Epigenetic therapy reprograms hereditary disease

被引：2

作者：

Bautista, Jose M. ^{[1
]}

机构：

[1] Univ Complutense Madrid, E-28040 Madrid, Spain

来源：

BLOOD | 2014年 / 124卷 / 01期

关键词：

DEFICIENCY; G6PD;

D O I：

10.1182/blood-2014-05-575423

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

In this issue of Blood, Makarona et al demonstrate that histone deacetylase (HDAC) inhibitors (HDACis) in glucose-6-phosphate dehydrogenase (G6PD)-deficient cells reinstates enzyme activity by boosting gene transcription. 1 This therapeutic approach opens new avenues for preclinical and clinical studies to treat not only chronic nonspherocytic hemolytic anemia caused by severe G6PD variants, 2 but also other genetic diseases.

引用

收藏

页码：7 / 8

页数：2

相关论文

共 10 条

[1] Brief report: Targeted therapy for inherited GPI deficiency. [J].

Almeida, Antonio M. ;

Murakami, Yoshiko ;

Baker, Alastair ;

Maeda, Yusuke ;

Roberts, Irene A. G. ;

Kinoshita, Taroh ;

Layton, D. Mark ;

Karadimitris, Anastasios .

NEW ENGLAND JOURNAL OF MEDICINE, 2007, 356 (16) :1641-1647

[2]

Atweh GF, 1999, BLOOD, V93, P1790

[3] Mass primaquine treatment to eliminate vivax malaria: lessons from the past [J].

Kondrashin, Anatoly ;

Baranova, Alla M. ;

Ashley, Elizabeth A. ;

Recht, Judith ;

White, Nicholas J. ;

Sergiev, Vladimir P. .

MALARIA JOURNAL, 2014, 13

[4] Precise Maps of RNA Polymerase Reveal How Promoters Direct Initiation and Pausing [J].

Kwak, Hojoong ;

Fuda, Nicholas J. ;

Core, Leighton J. ;

Lis, John T. .

SCIENCE, 2013, 339 (6122) :950-953

[5] Maternally transmitted severe glucose 6-phosphate dehydrogenase deficiency is an embryonic lethal [J].

Longo, L ;

Vanegas, OC ;

Patel, M ;

Rosti, V ;

Li, HQ ;

Waka, J ;

Merghoub, T ;

Pandolfi, PP ;

Notaro, R ;

Manova, K ;

Luzzatto, L .

EMBO JOURNAL, 2002, 21 (16) :4229-4239

[6] Transcriptional and epigenetic basis for restoration of G6PD enzymatic activity in human G6PD-deficient cells [J].

Makarona, Kalliopi ;

Caputo, Valentina S. ;

Costa, Joana R. ;

Liu, Binbin ;

O'Connor, David ;

Iskander, Deena ;

Roper, David ;

Robertson, Lynn ;

Bhatnagar, Neha ;

Terpos, Evangelos ;

Georgiou, Elisabeth ;

Papaioannou, Maria ;

Layton, D. Mark ;

Luzzatto, Lucio ;

Roberts, Irene ;

Karadimitris, Anastasios .

BLOOD, 2014, 124 (01) :134-141

[7] G6PD deficiency: the genotype-phenotype association [J].

Mason, Philip J. ;

Bautista, Jose M. ;

Gilsanz, Florinda .

BLOOD REVIEWS, 2007, 21 (05) :267-283

[8] PharmGKB summary: very important pharmacogene information for G6PD [J].

McDonagh, Ellen M. ;

Thorn, Caroline F. ;

Bautista, Jose M. ;

Youngster, Ilan ;

Altman, Russ B. ;

Klein, Teri E. .

PHARMACOGENETICS AND GENOMICS, 2012, 22 (03) :219-228

[9]

Roos D, 1999, BLOOD, V94, P2955

[10] Genome-wide Mapping of HATs and HDACs Reveals Distinct Functions in Active and Inactive Genes [J].

Wang, Zhibin ;

Zang, Chongzhi ;

Cui, Kairong ;

Schones, Dustin E. ;

Barski, Artem ;

Peng, Weiqun ;

Zhao, Keji .

CELL, 2009, 138 (05) :1019-1031