Lipid nanoparticles containing messenger RNA (mRNA-LNPs) encoding Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) base-editors that correct pseudoxanthoma elasticum-causing mutations.
A flexible toolkit for human gene editing, activation, and repression that can be applied in orthogonally and in various disease models.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy.
Nanoparticles for potent encapsulation and delivery of therapeutic molecules for prenatal fetal therapy in the amniotic sac
A novel treatment for rare disease Friedreich ataxia using p38 or MK2 kinase inhibitors
Human anti-ADAMTS13 antibodies cloned from patients with acquired thrombotic thromobocytopenia purpura (TTP) for use in targeted therapies, and generation of animal models that recapitulate pathologic features of TPP.