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1. Full Text Gene Therapy Studies in Hemoglobinopathies: Successes and Challenges
by Cavazzana, Marina
Blood, ISSN 0006-4971, 12/2016, Volume 128, Issue 22, pp. SCI-50 - SCI-50
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2. Full Text Hematopoietic Stem Cell Gene Therapy: Progress on the Clinical Front
by Cavazzana, Marina
Human gene therapy, ISSN 1043-0342, 03/2014, Volume 25, Issue 3, pp. 165 - 170
Pioneer Perspective | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Animals | Hematopoietic Stem Cells - cytology | Humans | Hematopoietic Stem Cell Transplantation | Hematopoietic Stem Cells - metabolism | Cell- and Tissue-Based Therapy | Genetic Therapy - trends | Genetic Therapy - adverse effects | Genetic Therapy - methods | Index Medicus
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3. Full Text Gene Therapy for β-Hemoglobinopathies
by Cavazzana, Marina and Antoniani, Chiara and Miccio, Annarita
Molecular therapy, ISSN 1525-0016, 05/2017, Volume 25, Issue 5, pp. 1142 - 1154
hematopoietic stem cell | gene therapy | thalassemias | hemoglobinopathies | sickle cell disease | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Anemia, Sickle Cell - pathology | beta-Thalassemia - pathology | Lentivirus - immunology | Humans | beta-Thalassemia - genetics | Transplantation, Homologous | Lentivirus - genetics | Gammaretrovirus - immunology | Gene Expression | beta-Thalassemia - therapy | Genetic Vectors - chemistry | Genetic Vectors - immunology | Hematopoietic Stem Cell Transplantation | Hematopoietic Stem Cells - metabolism | beta-Globins - genetics | Anemia, Sickle Cell - therapy | Gammaretrovirus - genetics | beta-Globins - metabolism | Anemia, Sickle Cell - metabolism | Hematopoietic Stem Cells - cytology | Gene Editing - methods | HLA Antigens | Mutation | Tissue Donors | beta-Thalassemia - metabolism | Anemia, Sickle Cell - genetics | Genetic Therapy - methods | Transcription factors | Disease | Hematology | Leukocyte migration | Anemia | Thalassemia | Genomes | Transplantation | Gene expression | Patients | Hematopoietic stem cells | Hemopoiesis | Genotype & phenotype | Efficiency | Stem cells | Cell cycle | Hemoglobin | Histocompatibility antigen HLA | Gene therapy | Sickle cell disease | Binding sites | Chronic illnesses | Index Medicus | Review
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4. Full Text Gene Therapy for Hemoglobinopathies
by Cavazzana, Marina and Mavilio, Fulvio
Human gene therapy, ISSN 1043-0342, 10/2018, Volume 29, Issue 10, pp. 116 - 1113
Reviews | thalassemia | sickle-cell disease | globin genes | lentiviral vectors | gene editing | Genetics & Heredity | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Autografts | Effectiveness | Transcription | Toxicity | Thalassemia | Transplantation | Regulatory sequences | Gene expression | Hematopoietic stem cells | Hemopoiesis | Stem cells | Safety | Gene therapy | Genetic modification | Expression vectors
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5. Full Text Lentiviral and genome-editing strategies for the treatment of beta-hemoglobinopathies
by Magrin, Elisa and Miccio, Annarita and Cavazzana, Marina
Blood, ISSN 0006-4971, 10/2019, Volume 134, Issue 15, pp. 1203 - 1213
Life Sciences & Biomedicine | Hematology | Science & Technology | beta-Thalassemia - therapy | Humans | beta-Thalassemia - genetics | Clinical Trials as Topic | Genetic Vectors - therapeutic use | beta-Globins - genetics | Genetic Vectors - genetics | Hemoglobinopathies - therapy | Anemia, Sickle Cell - therapy | Animals | Fetal Hemoglobin - genetics | Hemoglobinopathies - genetics | Lentivirus - genetics | Gene Editing - methods | Anemia, Sickle Cell - genetics | Genetic Therapy - adverse effects | Genetic Therapy - methods | Index Medicus | Abridged Index Medicus
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6. Full Text Gene therapy for severe combined immunodeficiency: Are we there yet?
by Cavazzana-Calvo, Marina and Fischer, Alain
The Journal of clinical investigation, ISSN 0021-9738, 06/2007, Volume 117, Issue 6, pp. 1456 - 1465
Life Sciences & Biomedicine | Medicine, Research & Experimental | Science & Technology | Research & Experimental Medicine | Humans | Hematopoietic Stem Cell Transplantation | Severe Combined Immunodeficiency - therapy | Transplantation, Autologous | Clinical Trials as Topic | Severe Combined Immunodeficiency - immunology | Genetic Therapy - trends | Transplantation, Homologous | Severe Combined Immunodeficiency - classification | Severe Combined Immunodeficiency - genetics | Recombination, Genetic | DNA Repair | Bone Marrow Transplantation | Mutation | Safety | Genetic Vectors | Genetic Therapy - adverse effects | Genetic Therapy - methods | Transplantation | Gene therapy | Drug therapy | Health aspects | Immunodeficiency | Stem cells | Index Medicus | Abridged Index Medicus | Science in Medicine
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7. Full Text Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges
by Cavazzana, Marina and Bushman, Frederic D and Miccio, Annarita and André-Schmutz, Isabelle and Six, Emmanuelle
Nature reviews. Drug discovery, ISSN 1474-1776, 06/2019, Volume 18, Issue 6, pp. 447 - 462
Pharmacology & Pharmacy | Life Sciences & Biomedicine | Biotechnology & Applied Microbiology | Science & Technology | beta-Thalassemia - therapy | Genetic Vectors - administration & dosage | Humans | beta-Thalassemia - genetics | Hematopoietic Stem Cell Transplantation | Severe Combined Immunodeficiency - therapy | Anemia, Sickle Cell - therapy | Severe Combined Immunodeficiency - genetics | Animals | Genetic Engineering | Hematopoietic Stem Cells - cytology | Anemia, Sickle Cell - genetics | Genetic Therapy - methods | Gene therapy | Vectors (Biology) | Genetic engineering | Index Medicus
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8. Full Text Innovations Needed for Effective Implementation of Ex Vivo Gene Therapies
by Cavazzana, Marina
Frontiers in medicine, ISSN 2296-858X, 2017, Volume 4, pp. 29 - 29
Forecasts and trends | Genetic aspects | Gene therapy | Health aspects | Methods | Hematopoietic stem cells | genome-editing technology | dyserythropoiesis | gene therapy | Wiskott–Aldrich syndrome | hematopoietic stem progenitor cells | hemoglobinopathies
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