With the improvement of gene vectors, the rise of chimeric antigen receptor T cell
immunotherapy and breakthroughs in the genome editing technology, gene therapy had …

During the past decade, genome‐wide association studies (GWAS) have transformed our
understanding of many heritable traits. Three recent large‐scale GWAS meta‐analyses now …

Infection with hepatitis B virus (HBV) remains a global health challenge. Approximately 292
million people worldwide are chronically infected with HBV and the annual mortality from the …

The two major goals of the treatment of hypertriglyceridemia are the prevention of
cardiovascular disease and pancreatitis. Here we discuss the drugs used for the treatment of …

Lipoprotein lipase deficiency (LPLD) results from mutations within the lipoprotein lipase
(LPL) gene that lead to a complete lack of catalytically active LPL protein. Glybera was one …

Background: Lipoprotein lipase (LPL) deficiency is a genetic condition. Affected individuals
typically develop symptoms related to severe and persistent hypertriglyceridemia, such as …

Introduction Familial chylomicronemia syndrome (FCS) is a rare subtype of severe
hypertriglyceridemia that affects~ 1 in 100, 000 to 1,000,000 individuals. The major risk to …

Recombinant adeno-associated virus (rAAV) platforms hold promise for in vivo gene therapy
but are undermined by the undesirable transduction of antigen presenting cells (APCs) …

GPIHBP1 is a protein found in the endothelial cells of capillaries that is anchored by
glycosylphosphatidylinositol and binds to high-density lipoproteins. GPIHBP1 attaches to …

The recent developments in the delivery and design of transcription factors put their
therapeutic applications within reach, exemplified by cell replacement, cancer differentiation …