Contemp Clin Trials Commun 2022 Aug 9;28:100946. Epub 2022 Jun 9.
Department of Endocrinology, Hematology and Gerontology, Chiba University Graduate School of Medicine, Chiba University, 1-8-1 Inohana Chuo-ku, Chiba, 260-0856, Japan.
Backgrounds: Despite the absolute need for life-long treatment of inherited and genetic diseases, there has been little effort to develop such treatments for most of these conditions due to their rarity. Familial lecithin:cholesterol acyltransferase (LCAT) deficiency is recognized as one such orphan disease. We have been developing an adipocyte-based gene therapy/regenerative medicine, a novel methodology that differs from the adeno-associated virus-mediated gene therapy or gene-transduced hematopoietic cell therapy, to treat familial LCAT deficiency. Read More