Can hemophilia be cured? It depends on the definitiondoi:10.1016/j.rpth.2024.102559ethicsgenetic therapyhemophilia Ahemophilia BOver the years, the palette of treatment options for hemophilia has grown extensively, leading to an increased life expectancy and quality of life for people living with ...
Providing a long-term cure of these diseases requires not only that the hepatocytes be efficiently transduced, but also that the vectors remain transcriptionally active and give rise to therapeutic levels of functional protein. We have recently demonstrated that hemophilia A could be cured in newborn...
Pharmacists can play an important role in identifying skin cancer when they see something suspicious or unfamiliar on the skin of a patient. They can suggest seeing a dermatologist with the caveat that they don’t know that is going to be anything serious, but it should be checked. Having a...
as a treatment for these two disorders. Other major genetic disorders like hemophilia and congenital blindness are curable through gene therapy. Acquired diseases are treated with different gene therapies. Such therapeutic strategies include gene vaccines and oncolytic virotherapy. However, for neurodegenera...