Gene editing is a growing gene engineering technique that allows accurate editing of a
broad spectrum of gene‐regulated diseases to achieve curative treatment and also has the …

The root cause of sickle cell disease (SCD) has been known for nearly a century, however,
few therapies to treat the disease are available. Over several decades of work, with …

In sickle cell disease (SCD), heme released during intravascular hemolysis promotes
oxidative stress, inflammation, and vaso-occlusion. Conversely, free heme can also activate …

Since first described, sickle cell disease (SCD) has represented a therapeutic challenge for
clinicians trying to help patients enduring the ramifications of this dreadful condition. This life …

Recent advancements in gene editing illuminate new potential therapeutic approaches for
Sickle Cell Disease (SCD), a debilitating monogenic disorder caused by a point mutation in …

The β-hemoglobinopathies, such as sickle cell disease and β-thalassemia, are one of the
most common genetic diseases worldwide and are caused by mutations affecting the …

A major limitation of gene therapy for sickle cell disease (SCD) is the availability and access
to a potentially curative one-time treatment, due to high treatment costs. We have developed …

Well-characterised mouse models of disease may provide valuable insights into
pathophysiology. This study characterises the Townes mouse model of sickle cell disease …

Pyruvate kinase (PK), a key ATP-generating enzyme in glycolysis, is a target for novel sickle
cell disease (SCD) therapies. Enhancing PK activity lowers 2, 3-diphosphyglycerate (2, 3 …

Sickle cell disease (SCD) is caused by the homozygous beta-globin gene mutation that can
lead to ischemic multi-organ damage and consequently reduce life expectancy. On the other …