Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model.

The approval of splice-switching oligonucleotides with phosphorodiamidate morpholino oligomers (PMOs) for treating Duchenne muscular dystrophy (DMD) has advanced the field of oligonucleotide therapy. Despite this progress, PMOs encounter challenges such as poor tissue uptake, particularly in the heart, diaphragm, and central nervous system (CNS), thereby affecting patient's prognosis and quality of life. To address these limitations, we have developed a PMOs-based heteroduplex oligonucleotide (HDO) technology.

Effects of combinations of gapmer antisense oligonucleotides on the target reduction.

Background: The co-administration of several therapeutic oligonucleotides targeting the same transcript is a beneficial approach. It broadens the target sites for diseases associated with various mutations or splice variants. However, little is known how a combination of antisense oligonucleotides (ASOs), which is one of the major modalities of therapeutic oligonucleotides, affects the potency.

Temporal correlation between serum CH level and symptom severity of myasthenia gravis during eculizumab therapy.

The correlation between serum 50 % hemolytic complement (CH50) level and myasthenic symptom severity has not been known in patients with anti-acetylcholine receptor (anti-AChR)-positive myasthenia gravis (MG) during eculizumab treatment. A patient with anti-AChR-positive MG showed severe bulbar symptoms. Eculizumab administration decreased CH50 level and improved the symptoms.

Optic perineuritis with anti-myelin oligodendrocyte glycoprotein antibody.

A 25-year-old male presented with blurred peripheral vision and movement pain in his right eye. Fundus examination revealed unilateral disc swelling in his right eye with normal intracranial pressure. MRI showed remarkably high intensity in the optic nerve sheath and slightly high intensity in the optic nerve, indicating optic perineuritis (OPN).