Vikram Khurana

This study uses a humanized mouse model to show that antisense oligonucleotides reduce seizure activity and mortality in DRPLA.

This study identifies tRNA-derived fragments (tRFs) as key regulators of Tau aggregation in Alzheimer's disease and shows their potential as therapeutic targets.

This study uses single-molecule imaging to show that Parkinson's disease-associated alpha-synuclein aggregates are more stable than physiological multimers under dilution.

NERINE tests gene network hypotheses with rare variant associations, identifying a subnetwork linked to Parkinson's disease.

This study shows that tRNA-derived fragments (tRFs) accumulate in aging mouse brains and are elevated in human neurodegenerative conditions, suggesting a role in neurodegeneration.

This paper demonstrates that antisense oligonucleotides targeting human ATN1 protect against DRPLA pathology in a fully humanized mouse model.

The paper introduces scJLIM, a cluster-free method for single-cell colocalization testing, improving power to link GWAS loci with gene expression changes in specific cell types.

This study develops a human brain tissue model (miBrain) to show that APOE4 astrocytes with cholesterol dysregulation promote α-synuclein pathology through endolysosomal dysfunction.

This paper introduces a multimodal foundation model for predicting protein phenotypes using sequence, structure, and natural language, showing competitive performance and novel insights into disease-related proteins.

This study shows that AAGGG repeat expansions cause synaptic defects in CANVAS neurons independently of RFC1, with phenotypes corrected by repeat deletion.

This paper develops an iPSC platform to rapidly generate alpha-synuclein inclusions and investigates their formation, subtypes, and molecular interactions.

The paper identifies a shared genetic risk module involving PSEN2 and actin regulation across alpha-synucleinopathies, linking them to Alzheimer's disease mechanisms.

The paper combines epidemiological pesticide exposure data with iPSC-derived dopaminergic neuron screening to identify and classify pesticides linked to Parkinson's disease.

This study develops a 3D cortical neurosphere model from patient-derived iPSCs to study Parkinson's Disease mechanisms and test small molecule therapeutics.

The paper evaluates the prevalence of RFC1 and DAB1 repeat expansions in an undiagnosed ataxia cohort, finding biallelic RFC1 expansions in 2.7% of patients.