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Relevant Scientific Publication Category:
Gene-based Therapeutic Platforms
Selective Xi reactivation and alternative methods to restore MECP2 function in Rett syndrome.
Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping.
Spinal muscular atrophy with respiratory distress type 1: Clinical phenotypes, molecular pathogenesis and therapeutic insights.
Detection of RNA-DNA binding sites in long noncoding RNAs.
MicroRNA in Control of Gene Expression: An Overview of Nuclear Functions.
X inactivation and reactivation in X-linked diseases.
Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model