Harry T. Orr

Mapping SCA1 regional vulnerabilities reveals neural and skeletal muscle contributions to disease

Dysregulation of zebrin-II cell subtypes in the cerebellum is a shared feature across polyglutamine ataxia mouse models and patients

Longitudinal single-cell transcriptional dynamics throughout neurodegeneration in SCA1

Decreasing mutant ATXN1 nuclear localization improves a spectrum of SCA1-like phenotypes and brain region transcriptomic profiles

Cross-species genetic screens identify transglutaminase 5 as a regulator of polyglutamine-expanded ataxin-1

Reduction of mutant ATXN1 rescues premature death in a conditional SCA1 mouse model

Consensus Paper: Strengths and Weaknesses of Animal Models of Spinocerebellar Ataxias and Their Clinical Implications

Modulation of ATXN1 S776 phosphorylation reveals the importance of allele-specific targeting in SCA1

Dual targeting of brain region‐specific kinases potentiates neurological rescue in Spinocerebellar ataxia type 1

Altered Capicua expression drives regional Purkinje neuron vulnerability through ion channel gene dysregulation in spinocerebellar ataxia type 1

Antisense oligonucleotide–mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles

ATXN1-CIC Complex Is the Primary Driver of Cerebellar Pathology in Spinocerebellar Ataxia Type 1 through a Gain-of-Function Mechanism

RBM17 Interacts with U2SURP and CHERP to Regulate Expression and Splicing of RNA-Processing Proteins

Reduction of protein kinase A-mediated phosphorylation of ATXN1-S776 in Purkinje cells delays onset of Ataxia in a SCA1 mouse model

PAK1 regulates ATXN1 levels providing an opportunity to modify its toxicity in spinocerebellar ataxia type 1

Disruption of the ATXN1–CIC complex causes a spectrum of neurobehavioral phenotypes in mice and humans

Tolerance is established in polyclonal CD4+ T cells by distinct mechanisms, according to self-peptide expression patterns

Extensive cryptic splicing upon loss of RBM17 and TDP43 in neurodegeneration models

Cerebellar Transcriptome Profiles of ATXN1 Transgenic Mice Reveal SCA1 Disease Progression and Protection Pathways

Pumilio1 Haploinsufficiency Leads to SCA1-like Neurodegeneration by Increasing Wild-Type Ataxin1 Levels

A native interactor scaffolds and stabilizes toxic ATAXIN-1 oligomers in SCA1

Ataxin-1 oligomers induce local spread of pathology and decreasing them by passive immunization slows Spinocerebellar ataxia type 1 phenotypes

The Unstable Repeats—Three Evolving Faces of Neurological Disease

RAS–MAPK–MSK1 pathway modulates ataxin 1 protein levels and toxicity in SCA1

Purkinje Cell Ataxin-1 Modulates Climbing Fiber Synaptic Input in Developing and Adult Mouse Cerebellum

Polyglutamine Disease Toxicity Is Regulated by Nemo-like Kinase in Spinocerebellar Ataxia Type 1