First-in-human autologous implantation of genetically modified adipocytes expressing LCAT for the treatment of familial LCAT deficiency

• Published in: Heliyon Vol. 8; no. 11; p. e11271
• Main Authors: Aso, Masayuki, Yamamoto, Tokuo T., Kuroda, Masayuki, Wada, Jun, Kubota, Yoshitaka, Ishikawa, Ko, Maezawa, Yoshiro, Teramoto, Naoya, Tawada, Ayako, Asada, Sakiyo, Aoyagi, Yasuyuki, Kirinashizawa, Mika, Onitake, Akinobu, Matsuura, Yuta, Yasunaga, Kunio, Konno, Shun-ichi, Nishino, Katsuaki, Yamamoto, Misato, Miyoshi, Junko, Kobayashi, Norihiko, Tanio, Masami, Ikeuchi, Takayuki, Igari, Hidetoshi, Mitsukawa, Nobuyuki, Hanaoka, Hideki, Yokote, Koutaro, Saito, Yasushi
• Format: Journal Article
• Language: English
• Published: Elsevier Ltd 01.11.2022; Elsevier
• Subjects: adipocytes; antihypertensive agents; blood pressure; blood serum; cholesterol; cholesterol acyltransferase; Cholesterol homeostasis; cornea; Ex vivo gene therapy; Familial LCAT deficiency; gene therapy; genes; genetic disorders; haptoglobins; HDL; hemoglobin; hemolytic anemia; hypertension; LCAT; lecithins; lipoproteins; opacity; patients; Proteinuria; Renal injury; Renal injury; HDL; Familial LCAT deficiency; LCAT; Ex vivo gene therapy; Cholesterol homeostasis; Proteinuria

Familial lecithin: cholesterol acyltransferase (LCAT) deficiency (FLD) is a severe inherited disease without effective treatment. Patients with FLD develop severe low HDL, corneal opacity, hemolytic anemia, and renal injury. We developed genetically modified adipocytes (GMAC) secreting LCAT (LCAT-GM...