Timothy M. Miller

This paper evaluates combining expression-based surrogate variables and genotype-based principal components in differential expression analysis, finding the combined approach improves replicability and biological recall in ALS datasets.

The paper demonstrates that antisense oligonucleotides (ASOs) can upregulate gene expression by stabilizing mRNA through targeting destabilizing elements like PREs, with therapeutic implications for haploinsufficiency disorders.

The paper characterizes big tau isoforms in human nervous system tissues and CSF, finding they are enriched in the peripheral nervous system and largely unaffected by neurodegenerative disease.

This study develops a lipid-siRNA conjugate that uses perivascular transport to achieve long-lasting gene knockdown in the CNS without toxicity.

This study shows that TDP-43 aggregation through prion-like seeding impairs its autoregulation, leading to dysfunction and loss of function in cells.

This paper identifies BIOIO-1001 as a dual mTOR/NAD+ acting gerotherapy that reduces metabolic derangements, inflammation, and fibrosis in NASH and ALS models.

This study evaluates the efficacy of commercially available antisense oligonucleotides (ASOs) targeting mouse and human tau in vivo, showing reduced tau levels and pathology in mouse models.

This study investigates DNAJB6 isoform-specific knockdown as a potential therapy for LGMDD1, showing that reducing DNAJB6b levels in a mouse model corrected proteomic disease signatures.

This study identifies TSC1 loss-of-function as a risk factor for tauopathies by promoting tau acetylation and impairing autophagy-mediated tau clearance.

The paper introduces FLEX Calling Cards, a method to map transcription factor-DNA interactions in specific cell types within heterogeneous tissues using viral delivery and transposon insertion.