首页> Molecular therapy : the journal of the American Society of Gene Therapy> BLOC1S1 depletion via splice-switching oligonucleotides improves mitochondrial respiration and rescues ALS phenotypes.

BLOC1S1 depletion via splice-switching oligonucleotides improves mitochondrial respiration and rescues ALS phenotypes.

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第一作者： Jin-Hui,Hor

第一单位： Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A∗STAR), 61 Biopolis Dr, Singapore 138673, Singapore.

作者： Jin-Hui,Hor ^[1] ; Jin Rong,Ow ^[1] ; Winanto,Ng ^[1] ; Boominathan,Ramasamy ^[1] ; Tommaso,Tabaglio ^[1] ; Kimberly Ngee Hui,Lim ^[1] ; Muhammad Ikhsan,Bin Muzakar ^[1] ; Valerie Jing Wen,Lim ^[1] ; Rajasekhar Reddy,Gurrampati ^[1] ; Ravisankar,Rajarethinam ^[1] ; Shyuan T,Ngo ^[2] ; Venkataramanan,Ramadass ^[1] ; Shuo-Chien,Ling ^[3] ; Manikandan,Lakshmanan ^[1] ; Keng Boon,Wee ^[4] ; Shi-Yan,Ng ^[5]

作者单位： Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A∗STAR), 61 Biopolis Dr, Singapore 138673, Singapore. ^[1] Australian Institute for Bioengineering and Nanotechnology, The University of Queensland, Brisbane, QLD 4072, Australia. ^[2] Department of Physiology, National University of Singapore, YLL School of Medicine, Singapore 117456, Singapore. ^[3] Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A∗STAR), 61 Biopolis Dr, Singapore 138673, Singapore. Electronic address: weekb@a-star.edu.sg. ^[4] Institute of Molecular and Cell Biology (IMCB), Agency for Science, Technology and Research (A∗STAR), 61 Biopolis Dr, Singapore 138673, Singapore; Department of Physiology, National University of Singapore, YLL School of Medicine, Singapore 117456, Singapore. Electronic address: ng_shi_yan@a-star.edu.sg. ^[5]

关键词 ALS BLOC1S1 GCN5L1 RNA therapeutics SSO amyotrophic lateral sclerosis iPSC-derived motor neurons mitochondrial protein acetylation splice-switching antisense oligonucleotide