Jonathan K. Watts

This study develops D-siRNA, an albumin-binding dendrimer-conjugated siRNA, showing effective and durable gene silencing in the CNS with no detectable toxicity.

The paper develops fully modified SpyCas9 guide RNAs that maintain or enhance genome editing efficacy in vitro and in vivo.

This paper develops a swine reporter model to evaluate gene delivery vectors in a large animal system and demonstrates its utility for assessing biodistribution, safety, and administration methods.

This study identifies organyl 5'-phosphate mimics that enhance AGO2 binding and metabolic stability by interacting with a hydrophobic pocket in the MID domain.

CRYPTID-exon identifies cryptic exons in human RNA-seq data, showing their impact on mRNA stability and gene expression through ASO targeting.

The paper tracks splicing intermediates in human cells using ultra-deep sequencing and finds pervasive usage of low-fidelity splice sites associated with genomic features that promote splicing noise.

This study designs and tests optimized oligonucleotide inhibitors for APOBEC3 enzymes, showing improved potency and nuclease stability compared to previous inhibitors.

This study shows adding high concentrations of Ca2+ and Mg2+ to oligonucleotide formulations prevents acute neurotoxicity in CNS delivery without affecting efficacy.

This study investigates the role of antisense and sense repeat RNAs in producing dipeptide repeat proteins in C9ORF72 expansion carriers and shows that targeting sense RNA with ASOs reduces both sense- and antisense-dependent DPR proteins.

This paper introduces a chemical adenylation method using formamide that achieves >90% yield for RNA sequencing ligation adaptors, improving scalability and reducing cleanup steps.

This paper engineers Nme2Cas9 base editors to improve activity and targeting scope, enabling correction of MECP2 mutations with minimal off-target effects.

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.

This study develops a compact adenine base editor (Nme2-ABE) that can be delivered via a single AAV vector to efficiently edit mouse genomic loci and revert disease mutations in a tyrosinemia model.

This paper describes a method to detect and quantify antisense oligonucleotides in biological samples using splint ligation and quantitative PCR, showing sensitivity across multiple tissues and ASO types.

This study shows that 5' modifications to DNA repair templates enhance precision genome editing efficiency in multiple animal models and human cells.

SisterC is a method to distinguish inter- and intra-sister chromatid interactions using BrdU labeling and Hi-C, revealing centromere alignment and cohesin-mediated loops.