Antisense oligonucleotide treatment for a pseudoexon-generating mutation in theNPC1gene causing Niemann-Pick type C disease-Reference-Cited by-同舟云学术

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Antisense oligonucleotide treatment for a pseudoexon-generating mutation in theNPC1gene causing Niemann-Pick type C disease

Published:2009-11 Issue:11 Volume:30 Page:E993-E1001
ISSN:1059-7794
Container-title:Human Mutation
language:en
Short-container-title:Hum. Mutat.

Author:

Rodríguez-Pascau Laura,Coll Maria Josep,Vilageliu Lluïsa,Grinberg Daniel

Publisher

Wiley

Subject

Genetics (clinical),Genetics

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/humu.21119/fullpdf

Cited by 45 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. Splice-Modulating Antisense Oligonucleotides as Therapeutics for Inherited Metabolic Diseases;BioDrugs;2024-01-22

2. Challenges in the Definitive Diagnosis of Niemann–Pick Type C—Leaky Variants and Alternative Transcripts;Genes;2023-10-25

3. Metabolic improvement in patients with Niemann-Pick disease following intravenous trehalose administration: An untargeted pharmacometabolomic study;2023-07-17

4. Emerging Therapies;Lysosomal Storage Disorders;2022-07-21

5. Therapeutic Strategies For Tay-Sachs Disease;Frontiers in Pharmacology;2022-07-05

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