Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice-Reference-Cited by-同舟云学术

Prenatal delivery of a therapeutic antisense oligonucleotide achieves broad biodistribution in the brain and ameliorates Angelman syndrome phenotype in mice

Published:2024-04 Issue:4 Volume:32 Page:935-951
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Clarke Maria T.,Remesal Laura,Lentz Lea,Tan Danielle J.,Young David,Thapa Slesha,Namuduri Shalini R.,Borges Beltran,Kirn Georgia,Valencia Jasmine,Lopez Manuel E.,Lui Jan H.,Shiow Lawrence R.,Dindot Scott,Villeda Saul,Sanders Stephan J.,MacKenzie Tippi C.^ORCID

Funder

UCSF School of Medicine

Publisher

Elsevier BV

Reference51 articles.

1. Prenatal Somatic Cell Gene Therapies: Charting a Path Toward Clinical Applications (Proceedings of the CERSI-FDA Meeting);Herzeg;J. Clin. Pharmacol.,2022

2. Prenatal Gene Therapy;Schwab;Clin. Obstet. Gynecol.,2021

3. Antisense oligonucleotides: the next frontier for treatment of neurological disorders;Rinaldi;Nat. Rev. Neurol.,2018

4. Antisense oligonucleotides delivered to the amniotic cavity in utero modulate gene expression in the postnatal mouse;Depreux;Nucleic Acids Res.,2016

5. Fetal antisense oligonucleotide therapy for congenital deafness and vestibular dysfunction;Wang;Nucleic Acids Res.,2020

Cited by 4 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. The Conundrum of Mechanics Versus Genetics in Congenital Hydrocephalus and Its Implications for Fetal Therapy Approaches: A Scoping Review;Prenatal Diagnosis;2024-09

2. Ube3a unsilencer for the potential treatment of Angelman syndrome;Nature Communications;2024-07-08

3. Ubiquitin system mutations in neurological diseases;Trends in Biochemical Sciences;2024-07

4. Forestwalk: A machine learning workflow brings new insights into posture and balance in rodent beam walking;2024-04-29