US scientists have developed a proof-of-concept treatment for blood disorders such as sickle cell disease and beta-thalassaemia.
Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a viral vector which can raise functional haemoglobin levels by activating production of both foetal and adult haemoglobin, while at the same time suppressing mutant haemoglobin.
Up until now, research into new genetic treatments for sickle cell disease and beta-thalassaemia have focused on either increasing foetal haemoglobin, which is not mutated in these conditions, or adding back a functional copy of adult haemoglobin via gene therapy, using a viral vector to supply new genetic material.
However, there are limitatio...
6 days ago
US scientists have developed a proof-of-concept treatment for blood disorders such as sickle cell disease and beta-thalassaemia.
Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a viral vector which can...
6 days ago
A new method of delivery developed by British scientists could avoid the risk of side effects from tissue plasminogen activator (tPA).
tPA is currently the most widely used thrombolytic drug, used to prevent strokes and heart a...
6 days ago