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M51D PMO
- Development stage
- Preclinical
- Lead developer
- GeneTools
- Modality
- MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intracerebroventricular, Intrathecal
01
Overview
M51D PMO is a phosphorodiamidate morpholino oligomer (PMO) research compound used in preclinical studies for Duchenne muscular dystrophy (DMD). It is designed to induce exon 51 skipping in the dystrophin gene by targeting the Dmd exon 51 splice donor site (position +10 to -15). As an antisense oligonucleotide, it works through steric blockade to alter RNA splicing. This compound is frequently utilized as a tool to investigate delivery methods to the central nervous system, such as intracerebroventricular and intra-cisterna magna injections, in mouse models like the mdx52 mouse. It is manufactured by Gene Tools and is not an approved therapeutic drug for human use.
- Other names
- M51DM-51DM 51D
02
Targets
DMD (Dystrophin)
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