[HTML][HTML] Monogenic diseases that can be cured by liver transplantation

S Fagiuoli, E Daina, L D'Antiga, M Colledan… - Journal of …, 2013 - Elsevier
While the prevalence of most diseases caused by single-gene mutations is low and defines
them as rare conditions, all together, monogenic diseases account for approximately 10 in
every 1000 births according to the World Health Organisation. Orthotopic liver
transplantation (LT) could offer a therapeutic option in monogenic diseases in two ways: by
substituting for an injured liver or by supplying a tissue that can replace a mutant protein. In
this respect, LT may be regarded as the correction of a disease at the level of the …