Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides
Author:
Publisher
Proceedings of the National Academy of Sciences
Subject
Multidisciplinary
Reference45 articles.
1. Restoration of Human β-Globin Gene Expression in Murine and Human IVS2–654 Thalassemic Erythroid Cells by Free Uptake of Antisense Oligonucleotides
2. Correction of Aberrant Splicing of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Gene by Antisense Oligonucleotides
3. Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients
4. Reversal of the cellular phenotype in the premature aging disease Hutchinson-Gilford progeria syndrome
5. Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD
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