cited by-同舟云学术

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Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice

Published:2024-02-01 Issue:1 Volume:34 Page:26-34
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Metz Tom¹^ORCID,Welling Mick M.²^ORCID,Suidgeest Ernst³,Nieuwenhuize Esmée¹,de Vlaam Thomas⁴,Curtis Daniel⁴,Hailu Tsinatkeab T.⁴,van der Weerd Louise¹³,van Roon-Mom Willeke M.C.¹

Affiliation:

1. Department of Human Genetics,Leiden University Medical Center, Leiden, The Netherlands.

2. Interventional Molecular Imaging Laboratory, Department of Radiology, Leiden University Medical Center, Leiden, The Netherlands.

3. Department of Radiology, Leiden University Medical Center, Leiden, The Netherlands.

4. Amylon Therapeutics, Leiden, The Netherlands.

Publisher

Mary Ann Liebert Inc

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2023.0018

Reference35 articles.

1. Intrathecal administration of nusinersen in adolescent and adult SMA type 2 and 3 patients

2. Antisense Oligonucleotide-Induced Amyloid Precursor Protein Splicing Modulation as a Therapeutic Approach for Dutch-Type Cerebral Amyloid Angiopathy

3. Targeting DMPK with Antisense Oligonucleotide Improves Muscle Strength in Myotonic Dystrophy Type 1 Mice

4. Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

5. A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration

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