相关参考文献
注意:仅列出部分参考文献,下载原文获取全部文献信息。β-Thalassemias
Ali T. Taher et al.
NEW ENGLAND JOURNAL OF MEDICINE (2021)
Patient- and Caregiver-Reported Burden of Transfusion-Dependent β-Thalassemia Measured Using a Digital Application
Clark Paramore et al.
PATIENT-PATIENT CENTERED OUTCOMES RESEARCH (2021)
The changing epidemiology of the ageing thalassaemia populations: A position statement of the Thalassaemia International Federation
Dimitrios Farmakis et al.
EUROPEAN JOURNAL OF HAEMATOLOGY (2020)
Myelodysplastic syndrome unrelated to lentiviral vector in a patient treated with gene therapy for sickle cell disease
Matthew M. Hsieh et al.
BLOOD ADVANCES (2020)
Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy
Stefan Radtke et al.
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2020)
Health-related quality of life (HRQoL) in beta-thalassemia major (β-TM) patients assessed by 36-item short form health survey (SF-36): a meta-analysis
Mahdieh Arian et al.
QUALITY OF LIFE RESEARCH (2019)
Chemistry, manufacturing and controls for gene modified hematopoietic stem cells
Sandeep Soni et al.
CYTOTHERAPY (2019)
Long-Term Clinical Outcomes of Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia in the Northstar (HGB-204) Study
Janet L. Kwiatkowski et al.
BLOOD (2019)
Results from the Completed Hgb-205 Trial of Lentiglobin for β-Thalassemia and Lentiglobin for Sickle Cell Disease Gene Therapy
Elisa Magrin et al.
BLOOD (2019)
Related and unrelated donor transplantation for β-thalassemia major: results of an international survey
Chunfu Li et al.
BLOOD ADVANCES (2019)
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
A. A. Thompson et al.
NEW ENGLAND JOURNAL OF MEDICINE (2018)
How I manage medical complications of β-thalassemia in adults
Ali T. Taher et al.
BLOOD (2018)
Improving Gene Therapy Efficiency through the Enrichment of Human Hematopoietic Stem Cells
Katelyn E. Masiuk et al.
MOLECULAR THERAPY (2017)
A Guide to Approaching Regulatory Considerations for Lentiviral-Mediated Gene Therapies
Michael White et al.
HUMAN GENE THERAPY METHODS (2017)
Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy
Erika Zonari et al.
STEM CELL REPORTS (2017)
A Multicenter Retrospective Analysis Stressing the Importance of Long-Term Follow-Up after Hematopoietic Cell Transplantation for β-Thalassemia
Sonali Chaudhury et al.
BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2017)
Hematopoietic Stem Cell Gene Therapy: Progress and Lessons Learned
Richard A. Morgan et al.
CELL STEM CELL (2017)
Hemopoietic stem cell transplantation in thalassemia: a report from the European Society for Blood and Bone Marrow Transplantation Hemoglobinopathy Registry, 2000-2010
D. Baronciani et al.
BONE MARROW TRANSPLANTATION (2016)
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the βA(T87Q)-Globin Gene
Olivier Negre et al.
HUMAN GENE THERAPY (2016)
Guidelines for the Standard Monitoring of Patients With Thalassemia: Report of the Thalassemia Longitudinal Cohort
Venee N. Tubman et al.
JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY (2015)
Preclinical Evaluation of Efficacy and Safety of an Improved Lentiviral Vector for the Treatment of β-Thalassemia and Sickle Cell Disease
Olivier Negre et al.
CURRENT GENE THERAPY (2014)
Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel
Emanuele Angelucci et al.
HAEMATOLOGICA (2014)
Biosafety Features of Lentiviral Vectors
Axel Schambach et al.
HUMAN GENE THERAPY (2013)
Lentiviral vectors: basic to translational
Toshie Sakuma et al.
BIOCHEMICAL JOURNAL (2012)
Quality of life in Thalassemia: A comparison of SF-36 results from the Thalassemia longitudinal cohort to reported literature and the US norms
A. Sobota et al.
AMERICAN JOURNAL OF HEMATOLOGY (2011)
Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
Stefan Stein et al.
NATURE MEDICINE (2010)
Long-term results of survival in patients with thalassemia major treated with bone marrow transplantation
Paolo Di Bartolomeo et al.
AMERICAN JOURNAL OF HEMATOLOGY (2008)
Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
Salima Hacein-Bey-Abina et al.
JOURNAL OF CLINICAL INVESTIGATION (2008)
A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency
S Hacein-Bey-Abina et al.
NEW ENGLAND JOURNAL OF MEDICINE (2003)
Cardiovascular T2-star (T2*) magnetic resonance for the early diagnosis of myocardial iron overload
LJ Anderson et al.
EUROPEAN HEART JOURNAL (2001)