Archives: Funded Research

Understanding UBE3A dysfunction in neuronal development

Long-read sequencing to evaluate individuals with suspected Angelman syndrome and incomplete or no molecular diagnosis

Unbiased Whole Genome CRISPR Screen to Identify Molecular Targets of Paternal Ube3a Silencing for Therapeutic Development

Role of UBE3A-HERC2 Complex in Angelman Syndrome: 3D Structure and Quantitative Interactomics

The Prevalence and Form of CVI in Angelman Syndrome

Newborn Screening for Angelman syndrome, Prader-Willi, Fragile X and Dup15q Syndromes

Validation of therapeutic guide RNAs targeting the UBE3A antisense transcript

Pre-clinical assessment of cannabidiol as a treatment for Angelman syndrome

Pilot study to validate three novel classes of small molecules to unsilence paternal Ube3a allele

Gene Therapy in Angelman Syndrome