GM1-gangliosidosis and mucopolysaccharidosis IVB, 2 rare diseases caused by beta-galactosidase deficiency.
Delivers siRNA, antisense oligonucleotides, linear or circular mRNA and other RNA modalities to extrahepatic tissues - demonstrated across various indications in oncology (e.g. PDAC, breast cancer, ovarian cancer, lung cancer, melanoma, ATLL, TME), and inflammatory / autoimmune diseases (e.g. OA, RA, enterocolitis), CV (e.g. atherosclerosis, aortic aneurysm)
LakeSAI is a membership-based AI research agent built for scientists who need faster, deeper and more transparent access to biomedical knowledge. Users interact via natural language or structured data inputs, enabling precise, reproducible and evidence-based exploration.Explore: https://lakesai.vitafluence.com
obesity and diabetes