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

BACKGROUND: 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. OBJECTIVE: We developed genetically modified adipocytes (GMAC)...

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Detalles Bibliográficos
Autores principales: 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9663876/
https://www.ncbi.nlm.nih.gov/pubmed/36387451
http://dx.doi.org/10.1016/j.heliyon.2022.e11271

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