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.
Technology allowing efficient formation, delivery, isolation, and enrichment of single-copy Human Artificial Chromosome (HAC) for use in research, plant production, gene and cell therapy and more.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
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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
Ten candidate serum and synovial protein levels provide early indication of joint impairment in lysosomal storage disorders.
A new class of anisimide-functionalized lipids that enables targeted delivery of lipid nanoparticles (LNPs) to activated fibroblasts in the liver.