Berit E. Powers

Preclinical evaluation of antisense oligonucleotide therapy in a mouse model of HNRNPH2-related neurodevelopmental disorder

This study tests antisense oligonucleotides targeting Hnrnph2 in a mouse model, showing reduced Hnrnph2 levels, increased Hnrnph1, and improved phenotypes like seizures and motor function.

Reversable Acute Sedation Response of Phosphorothioate Antisense Oligonucleotides Following Local Delivery to the Central Nervous System

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.

Transient Acute Neuronal Activation Response Caused by High Concentrations of Oligonucleotides in the Cerebral Spinal Fluid

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.

Genotypic and phenotypic analysis of 173 patients with extremely rare pathogenic mutations who applied for experimental antisense oligonucleotide treatment

The paper analyzes genotypic and phenotypic data from 173 patients with nano-rare mutations to explore genotype-phenotype relationships and the potential for antisense oligonucleotide therapy.

Antisense therapy in a new rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment.

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 atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

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

Convective forces increase rostral delivery of intrathecal antisense oligonucleotides in the cynomolgus monkey nervous system

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

Therapeutic suppression of proteolipid protein rescues Pelizaeus-Merzbacher Disease in mice

This paper shows that reducing PLP1 expression in a mouse model of PMD improves myelination, motor function, and lifespan, supporting PLP1 suppression as a therapeutic strategy.