Genetic drugs based on nucleic acid biomolecules are a rapidly emerging class of
medicines that directly reprogramme the central dogma of biology to prevent and treat …

There is overwhelming clinical and genetic evidence supporting the concept that low-density-
lipoprotein cholesterol should be as low as possible for as long as possible in patients at …

Lipid nanoparticles (LNPs) have delivered therapeutic RNA to hepatocytes in humans.
Adsorption of apolipoprotein E (ApoE) onto these clinical LNP-mRNA drugs has been …

Since the discovery of the LDL receptor in 1973 by Brown and Goldstein as a causative
protein in hypercholesterolemia, tremendous amounts of effort have gone into finding ways …

Liver dysfunction and failure account for a major portion of premature deaths in patients
suffering from various iron associated pathogeneses, particularly primary and secondary …

Effective lipid lowering therapies are essential for the prevention of atherosclerosis and
cardiovascular disease. Available treatments have evolved in both their efficacy and their …

In recent years, mesoporous silica particles have been revealed as promising drug delivery
systems combining high drug loading capacity, excellent biocompatibility, and easy and …

Aims Refractory hypercholesterolemia (RH), caused primarily by the loss-of-function
mutation of LDL receptor (LDLR) gene seen in HoFH and HeFH patients, remains a major …

Familial hypercholesterolemia (FH) is an autosomal dominant inherited disease
characterized by high circulating low-density lipoprotein (LDL) cholesterol. High circulating …

En primer lugar, me gustaría agradecer a mis tres directores de tesis Paco, David y María
Francesca. A Paco, por su ayuda infinita, su charlas e historietas y la cercanía que tanto le …