Curt Mazur

This study evaluates the analgesic effects of intrathecal NaV 1.7 antisense oligonucleotides in rats, showing reduced channel binding and pain behaviors with concentration-dependent knock-down of Scn9a mRNA.

This study shows that reducing EMC10 levels in 22q11.2 deletion syndrome models improves cognitive function and sustains benefits for over two months.

This study characterizes an acute sedation response caused by phosphorothioate ASOs after CNS delivery, showing it is sequence-, dose-, and chemistry-dependent and can be mitigated.

The paper characterizes a transient neurobehavioral response to high-dose oligonucleotide delivery in the CNS and provides a framework to mitigate it through magnesium supplementation.

This paper introduces a new rat model of Alexander disease that exhibits key pathological features and motor impairment, demonstrating that antisense oligonucleotide therapy can reverse GFAP pathology and white matter deficits.

The paper maps ASO distribution and activity in the CNS of rodents and NHP after central administration, showing widespread effects across cell types.

This study shows that increasing convective forces after intrathecal injection improves rostral distribution of therapeutic ASOs in the CNS of cynomolgus monkeys.