DNAX

Kwong-Man Ng1, Pamela Y Mok1, Amy W Butler1

  • 1From Cardiology Division, Department of Medicine (K.-M.N., J.C.Y.H., Y.-K.L., W.-H.L., K.-W.A., Y.-M.L., L.-Y.W., C.-W.S., H.-F.T.) and Department of Psychiatry (A.W.B., S.-W.C., P.C.S., A.C.), Queen Mary Hospital, Research Center of Heart, Brain, Hormone and Healthy Aging, Li Ka Shing Faculty of Medicine (K.-M.N, C.W.-S., H.-F.T.), Hong Kong-Guangdong Joint Laboratory on Stem Cell and Regenerative Medicine (M.A.E., H.-F.T.), Centre for Genomic Sciences, Li Ka Shing Faculty of Medicine (P.C.S.), State Key Laboratory for Cognitive and Brain Sciences, Li Ka Shing Faculty of Medicine (P.C.S.), and Shenzhen Institutes of Research and Innovation (H.-F.T.), University of Hong Kong, Hong Kong SAR, China; Stem Cell Disease Models, A*STAR Institute of Medical Biology, Singapore (P.Y.M.); MRC Social Genetic and Developmental Psychiatry Centre, Institute of Psychiatry, King's College London, London, UK (A.W.B.); and Key Laboratory of Regenerative Biology of the Chinese Academy of Sciences and Guangdong Provincial Key Laboratory of Stem Cells and Regenerative Medicine, South China Institute for Stem Cell Biology and Regenerative Medicine, Guangzhou Institutes of Biomedicine and Health, Guangzhou, China (M.A.E.).

Circulation
|September 29, 2016
PubMed
概括

丹恩病是一种影响心脏的遗传性疾病,由于X染色体不活化,女性的严重程度各不相同. 用DNA甲基化抑制剂在患者细胞中重新激活健康的LAMP2基因改善了细胞功能,提供了潜在的治疗策略.