当前位置： SCI文献检索 > INTERNATIONAL JOURNAL OF HEMATOLOGY期刊下所有文献 > Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world.

Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world.

Abstract：

:Gene therapies are classified into two major categories, namely, in vivo and ex vivo. Clinical trials of human gene therapy began with the ex vivo techniques. Based on the initial successes of gene-therapy clinical trials, these approaches have spread worldwide. The number of gene therapy trials approved worldwide increased gradually starting in 1989, reaching 116 protocols per year in 1999, and a total of 2210 protocols had been approved by 2015. Accumulating clinical evidence has demonstrated the safety and benefits of several types of gene therapy, with the exception of serious adverse events in several clinical trials. These painful experiences were translated backward to basic science, resulting in the development of several new technologies that have influenced the recent development of ex vivo gene therapy in this field. To date, six gene therapies have been approved in a limited number of countries worldwide. In Japan, clinical trials of gene therapy have developed under the strong influence of trials in the US and Europe. Since the initial stages, 50 clinical trials have been approved by the Japanese government. In this review, the history and current status of clinical trials of ex vivo gene therapy for hematological disorders are introduced and discussed.

journal_name

Int J Hematol

journal_title

International journal of hematology

authors

Tani K

doi

10.1007/s12185-016-2030-2

subject

Has Abstract

pub_date

2016-07-01 00:00:00

pages

42-72

issue

1

eissn

0925-5710

issn

1865-3774

pii

10.1007/s12185-016-2030-2

journal_volume

104

pub_type

杂志文章,评审

相关文献

INTERNATIONAL JOURNAL OF HEMATOLOGY文献大全
  • Outcome of reduced-intensity allogeneic hematopoietic stem cell transplantation for multiple myeloma.

    abstract：:We retrospectively evaluated single-institute outcomes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) with a reduced-intensity conditioning regimen consisting of fludarabine (125 mg/m²) and melphalan (140 mg/m²) for multiple myeloma. Twenty-three patients (median age: 46 years) were evaluated. Stem ...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-015-1873-2

    authors： Kikuchi T,Mori T,Koda Y,Kohashi S,Kato J,Toyama T,Nakazato T,Aisa Y,Shimizu T,Okamoto S

    更新日期：2015-12-01 00:00:00

  • Importance of CD4+ helper T-cells in antitumor immunity.

    abstract：:CD8+ cytotoxic T-lymphocytes are major effector cells involved in immunologically specific tumor destruction in vivo, and CD4+ T-cells are essential for controlling this CD8+ T-cell-dependent tumor eradication. The presence of CD4+ T-cells with distinct functional roles has been recognized. The further understanding o...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1007/BF02986610

    authors： Shiku H

    更新日期：2003-06-01 00:00:00

  • Correlation between immunological abnormalities and prognosis in myelodysplastic syndrome patients.

    abstract：:Immunological abnormalities (IA) are frequently observed in patients with myelodysplastic syndromes (MDS). Although there have been a number of analyses of the prognostic factors, there have been few studies, if any, to determine whether IA affects prognosis. We investigated the prognosis of 153 MDS patients with or w...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1016/s0925-5710(97)00042-x

    authors： Okamoto T,Okada M,Mori A,Saheki K,Takatsuka H,Wada H,Tamura A,Fujimori Y,Takemoto Y,Kanamaru A,Kakishita E

    更新日期：1997-10-01 00:00:00

  • Somatic thrombopoietin (THPO) gene mutations in childhood myeloid leukemias.

    abstract：:We report, for the first time, a non-syndromic infant with a reversible myeloproliferative disease that harbors a germline hereditary thrombopoietin (THPO) gene mutation, a condition that is known to induce familial thrombocytosis at increasing age. In order to investigate whether somatic THPO gene mutations play a ro...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-015-1759-3

    authors： Houwing ME,Koopman-Coenen EA,Kersseboom R,Gooskens S,Appel IM,Arentsen-Peters ST,de Vries AC,Reinhardt D,Stary J,Baruchel A,de Haas V,Blink M,Lopes Cardozo RH,Pieters R,Michel Zwaan C,van den Heuvel-Eibrink MM

    更新日期：2015-07-01 00:00:00

  • Frequent expression of human Thy-1 antigen on pre-B cell acute lymphoblastic leukemia with t(9;22).

    abstract：:Thy-1 (CDw90) is a phosphatidylinositol-anchored protein, and is expressed on human pluripotential hematopoietic stem cells. The expression pattern of this antigen on leukemia cells is still controversial. In this study, 72 adult patients with pre-B cell acute lymphoblastic leukemia (pre-B ALL) were examined for the e...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1016/s0925-5710(98)00021-8

    authors： Takahashi T,Mizutani M,Miwa H,Katayama N,Nishii K,Shikami M,Yamaguchi M,Shiku H,Kamada N,Kita K

    更新日期：1998-06-01 00:00:00

  • Association between OGG1 S326C CC genotype and elevated relapse risk in acute myeloid leukemia.

    abstract：:Recent studies have shown that tumors of relapsed acute myeloid leukemia (AML) present additional genetic mutations compared to the primary tumors. The base excision repair (BER) pathway corrects oxidatively damaged mutagenic bases and plays an important role in maintaining genetic stability. The purpose of the presen...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-018-2464-9

    authors： Gotoh N,Saitoh T,Takahashi N,Kasamatsu T,Minato Y,Lobna A,Oda T,Hoshino T,Sakura T,Shimizu H,Takizawa M,Handa H,Yokohama A,Tsukamoto N,Murakami H

    更新日期：2018-09-01 00:00:00

  • Superior mesenteric venous thrombosis associated with a familial missense mutation (Pro626Leu) in the SHBG-like domain of the protein S molecule.

    abstract：:A 76-year-old Japanese woman was hospitalized for ileus symptoms caused by extensive thrombosis of the superior mesenteric vein. Because laboratory test results suggested type III protein S (PS) deficiency, molecular changes in PS were investigated. A single-base transition, CCG to CTG at codon 626 in exon XV, resulti...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/BF02981988

    authors： Kato M,Iida S,Sato M,Hayami Y,Hanamura I,Miura K,Ito M,Harada S,Komatsu H,Wakita A,Manabe T,Ueda R

    更新日期：2002-01-01 00:00:00

  • Early electrocardiographic evaluation of atrial fibrillation risk in beta-thalassemia major patients.

    abstract：:Although previous studies have documented a variety of electrocardiogram abnormalities in beta-thalassemia major (β-TM), little is known about P-wave dispersion (PD), an independent risk factor for development of atrial fibrillation. The aim of our study was to evaluate PD in β-TM patients with conserved systolic and ...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-011-0801-3

    authors： Russo V,Rago A,Pannone B,Di Meo F,Papa AA,Mayer MC,Spasiano A,Russo MG,Golino P,Calabrò R,Nigro G

    更新日期：2011-04-01 00:00:00

  • Systemic HD-MTX for CNS prophylaxis in high-risk DLBCL patients: a prospectively collected, single-center cohort analysis.

    abstract：:We evaluated the efficacy of systemic high-dose methotrexate (HD-MTX) for CNS prophylaxis in a prospectively recruited cohort of DLBCL patients at high risk of CNS relapse. High-risk CNS relapse was defined as the involvement of ≥ 2 extranodal sites with elevated lactate dehydrogenase (LDH); CNS international prognost...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-019-02653-7

    authors： Lee K,Yoon DH,Hong JY,Kim S,Lee K,Kang EH,Huh J,Park CS,Lee SW,Suh C

    更新日期：2019-07-01 00:00:00

  • Successful treatment of two patients with primary cardiac malignant lymphoma.

    abstract：:We describe two patients with primary cardiac malignant lymphoma involving the right atrium and superior vena cava, resulting in intractable right cardiac failure and superior vena cava syndrome. Patients were diagnosed by surgical myocardial biopsy and were treated with combination chemotherapy for non-Hodgkin's lymp...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：

    authors： Enomoto S,Abo T,Sugawara T,Ishida Y,Murai K,Itoh S,Kuriya S

    更新日期：1999-10-01 00:00:00

  • N-(4-Hydroxyphenyl)retinamide (4-HPR) induces leukemia cell death via generation of reactive oxygen species.

    abstract：:The role of reactive oxygen species (ROS) in the cytotoxicity of N-(4-hydroxyphenyl)retinamide (4-HPR) was studied with use of the B-precursor lymphoblastic leukemia cell line YCUB-2. The increase in intracellular ROS measured with 2'-7'-dichlorodihydrofluorescein diacetate after 3 hours' incubation was 3.7-fold with ...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/BF02983798

    authors： Goto H,Takahashi H,Fujii H,Ikuta K,Yokota S

    更新日期：2003-10-01 00:00:00

  • Hematopoiesis and angiogenesis.

    abstract：:Hematopoiesis is closely linked with angiogenesis, because they interact with each other and have common ancestors: hemangioblasts or hematogenic endothelial cells. The relationship is reasonable, because vascular and hematopoietic systems must develop together in order to establish the body's oxygen-delivery system d...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：

    authors： Suda T,Takakura N,Oike Y

    更新日期：2000-02-01 00:00:00

  • Multiple myeloma presenting as bilateral breast lumps in pregnant woman.

    abstract：BACKGROUND:Multiple myeloma (MM) is a systemic disease in the elderly. Its incidence in patients younger than 40 years old and especially in pregnant women is extremely rare. MM may involve extraosseous sites, and only in rare cases it is observed in the breast. CASE REPORT:We describe the case of a 39-year-old woman ...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-013-1420-y

    authors： Bouzguenda R,Khanfir A,Toumi N,Chaaben K,Hentati Y,Ayadi L,Feki W,Daoud J,Frikha M

    更新日期：2013-10-01 00:00:00

  • Aortic thrombosis resolved with enoxaparin in a patient treated with cisplatin-based regimen for small cell lung cancer.

    abstract：:We present a rare case of a thrombus at the aortic arch found 1 month after cisplatin-based chemotherapy in a 50-year-old patient with a diagnosis of small cell lung cancer; there were no symptoms related to the thrombus. This patient did not have any predisposing factors for the development of an aortic thrombus befo...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-010-0571-3

    authors： Chin SO,Lee JJ,Hwang YH,Han JJ,Maeng CH,Baek SK,Choi CW

    更新日期：2010-06-01 00:00:00

  • Pathogenesis of graft-versus-host disease: innate immunity amplifying acute alloimmune responses.

    abstract：:In addition to reduced-intensity conditioning, which has expanded the eligibility for hematopoietic cell transplantation (HCT) to older patients, increased availability of alternative donors, including HLA-mismatched unrelated donors, has increased access to allogeneic HCT for more patients. However, acute graft-versu...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1007/s12185-013-1421-x

    authors： Maeda Y

    更新日期：2013-09-01 00:00:00

  • DNA methylation as a therapeutic target in hematologic disorders: recent results in older patients with myelodysplasia and acute myeloid leukemia.

    abstract：:DNA methylation provides a major epigenetic code (besides histone modification) of the lineage- and development-specific genes (such as regulators of differentiation in the hematopoietic lineages) that control expression of normal cells. However, DNA methylation is also involved in malignancies because aberrant methyl...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1532/ijh97.04094

    authors： Rüter B,Wijermans PW,Lübbert M

    更新日期：2004-08-01 00:00:00

  • Evi-1 as a critical regulator of leukemic cells.

    abstract：:Ecotropic viral integration site-1 (EVI-1) has been recognized as one of the dominant oncogenes associated with murine and human myeloid leukemia. Recent clinical studies demonstrated that high EVI-1 expression was an independent negative prognostic indicator of survival in leukemia patients. In addition, gene-targeti...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1007/s12185-010-0618-5

    authors： Goyama S,Kurokawa M

    更新日期：2010-06-01 00:00:00

  • Combination of high-dose melphalan and bortezomib as conditioning regimen for autologous peripheral blood stem cell transplantation in multiple myeloma.

    abstract：:Bortezomib and melphalan have synergistic effects against multiple myeloma (MM) cells. We conducted a pilot study on the combination of bortezomib and high-dose melphalan (Bor-HDM) as a conditioning regimen followed by autologous stem cell transplant (ASCT) in 17 Japanese patients with newly diagnosed MM, in compariso...

    journal_title：International journal of hematology

    pub_type： 临床试验,杂志文章,多中心研究

    doi：10.1007/s12185-013-1402-0

    authors： Miyamoto T,Yoshimoto G,Kamimura T,Muta T,Takashima S,Ito Y,Shiratsuchi M,Choi I,Kato K,Takenaka K,Iwasaki H,Takamatsu Y,Teshima T,Akashi K

    更新日期：2013-09-01 00:00:00

  • An unusual case of transient dermatological reaction to bortezomib in AL amyloidosis.

    abstract：:We report an unusual dermatological reaction to bortezomib in a 61-year-old man with AL amyloidosis. Systemic AL amyloidosis is a rare complication of monoclonal gammopathy or myeloma in which abnormally unstable free light chains cause fibrillary deposits in organs leading to multisystem disease. The treatment of AL ...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-009-0460-9

    authors： Shah AD,Watts AJ,Mehta AB,Wechalekar AD

    更新日期：2010-01-01 00:00:00

  • Ras/MAPK syndromes and childhood hemato-oncological diseases.

    abstract：:Noonan syndrome (NS) is an autosomal-dominant disease characterized by distinctive facial features, webbed neck, cardiac anomalies, short stature and cryptorchidism. NS exhibits phenotypic overlap with Costello syndrome and cardio-facio-cutaneous (CFC) syndrome. Germline mutations of genes encoding proteins in the RAS...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1007/s12185-012-1239-y

    authors： Aoki Y,Matsubara Y

    更新日期：2013-01-01 00:00:00

  • Persistent remission after immunosuppressive therapy of hairy cell leukemia mimicking aplastic anemia: two case reports.

    abstract：:Some patients with hairy cell leukemia (HCL) manifest pancytopenia and bone marrow hypoplasia without an apparent increase in atypical cells, so their disease resembles severe aplastic anemia at onset. We treated 2 HCL patients, who were initially diagnosed with aplastic anemia, with antithymocyte globulin (ATG) in co...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1007/BF02982650

    authors： Sugimori C,Kaito K,Nakao S

    更新日期：2003-05-01 00:00:00

  • Usefulness of antithrombin deficiency phenotypes for risk assessment of venous thromboembolism: type I deficiency as a strong risk factor for venous thromboembolism.

    abstract：:Inherited antithrombin deficiency, an established risk factor for venous thromboembolism (VTE), can be classified into type I (quantitative deficiency) or type II (qualitative deficiency). In the present study, we assessed the VTE risk associated with the phenotypes of antithrombin deficiency in patients admitted to o...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-010-0687-5

    authors： Mitsuguro M,Sakata T,Okamoto A,Kameda S,Kokubo Y,Tsutsumi Y,Sano M,Miyata T

    更新日期：2010-10-01 00:00:00

  • Tuberculosis-associated hemophagocytic syndrome in a hemodialysis patient with protracted fever.

    abstract：:Hemophagocytic syndrome (HPS) is a clinicopathological reflection of uncontrolled activation of macrophages. To our knowledge, only a few cases of tuberculosis-associated HPS in hemodialysis have been reported in the English literature. We report a case of tuberculosis-associated HPS during hemodialysis. Bone marrow a...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1532/ijh97.a10315

    authors： Chien CC,Chiou TJ,Lee MY,Hsiao LT,Kwang WK

    更新日期：2004-05-01 00:00:00

  • Sustained improvement in cardiac function with persistent amyloid deposition in a patient with multiple myeloma-associated cardiac amyloidosis treated with bortezomib.

    abstract：:We describe the case of a 62-year-old man with biopsy-proven cardiac involvement of multiple myeloma-associated immunoglobulin light-chain amyloidosis, whose cardiac function improved after bortezomib therapy. Angiotensin-converting enzyme inhibitors and diuretics were initially administered, resulting in improvement ...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-010-0710-x

    authors： Tamaki H,Naito Y,Lee-Kawabata M,Taniguchi Y,Hao H,Hirota S,Hasegawa S,Masuyama T,Ogawa H

    更新日期：2010-11-01 00:00:00

  • Hypocalcemic heart failure in thalassemic patients.

    abstract：:Hypocalcemic cardiomyopathy in primary or secondary hypoparathyroidism is usually refractory to conventional treatment of cardiac failure. We report the case of a thalassemic patient with severe cardiac failure that might have been attributed to several factors, such as hemosiderosis, hypomagnesemia, and hypocalcemia,...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1532/IJH97.E0532

    authors： Tsironi M,Korovesis K,Farmakis D,Deftereos S,Aessopos A

    更新日期：2006-05-01 00:00:00

  • Biology and clinical application of CAR T cells for B cell malignancies.

    abstract：:Chimeric antigen receptor (CAR)-modified T cells have generated broad interest in oncology following a series of dramatic clinical successes in patients with chemorefractory B cell malignancies. CAR therapy now appears to be on the cusp of regulatory approval as a cell-based immunotherapy. We review here the T cell bi...

    journal_title：International journal of hematology

    pub_type： 杂志文章,评审

    doi：10.1007/s12185-016-2039-6

    authors： Davila ML,Sadelain M

    更新日期：2016-07-01 00:00:00

  • A retrospective clinical analysis of Japanese patients with peripheral T-cell lymphoma not otherwise specified: Hokkaido Hematology Study Group.

    abstract：:Peripheral T-cell lymphoma not otherwise specified (PTCL-NOS) comprises a group of heterogeneous lymphomas that do not fit any other identified PTCL-subgroup and show poor prognosis. To clarify clinical aspects of Japanese PTCL-NOS patients, the Hokkaido Hematology Study Group conducted a multicenter retrospective ana...

    journal_title：International journal of hematology

    pub_type： 临床试验,杂志文章,多中心研究

    doi：10.1007/s12185-013-1383-z

    authors： Torimoto Y,Sato K,Ikuta K,Hayashi T,Hirayama Y,Inamura J,Kobayashi H,Kobayashi R,Koda K,Kurosawa M,Mori A,Ota S,Sakai H,Shigematsu A,Shindo M,Shinzaki H,Takahashi F,Takimoto R,Tanaka J,Yamamoto S,Kohgo Y,Fukuhar

    更新日期：2013-08-01 00:00:00

  • Diffuse follicular lymphoma variant with a typical diagnostic pattern and an unusually aggressive clinical presentation.

    abstract：:Diffuse follicular lymphoma (FL) variant is a rare condition that shows distinctive clinical, morphological, immunophenotypic, and molecular features that distinguish it from classical FL. Diffuse FL variant is characterized by a predominantly diffuse growth pattern, absence of the t (14;18) IGH/BCL2 translocation, CD...

    journal_title：International journal of hematology

    pub_type： 信件

    doi：10.1007/s12185-020-02901-1

    authors： Martín-Moro F,Marquet-Palomanes J,Piris-Villaespesa M,Lopez-Jiménez J,García-Cosío M

    更新日期：2020-08-01 00:00:00

  • Low proportion of G0-phase cells during induction chemotherapy correlates with subsequent remission in acute myeloid leukemia.

    abstract：:Kinetic resistance is assumed to be one of the main mechanisms of drug resistance in acute myeloid leukemia (AML), but the relationship between cell cycle status at diagnosis and achievement of complete remission (CR) is controversial. Based on the possibility that the cell cycle data after starting induction chemothe...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：

    authors： Kamikubo KH,Ogata K,An E,Dan K

    更新日期：2000-04-01 00:00:00

  • Response to cyclosporine A and corticosteroids in adult patients with acquired pure red cell aplasia: serial experience at a single center.

    abstract：:To assess response to cyclosporine A, and/or corticosteroids, and possible factors influencing the response in adult patients with acquired pure red cell aplasia (PRCA). Clinical data from 42 cases were retrospectively analyzed. These patients received cyclosporine A (CsA), and/or corticosteroids (CS), or other immuno...

    journal_title：International journal of hematology

    pub_type： 杂志文章

    doi：10.1007/s12185-018-2446-y

    authors： Wu X,Wang S,Lu X,Shen W,Qiao C,Wu Y,Lu R,Wang S,Zhang J,Hong M,Zhu Y,Li J,He G

    更新日期：2018-08-01 00:00:00