Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria-Reference-Cited by-同舟云学术

登录注册会员服务联系我们

Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria

Published:2024-06-01 Issue:3 Volume:34 Page:134-142
ISSN:2159-3337
Container-title:Nucleic Acid Therapeutics
language:en
Short-container-title:Nucleic Acid Therapeutics

Author:

Martínez-Pizarro Ainhoa¹,Álvarez Mar¹,Dembic Maja²,Lindegaard Caroline A.²,Castro Margarita³,Richard Eva¹,Andresen Brage S.²,Desviat Lourdes R.¹^ORCID

Affiliation:

1. Centro de Biología Molecular Severo Ochoa UAM-CSIC, IUBM, CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.

2. Department of Biochemistry and Molecular Biology, University of Southern Denmark, Odense, Denmark.

3. Centro de Diagnóstico de Enfermedades Moleculares (CEDEM), CIBERER, IdiPaz, Universidad Autónoma de Madrid, Madrid, Spain.

Publisher

Mary Ann Liebert Inc

Link

https://www.liebertpub.com/doi/pdf/10.1089/nat.2024.0014

Reference39 articles.

1. Therapeutic Antisense Oligonucleotides Are Coming of Age

2. Splice-switching antisense oligonucleotides as therapeutic drugs

3. Targeting RNA with synthetic oligonucleotides: Clinical success invites new challenges

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

5. Chemistry, structure and function of approved oligonucleotide therapeutics

同舟云学术

1.学者识别学者识别

2.学术分析学术分析

3.人才评估人才评估

"同舟云学术"是以全球学者为主线，采集、加工和组织学术论文而形成的新型学术文献查询和分析系统，可以对全球学者进行文献检索和人才价值评估。用户可以通过关注某些学科领域的顶尖人物而持续追踪该领域的学科进展和研究前沿。经过近期的数据扩容，当前同舟云学术共收录了国内外主流学术期刊6万余种，收集的期刊论文及会议论文总量共计约1.5亿篇，并以每天添加12000余篇中外论文的速度递增。我们也可以为用户提供个性化、定制化的学者数据。欢迎来电咨询！咨询电话：010-8811{复制后删除}0370

www.globalauthorid.com

TOP