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Updated: Jun 29, 2025

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts
07:02

Evaluation of Exon Inclusion Induced by Splice Switching Antisense Oligonucleotides in SMA Patient Fibroblasts

Published on: May 11, 2018

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拼接切换反意义的寡核化物 正确的氨酸氧化酶外子11跳过缺陷和救援酶活性在基尿症

Ainhoa Martínez-Pizarro1, Mar Álvarez1, Maja Dembic2

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

Nucleic acid therapeutics
|April 9, 2024
PubMed
概括

我们开发了一种新的反感应策略,通过向氨酸氧化酶 (PAH) 基因的错误拼接来纠正氨酸尿症 (PKU). 这种方法对治疗各种PAH拼接缺陷充满希望.

关键词:
在CRIPSR/Cas.反感的寡核化物.敲进细胞模型的模型.基尿症的现象 基尿症的现象拼接 拼接 拼接 拼接

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