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References from Silencing the mutant allele without compromising gene dosage. Local targets link to admitted publications; unresolved targets remain external evidence.
Epi-Allele elicits compensatory expression of the non-targeted allele and prevents haploinsufficiency in dominant genetic diseases
10.1016/j.stem.2026.08.004 · 2026 · External reference
Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy
10.1016/j.omtn.2017.07.006 · 2017 · External reference
Allele-specific silencing of the gain-of-function mutation in Huntington’s disease using CRISPR/Cas9
10.1172/jci.insight.141042 · 2022 · External reference
A cross-disorder dosage sensitivity map of the human genome
10.1016/j.cell.2022.06.036 · 2022 · External reference
Antisense oligonucleotide therapy reduces seizures and extends life span in an SCN2A gain-of-function epilepsy model
10.1172/jci152079 · 2021 · External reference
Genome-wide programmable transcriptional memory by CRISPR-based epigenome editing
10.1016/j.cell.2021.03.025 · 2021 · External reference
The transcription factor titration effect dictates level of gene expression
10.1016/j.cell.2014.02.022 · 2014 · External reference
Tuning Transcription Factor Availability through Acetylation-Mediated Genomic Redistribution
10.1016/j.molcel.2020.05.025 · 2020 · External reference
