Preclinical Evaluation of Stereopure Antisense Oligonucleotides for Allele-selective Lowering of Mutant HTT
MOLECULAR THERAPY NUCLEIC ACIDS (June 2024)
Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference
NUCLEIC ACIDS RESEARCH (April 2023)
Preclinical Evaluation of WVE-004, an Investigational Stereopure Antisense Oligonucleotide for the Treatment of C9orf72-associated ALS or FTD
MOLECULAR THERAPY NUCLEIC ACIDS (April 2022)
Improvements to Hybridization-Ligation Enzyme-Linked Immunosorbent Assay Methods to Overcome Bioanalytical Challenges Posed by Novel Oligonucleotide Therapeutics
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
JOURNAL OF NEUROLOGY, NEUROSURGERY & PSYCHIATRY (April 2022)
Predicting Exon Criticality from Protein Sequence
NUCLEIC ACIDS RESEARCH (March 2022)
Endogenous ADAR-mediated RNA Editing in Non-human Primates using Stereopure Chemically Modified Oligonucleotides
NATURE BIOTECHNOLOGY (March 2022)
Impact of Guanidine-containing Backbone Linkages on Stereopure Antisense Oligonucleotides in the CNS
NUCLEIC ACIDS RESEARCH (February 2022)
Control of Backbone Chemistry and Chirality Boost Oligonucleotide Splice Switching Activity
NUCLEIC ACIDS RESEARCH (January 2022)
Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models
NATURE COMMUNICATIONS (February 2021)
Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species
TRANSLATIONAL VISION SCIENCE & TECHNOLOGY (January 2021)
Lessons from an Experiential Approach to Patient Community Engagement in Rare Disease
CLINICAL THERAPEUTICS (December 2020)
Investigational Assay for Haplotype Phasing of the Huntingtin Gene
MOLECULAR THERAPY: METHODS & CLINICAL DEVELOPMENT (September 2020)
Genotyping Single Nucleotide Polymorphisms for Allele-Selective Therapy in Huntington’s Disease
NEUROLOGY GENETICS (June 2020)
Control of Phosphorothioate Stereochemistry Substantially Increases the Efficacy of Antisense Oligonucleotides
NATURE BIOTECHNOLOGY (September 2017)