公开(公告)号：US20100143961A1

公开(公告)日：2010-06-10

申请号：US12545712

申请日：2009-08-21

Applicant: Hsuan-Lin Lan-Hargest ,  Robert J. Kaufman

Inventor： Hsuan-Lin Lan-Hargest ,  Robert J. Kaufman

IPC: C12Q1/02

CPC classification number: C07D303/32

Abstract: Histone deacetylase is a metallo-enzyme with zinc at the active site. Compounds having a zinc-binding moiety, for example, an alpha-ketoepoxide group, such as an alpha-ketothio group, can inhibit histone deacetylase. Histone deacetylase inhibition can repress gene expression, including expression of genes related to tumor suppression. Accordingly, inhibition of histone deacetylase can provide an alternate route for treating cancer, hematological disorders, e.g., hemoglobinopathies, autosomal dominant disorders, e.g. spinal muscular atrophy and Huntington's disease, genetic related metabolic disorders, e.g., cystic fibrosis and adrenoleukodystrophy, or to stimulate hematopoietic cells ex vivo.

Abstract translation: 组蛋白脱乙酰酶是在活性位点具有锌的金属酶。 具有锌结合部分的化合物，例如α-酮氧化物基团，例如α-酮硫基，可以抑制组蛋白脱乙酰酶。 组蛋白脱乙酰酶抑制可抑制基因表达，包括与肿瘤抑制相关的基因的表达。 因此，组蛋白脱乙酰酶的抑制可以提供治疗癌症的替代途径，血液学病症，例如血红蛋白病，常染色体显性疾病，例如常染色体显性疾病。 脊髓性肌肉萎缩和亨廷顿病，遗传相关的代谢紊乱，例如囊性纤维化和肾上腺脑白质营养不良，或离体刺激造血细胞。