A Prospective Study in the Rational Design of Efficient Antisense Oligonucleotides for Exon Skipping in the DMD Gene-Reference-Cited by-同舟云学术

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A Prospective Study in the Rational Design of Efficient Antisense Oligonucleotides for Exon Skipping in the DMD Gene

Published:2012-07 Issue:7 Volume:23 Page:781-790
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Pramono Zacharias Aloysius Dwi¹,Wee Keng Boon²,Wang Jian Li³,Chen Yi Jun⁴,Xiong Qian Bin⁴,Lai Poh San⁵,Yee Woon Chee⁴

Affiliation:

1. National Skin Centre, Singapore 308205, Singapore.

2. A*STAR Institute of High Performance Computing, Singapore 138632, Singapore.

3. Formerly Neuromuscular Research Laboratory, National Neuroscience Institute, Singapore 308433, Singapore.

4. Department of Clinical Research, Singapore General Hospital, Singapore 169608, Singapore.

5. Department of Paediatrics, Yong Loo Lin School of Medicine, National University of Singapore, Singapore 119228, Singapore.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2011.205

Reference34 articles.

1. Antisense-mediated modulation of splicing: Therapeutic implications for Duchenne muscular dystrophy

2. Functional Analysis of 114 Exon-Internal AONs for Targeted DMD Exon Skipping: Indication for Steric Hindrance of SR Protein Binding Sites

3. Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications

4. Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

5. Guidelines for Antisense Oligonucleotide Design and Insight Into Splice-modulating Mechanisms

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1. Deep indel mutagenesis reveals the regulatory and modulatory architecture of alternative exon splicing;2024-04-23

2. Splice‐switch oligonucleotide‐based combinatorial platform prioritizes synthetic lethal targets CHK1 and BRD4 against MYC ‐driven hepatocellular carcinoma;Bioengineering & Translational Medicine;2022-09-03

3. RBFOX2 deregulation promotes pancreatic cancer progression and metastasis through alternative splicing;2022-06-04

4. eSkip-Finder: a machine learning-based web application and database to identify the optimal sequences of antisense oligonucleotides for exon skipping;Nucleic Acids Research;2021-06-09

5. Splice-Switching Antisense Oligonucleotides as a Targeted Intrinsic Engineering Tool for Generating Armored Redirected T Cells;Nucleic Acid Therapeutics;2021-04-01

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