Search results (236)
« Back to PublicationsHuman-specific features of the cerebellum and ZP2-regulated synapse development.
Journal article
Kim S-K. et al, (2026), Cell
Targeted BDNF upregulation via upstream open reading frame disruption.
Journal article
Feng N. et al, (2026), Mol Ther, 34, 1652 - 1671
Association of Carboxypeptidase B2 Gene Polymorphisms With Graft Loss in Kidney Transplantation.
Journal article
Poppelaars F. et al, (2026), Transplant Direct, 12
Screening rare genetic diagnoses for amenability to bespoke antisense oligonucleotide therapy development: A retrospective cohort study.
Journal article
Cheerie D. et al, (2026), Genet Med, 28
Publisher Correction: CRISPR activation for SCN2A-related neurodevelopmental disorders.
Journal article
Tamura S. et al, (2026), Nature, 649
Engineered AAV Capsids with Enhanced Extracellular Vesicle Loading via Rational Design and Directed Evolution
Preprint
Vorobieva I. et al, (2025)
Modulating splicing in 5' untranslated regions to treat rare haploinsufficient disease.
Preprint
Beer Wells ES. et al, (2025)
Clinical Manifestations
Journal article
Jung Y. et al, (2025), Alzheimer S Dementia the Journal of the Alzheimer S Association, 21
Common and rare variant genetic contributions in African Americans with autism.
Preprint
Cirnigliaro M. et al, (2025)
Disruption of Cell-Type-Specific Molecular Programs of Medium Spiny Neurons in Autism.
Preprint
Yuan G. et al, (2025)
Accurately modelling RNase H-mediated antisense oligonucleotide efficacy
Preprint
Hill B. et al, (2025)
Genotypic, functional, and phenotypic characterization in CTNNB1 neurodevelopmental syndrome.
Journal article
Žakelj N. et al, (2025), HGG Adv, 6
CRISPR activation for SCN2A-related neurodevelopmental disorders.
Journal article
Tamura S. et al, (2025), Nature, 646, 983 - 991
Human-specific features of the cerebellum and ZP2-regulated synapse development.
Journal article
Kim S-K. et al, (2025), bioRxiv
Publisher Correction: Evaluation of familial phenotype deviation to measure the impact of de novo mutations in autism.
Journal article
Kim S-W. et al, (2025), Genome Med, 17