Publications
Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference
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NUCLEIC ACIDS RESEARCH (April 2023)
Preclinical Evaluation of WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of C9orf72-associated ALS or FTD
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MOLECULAR THERAPY NUCLEIC ACIDS (April 2022)
Improvements to Hybridization-Ligation Enzyme-Linked Immunosorbent Assay Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics
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NUCLEIC ACID THERAPEUTICS (April 2022)
Development of a Sensitive Trial-ready Poly(GP) CSF Biomarker Assay for C9orf72-associated Frontotemporal Dementia and Amyotrophic Lateral Sclerosis
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JOURNAL OF NEUROLOGY, NEUROSURGERY & PSYCHIATRY (April 2022)
Predicting Exon Criticality from Protein Sequence
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NUCLEIC ACIDS RESEARCH (March 2022)
Endogenous ADAR-mediated RNA Editing in Non-human Primates using Stereopure Chemically Modified Oligonucleotides
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NATURE BIOTECHNOLOGY (March 2022)
Impact of Guanidine-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
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NUCLEIC ACIDS RESEARCH (February 2022)
Control of Backbone Chemistry and Chirality Boost Oligonucleotide Splice Switching Activity
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NUCLEIC ACIDS RESEARCH (January 2022)
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
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NATURE COMMUNICATIONS (February 2021)
Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species
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TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (January 2021)
Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease
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CLINICAL THERAPEUTICS (December 2020)
Investigational Assay for Haplotype Phasing of the Huntingtin Gene
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MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT (September 2020)
Genotyping Single Nucleotide Polymorphisms for Allele-Selective Therapy in Huntington’s Disease
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NEUROLOGY GENETICS (June 2020)
Control of Phosphorothioate Stereochemistry Substantially Increases the Efficacy of Antisense Oligonucleotides
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NATURE BIOTECHNOLOGY (September 2017)