Iron overload disorders are conditions that can lead to increased body iron stores and end-
organ damage in affected organs. Increased iron deposition most commonly occurs in the …

Haemochromatosis is one of the most common genetic diseases affecting patients of
northern European ancestry. It is overdiagnosed in patients without iron overload and is …

Advancements in gene and cell therapy have resulted in novel therapeutics for diseases
previously considered incurable or challenging to treat. Among the various contributing …

Hereditary hemochromatosis with the homozygous C282Y HFE mutation (HH-282H) is a
genetic condition which causes iron overload (IO) and elevated reactive oxygen species …

Iron overload is a significant complication commonly observed in individuals with β-
thalassemia, resulting from enhanced iron absorption due to ineffective erythropoiesis and …

Splicing bridges the gap between static DNA sequence and the diverse and dynamic set of
protein products that execute a gene's biological functions. While exon skipping …

Over 6500 Mendelian disorders have been documented, with approximately 4500 genes
linked to these conditions. The majority of inherited diseases present in childhood and …

CRISPR is a gene editing tool adapted from naturally occurring defense systems from
bacteria. It is a technology that is revolutionizing the interrogation of gene functions in driving …

The invention relates to antisense oligonucleotides that are capable of bringing about
specific editing of a target nucleotide (adenosine) in a target RNA in a eukaryotic cell …