LCAT gene-transduced autologous human preadipocytes

LCAT gene-transduced autologous human preadipocytes is an ex vivo gene and cell therapy developed for the treatment of familial lecithin-cholesterol acyltransferase (LCAT) deficiency. The therapy involves harvesting preadipocytes from the patient's own adipose tissue, transducing them with a viral vector (typically retroviral) carrying the wild-type human LCAT gene, and then transplanting the modified cells back into the patient via subcutaneous injection. Once engrafted, these cells function as a continuous 'bio-factory,' secreting functional LCAT enzyme into the systemic circulation. This approach is designed to restore cholesterol esterification activity, thereby addressing the underlying cause of the disease and potentially preventing or slowing the progression of clinical manifestations such as corneal opacities, hemolytic anemia, and progressive renal failure.

Other names

LCAT gene-transduced autologous preadipocytesLCAT-transduced preadipocytes