Area of research
Molecular Biology · Genetics
Research interest
Research interests include Neurogenetic and Muscular Disorders Research, RNA Interference and Gene Delivery, RNA Research and Splicing, and DNA and Nucleic Acid Chemistry.
Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy
A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies
Mechanism of <i>STMN2</i> cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies
Antisense oligonucleotide targeting DMPK in patients with myotonic dystrophy type 1: a multicentre, randomised, dose-escalation, placebo-controlled, phase 1/2a trial
Stathmin-2 loss leads to neurofilament-dependent axonal collapse driving motor and sensory denervation
Personalized antisense oligonucleotides ‘for free, for life’ — the n-Lorem Foundation
Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS
Antisense Drugs Make Sense for Neurological Diseases
High-resolution visualization and quantification of nucleic acid–based therapeutics in cells and tissues using Nanoscale secondary ion mass spectrometry (NanoSIMS)
ALS-associated genes in SCA2 mouse spinal cord transcriptomes
Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration
Antisense Oligonucleotide Therapies for Neurodegenerative Diseases
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
Nusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
RNA-Targeted Therapeutics
Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models
Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells
Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7
Antisense oligonucleotide therapy for spinocerebellar ataxia type 2
Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study
Results from a phase 1 study of nusinersen (ISIS-SMN <sub>Rx</sub> ) in children with spinal muscular atrophy
Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs
Protein-RNA Networks Regulated by Normal and ALS-Associated Mutant HNRNPA2B1 in the Nervous System
The Antisense Transcript SMN-AS1 Regulates SMN Expression and Is a Novel Therapeutic Target for Spinal Muscular Atrophy
Modulation of LMNA splicing as a strategy to treat prelamin A diseases
Deletion or Inhibition of the Oxygen Sensor PHD1 Protects against Ischemic Stroke via Reprogramming of Neuronal Metabolism
Chronic and acute LRRK2 silencing has no long-term behavioral effects, whereas wild-type and mutant LRRK2 overexpression induce motor and cognitive deficits and altered regulation of dopamine release
Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy
ADVANCE Partnership: Supporting Public Impact Research through Institutional Transformation (SPIRIT)
Conference: A Three-Part Series to Envision & Enact an Inclusive & Diverse STEM Professoriate
Collaborative Research: NSF INCLUDES Alliance: National Alliance for Inclusive and Diverse STEM Faculty (NAIDSF)
The geochemical ecology of cryptoendolithic microorganisms: relationships between cyanobacteria and sandstone weathering in the Canadian High Arctic
Collaborative Research: Biogeochemical Controls on Antimony and Arsenic Mobility in a Siliceous Hydrothermal System
The Kinetics of Microbial Sulfuric Acid Speleogenesis
Optimized Variable Stiffness Energy Returning Ankle Foot Orthosis
Sediment Transport in an Urbanizing Karst Aquifer-Watershed
LExEn: Sulfuric Acid Speleogenesis by Chemolithotrophic Bacteria: Community Structure and Habitat Modification by Acid Extremophiles
Microbial Weathering of Silicates: Aggressive Release of Limiting Nutrients
Mass Transport Properties of Fracture Skins
ARFMP: Replacement of Chemistry Research and Research Training Laboratories
Silicate Dissolution in Organic-Rich Aqueous Systems: Reaction Mechanisms and Rate Controls
NSF Postdoctoral Fellowship in Plant Biology
Research Initiation: Interaction of Large-Scale Motion WithA Secondary Contraction (Mechanical Engineering and Applied Mechanics)