LCAT-trial-24 weeks: Protocol for a clinical study to evaluate the safety of regenerative medicine and gene therapy by the autologous transplantation of human lecithin:cholesterol acyltransferase gene-transduced human pre-adipocytes

LCAT-trial-24 weeks: Protocol for a clinical study to evaluate the safety of regenerative medicine and gene therapy by the autologous transplantation of human lecithin:cholesterol acyltransferase gene-transduced human pre-adipocytes

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...

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Bibliographic Details
Main Authors: Masayuki Kuroda, Makoto Hori, Yoshiro Maezawa, Yoshitaka Kubota, Nobuyuki Mitsukawa, Yuki Shiko, Yoshihito Ozawa, Yohei Kawasaki, Yasushi Saito, Hideki Hanaoka, Koutaro Yokote
Format: Article
Language:English
Published: Elsevier 2022-08-01
Series:Contemporary Clinical Trials Communications
Subjects:
Familial lecithin:cholesterol acyltransferase deficiency
ex vivo gene therapy
Adipocytes
Regenerative medicine
Enzyme replacement therapy
Online Access:http://www.sciencedirect.com/science/article/pii/S2451865422000631

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http://www.sciencedirect.com/science/article/pii/S2451865422000631

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