Identification of AAV serotypes for gene therapy in Krabbe iPSCs-derived brain organoids  

作　　者：Yafeng Lv Zhi Cui Hongbo Li Jing Wang Mulan Wei Yuanlang Hu Xun Li Chunyu Cao Ye Zhang Wei Wang 

机构地区：[1]Hubei Key Laboratory of Tumor Microenvironment and Immunotherapy,College of Basic Medical Sciences,China Three Gorges University,Yichang,Hubei 443000,China [2]State Key Laboratory of Medical Molecular Biology,Department of Biochemistry and Molecular Biology,Institute of Basic Medical Sciences,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100005,China [3]Yiling People’s Hospital of Yichang,Yichang,Hubei 443000,China [4]China-Japan Friendship Hospital,Beijing 100029,China

出　　处：《Genes & Diseases》2025年第1期144-147,共4页基因与疾病(英文)

基　　金：funded by grants from the Beijing Municipal Science&TechnologyCommission(China)(No.Z211100002921005 to W.W.);National High-Level Hospital Clinical Research Funding(China)(No.2022-NHLHCRF-PY-12 to W.W.);Hubei Provincial Department of Education's Scientific and Technological Research Project(China)(No.Q20211207 to Y.L.);Yichang Medical and Health Science and Technology Project(Hubei,China)(No.A22-2-069 to Y.L.);the Open Foundation of Hubei Province Key Laboratory of Tumor Microenvironment and Immunotherapy(China)(No.2023KZL08 to Y.L.).

摘　　要：Krabbe disease,also known as globoid cell leukodystrophy,is a rare lysosomal storage disorder.It is primarily caused by mutations in the GALC gene on chromosome 14q31,leading to GALC enzyme deficiency in lysosomes.This results in the accumulation of toxic substrate psychosine in the nervous systems.1 Currently,hematopoietic stem cell transplantation is the only available treatment,offering only a delay in neurological deterioration.Gene therapy,particularly using recombinant adeno-associated viruses(AAVs),shows promise for treating genetic diseases by introducing functional genes into target cells.

关 键 词：AAV DYSTROPHY diseases 

分 类 号：R73[医药卫生—肿瘤]