Problems and prospects in gene therapy for hemophilia.

abstract：PURPOSE OF REVIEW:Intravenous immunoglobulin (IVIg) is an effective treatment for an increasing number of autoimmune and inflammatory conditions. However, IVIg continues to be limited by problems of potential shortages and cost. A number of mechanisms have been described for IVIg, which have been captured in newly emer...

journal_title：Current opinion in hematology

authors： Norris PAA,Kaur G,Lazarus AH

更新日期：2020-11-01 00:00:00

abstract：PURPOSE OF REVIEW:The leukocyte adhesion deficiency (LAD) syndromes are rare genetically determined conditions with challenging clinical features. These immunodeficiencies also provide insights that are broadly relevant to the biology of leukocytes, platelets, intercellular interactions, and intracellular signaling. Re...

journal_title：Current opinion in hematology

authors： Harris ES,Weyrich AS,Zimmerman GA

更新日期：2013-01-01 00:00:00

abstract：:Disorders of the erythrocyte membrane, such as hereditary spherocytosis, hereditary elliptocytosis, and hereditary pyropoikilocytosis, are characterized by heterogeneity in their clinical and laboratory manifestations. Recent work has shown that these disorders also exhibit marked genetic heterogeneity. Multiple genet...

journal_title：Current opinion in hematology

authors： Gallagher PG,Ferriera JD

更新日期：1997-03-01 00:00:00

abstract：PURPOSE OF REVIEW:Over the last years, long non-coding RNAs (lncRNAs) have emerged as putative regulators of malignant hematopoietic development. Here, we review recent literature on the involvement of lncRNAs in leukemia, including their role in driving or sustaining disease and their potential impact on diagnosis, cl...

journal_title：Current opinion in hematology

authors： Lammens T,Durinck K,Wallaert A,Speleman F,Van Vlierberghe P

更新日期：2017-07-01 00:00:00

abstract：:Thrombopoietin, also termed the c-Mpl ligand, is a lineage-dominant hematopoietic factor that primarily regulates megakaryopoiesis and thrombopoiesis. Treatment of normal animals with recombinant human megakaryocyte growth and development factor, a truncated molecule of the c-Mpl ligand, which is modified with polyeth...

journal_title：Current opinion in hematology

authors： Miyazaki H

更新日期：1998-05-01 00:00:00

abstract：PURPOSE OF REVIEW:Hematopoietic stem cells are functionally heterogeneous even when isolated as phenotypically homogenous populations. How this heterogeneity is generated is incompletely understood. Several models have been formulated to explain the generation of diversity. All of these assume the existence of a single...

journal_title：Current opinion in hematology

authors： Muller-Sieburg CE,Sieburg HB

更新日期：2006-07-01 00:00:00

abstract：PURPOSE OF REVIEW:A major goal in repopulating hematopoietic stem cell (HSC) gene therapies is achieving high-efficacy gene transfer, while maintaining robust HSC engraftment and differentiation in vivo. Recent studies have documented that rapamycin treatment of HSC during lentiviral vector transduction enhances gene t...

journal_title：Current opinion in hematology

authors： Wang CX,Torbett BE

更新日期：2015-07-01 00:00:00

abstract：PURPOSE OF REVIEW:The role of a patent foramen ovale (PFO) as a risk factor for ischemic stroke has been established in recent years. However, the best therapeutic option to prevent recurrent events is still controversial, with antithrombotic treatment or transcatheter PFO closure being favored by different clinicians....

journal_title：Current opinion in hematology

authors： Di Tullio MR,Homma S

更新日期：2009-09-01 00:00:00

abstract：:Platelet production is primarily regulated by the thrombopoietic cytokine thrombopoietin (TPO). In most cases thrombopoietin serum levels are determined by the rate of c-mpl receptor-mediated degradation after TPO uptake into platelets and megakaryocytes. The contribution of increased TPO protein synthesis by a transl...

journal_title：Current opinion in hematology

authors： Verbeek W,Faulhaber M,Griesinger F,Brittinger G

更新日期：2000-05-01 00:00:00

abstract：PURPOSE OF REVIEW:Mortality and morbidity associated with leukemia are largely due to frequently occurring cytopenias or the dysfunction of normal blood cells in patients. Our knowledge of how normal blood cells degenerate in response to leukemic cell infiltration has been quite limited. This review summarizes recent f...

journal_title：Current opinion in hematology

authors： Cheng H,Cheng T

更新日期：2016-07-01 00:00:00

abstract：PURPOSE OF REVIEW:Despite improvements in the achievement of complete remission and progress in the supportive care of adults with acute lymphoblastic leukemia during the last decade, the majority of patients have eventually relapsed with overall survival in adult acute lymphoblastic leukemia of only 30-40%. However, t...

journal_title：Current opinion in hematology

authors： Larson S,Stock W

更新日期：2008-07-01 00:00:00

abstract：:Over a decade has passed since the first cases of AIDS were reported in hemophiliacs and transfusion recipients. Studies published over the past year offer an increasingly clear understanding of the extent of the early transfusion-associated AIDS epidemic, and of the effectiveness of progressive donor screening measur...

journal_title：Current opinion in hematology

authors： Busch MP

更新日期：1994-11-01 00:00:00

abstract：PURPOSE OF REVIEW:Platelet microparticles were identified more than 40 years ago and are the most abundant circulating microparticle subtype. Yet fundamental questions about their formation and role in human disease are just beginning to be understood at the cellular and molecular level. This review will address mechan...

journal_title：Current opinion in hematology

authors： Italiano JE Jr,Mairuhu AT,Flaumenhaft R

更新日期：2010-11-01 00:00:00

abstract：:Modern molecular technology helped identify more than 10 protein tyrosine kinases related to myeloid malignancies, which allowed the development of small molecule inhibitors targeting deregulated protein tyrosine kinase activity. Protein tyrosine kinase deregulation can occur as a consequence of fusion gene formation ...

journal_title：Current opinion in hematology

authors： Anastasiadou E,Schwaller J

更新日期：2003-01-01 00:00:00

abstract：:Anemia is a common disorder in patients with cancer and can be caused by the disease itself or by cancer-related therapy. The cardinal symptom of anemia, fatigue, is the most commonly reported symptom in patients with cancer and has profound effects on patient well-being and quality of life. Until recently, blood tran...

journal_title：Current opinion in hematology

authors： Yount S,Lai JS,Cella D

更新日期：2002-05-01 00:00:00

abstract：PURPOSE OF REVIEW:The transmembrane protein tissue factor (TF), the initiator of the extrinsic coagulation cascade and a risk factor for thrombotic disease, largely resides on the cell surface in an inactive, encrypted conformation. Whereas TF procoagulant activation, or decryption, is closely associated with the expos...

journal_title：Current opinion in hematology

authors： Versteeg HH,Ruf W

更新日期：2011-09-01 00:00:00

abstract：:Platelet glycoprotein (GP) IIb/IIIa receptor antagonists are being used with increasing frequency in the settings of percutaneous coronary interventions and acute ischemic syndromes. The development of bleeding complications following GPIIb/IIIa blockade represents a significant limitation to its effectiveness. Baseli...

journal_title：Current opinion in hematology

authors： Madan M,Blankenship JC,Berkowitz SD

更新日期：1999-09-01 00:00:00

abstract：PURPOSE OF REVIEW:Deficiency of antithrombin, protein C, and protein S increases the risk of a first venous thromboembolism (VTE) by at least 10-fold and is rare (i.e., <0.5% of population), whereas factor V Leiden and the prothrombin G20210A gene increase this risk by 2-5-fold and are common (2-5% of whites). Antiphos...

journal_title：Current opinion in hematology

authors： Kearon C

更新日期：2012-09-01 00:00:00

abstract：PURPOSE OF REVIEW:The proteome is the pool of proteins expressed at a given time and circumstance. The word 'proteomics' summarizes several technologies for visualization, quantitation and identification of these proteins. Recent advances in these techniques are helping to elucidate platelet processes which are relevan...

journal_title：Current opinion in hematology

authors： Senzel L,Gnatenko DV,Bahou WF

更新日期：2009-09-01 00:00:00

abstract：PURPOSE OF REVIEW:To discuss how mechanical cues and Rho signaling contribute to control of vascular development and hematopoiesis. RECENT FINDINGS:Rho guanine trinucleotide phosphatases are ubiquitious regulators of cytoskeletal structure and tension generation. Recent work shows that Rho-dependent mechanical interac...

journal_title：Current opinion in hematology

authors： Mammoto A,Mammoto T,Ingber DE

更新日期：2008-05-01 00:00:00

abstract：:Hematopoietic stem cells, defined as cells with extensive self-renewal and pluripotent differentiation potential, represent a minor population of adult bone marrow (< 1 in 10(4) to 10(5) nucleated marrow cells). Recent advances in cell surface phenotype analysis and separation technology have permitted enrichment of h...

journal_title：Current opinion in hematology

authors： Moore MA,Shapiro F

更新日期：1994-05-01 00:00:00

abstract：:The Epstein-Barr virus is a ubiquitous human herpesvirus that is associated with an increasing number of human malignancies. Among these are Epstein-Barr virus-associated lymphoproliferative diseases in immunocompromised patients, a spectrum of mainly B-cell diseases that range from polyclonal lymphoproliferative dise...

journal_title：Current opinion in hematology

authors： Mosier DE

更新日期：1999-01-01 00:00:00

abstract：PURPOSE OF REVIEW:The 'mobilization' of hematopoietic stem cells (HSCs) out of the bone marrow and into the peripheral blood is used clinically to obtain HSCs for transplantation. Although generally successful, mobilization protocols remain imperfect and the mechanisms involved are not fully understood. This review dis...

journal_title：Current opinion in hematology

authors： Bendall LJ,Basnett J

更新日期：2013-07-01 00:00:00

abstract：:This article presents new insights into potential roles that three erythrocyte cytoskeletal proteins, protein 4.1, ankyrin, and spectrin, may play in nonerythroid nucleated cells. Each of these proteins is encoded by several closely related genes characterized by complex alternative splicing of their pre-mRNA, thus re...

journal_title：Current opinion in hematology

authors： Gascard P,Mohandas N

更新日期：2000-03-01 00:00:00

abstract：:Following on the heels of multiple failed clinical trials of adjunctive therapeutic agents in sepsis, the positive outcome of a recent phase III study using activated protein C (APC) has led to a renewed optimism in targeted biotherapies for this syndrome. A growing body of data (both preclinical and clinical) suggest...

journal_title：Current opinion in hematology

authors： Key NS,Ely EW

更新日期：2002-09-01 00:00:00

abstract：PURPOSE OF REVIEW:The aim of this short study is to review recently published data bearing on how to resuscitate massive uncontrolled hemorrhage. RECENT FINDINGS:New data inform our understanding of the mechanisms of the acute coagulopathy of trauma, the median time to death of trauma patients with uncontrolled hemorr...

journal_title：Current opinion in hematology

authors： Murphy CH,Hess JR

更新日期：2015-11-01 00:00:00

abstract：PURPOSE OF REVIEW:Microtransplantation (MST) has been successfully used to manage patients with hematological malignancies, especially older patients with acute myeloid leukemia (AML). Recent clinical applications and mechanistic studies of MST are reviewed. RECENT FINDINGS:MST improves complete remission rates which ...

journal_title：Current opinion in hematology

authors： Cai B,Guo M,Ai H

更新日期：2018-11-01 00:00:00

abstract：PURPOSE OF REVIEW:This review discusses the rationale, efficacy, and toxicity of a variety of immune approaches being evaluated in the therapy of acute myeloid leukemia (AML) including naked and conjugated monoclonal antibodies, bispecific T-cell engager antibodies, and immune checkpoint blockade via antibodies targeti...

journal_title：Current opinion in hematology

authors： Assi R,Kantarjian H,Ravandi F,Daver N

更新日期：2018-03-01 00:00:00

abstract：PURPOSE OF REVIEW:Glycogen storage disease Ib (GSD Ib) is characterized by hepatomegaly, hypoglycemia, neutropenia, enterocolitis and recurrent bacterial infections. It is attributable to mutations in G6PT1, the gene for the glucose-6-phosphate transporter responsible for transport of glucose into the endoplasmic retic...

journal_title：Current opinion in hematology

authors： Dale DC,Bolyard AA,Marrero T,Kelley ML,Makaryan V,Tran E,Leung J,Boxer LA,Kishnani PS,Austin S,Wanner C,Ferrecchia IA,Khalaf D,Maze D,Kurtzberg J,Zeidler C,Welte K,Weinstein DA

更新日期：2019-01-01 00:00:00

abstract：:Cytomegalovirus (CMV) infection remains a major cause of morbidity and mortality in recipients of an allogeneic stem cell transplant (SCT). Due to the broad application of antiviral prophylaxis and preemptive therapy, a decrease in early-onset and a subsequent increase in late-onset CMV disease has been observed. New ...

journal_title：Current opinion in hematology

authors： Hebart H,Einsele H

更新日期：1998-11-01 00:00:00