Publications by authors named "Mikayla L Brown"

Article Synopsis
  • ALS is a severe neurodegenerative disease characterized by the buildup of misfolded proteins in motor neurons, prompting researchers to find ways to reduce this burden for potential treatment.
  • Antisense oligonucleotides (ASOs) have been identified as a promising option to target proteins like SOD1 that cause mutations, but their delivery to the central nervous system is challenging due to the blood-brain barrier.
  • The study demonstrates that using transcranial focused ultrasound (FUS) along with calcium phosphate lipid nanoparticles significantly enhances the delivery of a SOD1 ASO into the brain of mice, leading to reduced SOD1 levels and improved motor neuron survival without damaging brain tissue.
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Matrix-assisted laser desorption/ionization mass spectrometry imaging (MALDI-MSI) can provide valuable insights into the metabolome of complex biological systems such as organ tissues and cells. However, obtaining metabolite data at single-cell spatial resolutions presents a few technological challenges. Generally, spatial resolution is defined by the increment the sample stage moves between laser ablation spots.

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Variants of the oxygen free radical scavenging enzyme superoxide dismutase-1 (SOD1) are associated with the neurodegenerative disease amyotrophic lateral sclerosis (ALS). These variants occur in roughly 20% of familial ALS cases, and 1% of sporadic ALS cases. Here, we identified a novel SOD1 variant in a patient in their 50s who presented with movement deficiencies and neuropsychiatric features.

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CuATSM has repeatedly demonstrated to be therapeutically effective in mouse models of amyotrophic lateral sclerosis (ALS), leading to current clinical trials. CuATSM acts to stabilize ALS-associated mutant SOD1 protein by supplying copper. However, work has demonstrated that CuATSM is only therapeutic for wild-type-like SOD1 mutants, not metal-binding-region mutants, suggesting that CuATSM may have genotype-specific effects.

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The synthetic copper-containing compound, CuATSM, has emerged as one of the most promising drug candidates developed for the treatment of amyotrophic lateral sclerosis (ALS). Multiple studies have reported CuATSM treatment provides therapeutic efficacy in various mouse models of ALS without any observable adverse effects. Moreover, recent results from an open label clinical study suggested that daily oral dosing with CuATSM slows disease progression in patients with both sporadic and familial ALS, providing encouraging support for CuATSM in the treatment of ALS.

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