Abstract:
:β-thalassemias are caused by nearly 300 mutations of the β-globin gene, leading to a low or absent production of adult hemoglobin (HbA). Two major therapeutic approaches have recently been proposed: gene therapy and induction of fetal hemoglobin (HbF) with the objective of achieving clinically relevant levels of Hbs. The objective of this article is to describe the development of therapeutic strategies based on a combination of gene therapy and induction of HbFs. An increase of β-globin gene expression in β-thalassemia cells can be achieved by gene therapy, although de novo production of clinically relevant levels of adult Hb may be difficult to obtain. On the other hand, an increased production of HbF is beneficial in β-thalassemia. The combination of gene therapy and HbF induction appears to be a pertinent strategy to achieve clinically relevant results.
journal_name
Expert Rev Hematoljournal_title
Expert review of hematologyauthors
Breda L,Rivella S,Zuccato C,Gambari Rdoi
10.1586/ehm.13.24subject
Has Abstractpub_date
2013-06-01 00:00:00pages
255-64issue
3eissn
1747-4086issn
1747-4094journal_volume
6pub_type
杂志文章abstract::In chronic lymphocytic leukemia (CLL), the most prevalent lymphoid malignancy in western countries, patients have a median age at diagnosis of 72 years. In the last few years, there has been remarkable progress in understanding the biology of CLL, the detection of molecular prognostic factors and the development of mo...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2013.814845
更新日期:2013-08-01 00:00:00
abstract:BACKGROUND:Hb H disease is an alpha-thalassemia (α-thal) syndrome characterized by chronic hemolytic anemia that occurs when three of total four α-globin genes lost their function due to completely deletions or different kind of mutations. OBJECTIVE:We here described 66 patients who have been diagnosed for Hb H diseas...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1586/17474086.2016.1107470
更新日期:2016-01-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 评论,杂志文章
doi:10.1586/ehm.10.36
更新日期:2010-08-01 00:00:00
abstract::Professor John Gribben is Chair of the International Workshop on non-Hodgkin Lymphoma and the Gordon Hamilton Fairley Chair of Medical Oncology at St. Bartholomew's Hospital, Bart's Cancer Institute, London, UK, a Cancer Research UK Centre of Excellence. His doctoral studies were performed at University College London...
journal_title:Expert review of hematology
pub_type: 面试
doi:10.1586/ehm.13.13
更新日期:2013-04-01 00:00:00
abstract:INTRODUCTION:In sickle cell disease (SCD), hemoglobin S (HbS) red blood cells (RBCs) are characteristically deformed and inflexible. Often breaking down in the circulation, they exhibit increased adhesive properties with the endothelium and activated neutrophils and platelets, increasing the risk of occlusion of the mi...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1758555
更新日期:2020-06-01 00:00:00
abstract::Introduction: Thrombotic thrombocytopenic purpura (TTP) is a rare and life-threatening thrombotic microangiopathy characterized by microangiopathic hemolytic anemia, consumption thrombocytopenia, and organ injury. TTP pathophysiology is based on a severe ADAMTS13 deficiency, the specific von Willebrand factor (VWF)-cl...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2019.1611423
更新日期:2019-06-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章
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更新日期:2015-08-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2019.1642103
更新日期:2019-09-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1755958
更新日期:2020-06-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
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更新日期:2018-08-01 00:00:00
abstract:INTRODUCTION:Catastrophic antiphospholipid syndrome (CAPS) is a rare, life-threatening disease. In 1992, Asherson defined it as a widespread coagulopathy related to the antiphospholipid antibodies (aPL). CAPS requires rapid diagnosis and prompt initiation of treatment. Areas covered: This paper discusses all aspects of...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1300522
更新日期:2017-04-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2017.1343140
更新日期:2017-08-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2018.1518712
更新日期:2018-10-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.11.58
更新日期:2011-12-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2016.1142370
更新日期:2016-03-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2019.1627191
更新日期:2019-07-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2.1.17
更新日期:2009-02-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2016.1134309
更新日期:2016-03-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1313701
更新日期:2017-05-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2016.1232163
更新日期:2016-10-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.10.81
更新日期:2011-02-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2019.1649132
更新日期:2019-10-01 00:00:00
abstract:INTRODUCTION:Treatment of elderly patients with acute leukemia is challenging. Older age is associated with increased risk of treatment-related toxicity. Currently, no consensus exists regarding optimal therapy in this patient population. Areas covered: The following review is a comprehensive summary of various therape...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1080/17474086.2017.1382345
更新日期:2017-11-01 00:00:00
abstract::Thromboembolic diseases require anticoagulation for their prevention and treatment. New oral anticoagulants, specifically direct factor Xa and thrombin inhibitors, were developed to overcome the limitations of conventional anticoagulants. Their benefit has been demonstrated using fixed doses without laboratory-guided ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.11.79
更新日期:2012-02-01 00:00:00
abstract:INTRODUCTION:The most common forms of hematological malignancies that occur in female reproductive years are lymphoma and leukemia. Areas covered: Several aggressive gonadotoxic regimens such as alkylating chemotherapy and total body irradiation are used frequently in treatment of lymphoma and leukemia leading to subse...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2017.1371009
更新日期:2017-11-01 00:00:00
abstract:INTRODUCTION:Peripheral T cell lymphomas (PTCL) are a heterogenous group of lymphoproliferative disorders which are generally not curable with conventional chemotherapy and associated with inferior outcomes. Pralatrexate is a novel folate analog, the first FDA approved drug) for the treatment of relapsed/refractory (R/...
journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2020.1756257
更新日期:2020-06-01 00:00:00
abstract::Primary mediastinal large B-cell lymphoma (PMBCL) is a unique B-cell lymphoma variant that arises from a putative thymic medulla B cell. It constitutes 2-4% of non-Hodgkin lymphomas and occurs most frequently in young females. PMBCL is characterized by a diffuse proliferation of medium-to-large B cells associated with...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2015.994604
更新日期:2015-04-01 00:00:00
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journal_title:Expert review of hematology
pub_type: 杂志文章
doi:10.1080/17474086.2021.1856653
更新日期:2021-01-01 00:00:00
abstract::The incidence of non-Hodgkin's lymphoma (NHL) is increasing among all age groups, with a median age at diagnosis of 67 years. With the increase in the geriatric population, there is a need for the development and validation of treatment strategies for NHL for these patients. Therapy in elderly patients is affected by ...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/ehm.09.7
更新日期:2009-04-01 00:00:00
abstract::Recent studies have identified recurrent mutations in genes that encode proteins crucial in the epigenetic regulation of gene transcription in hematologic malignancies. Somatic mutations in epigenetic modifiers, including IDH1, IDH2, TET2, DNAMT3A, ASXL1, MLL and EZH2 are enriched in patients with acute myeloid leukem...
journal_title:Expert review of hematology
pub_type: 杂志文章,评审
doi:10.1586/17474086.2016.1144469
更新日期:2016-05-01 00:00:00