To target a Charcot-Marie-Tooth disease Type 2S (CMT2S) patient-specific cryptic splice site variant within immunoglobulin mu-binding protein 2 (IGHMBP2) with a novel antisense oligonucleotide (ASO), rescuing IGHMBP2 protein levels.
No takes yet. Share an insight, caveat, or question.
Tyner et al. (2024) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: