Marina Cavazzana
- Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
2009/11/05 by Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae +23 · 13 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Virus-based gene therapy research #Peroxisome Proliferator-Activated Receptors #Neurogenetic and Muscular Disorders Research
- Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
2010/09/01 by Marina Cavazzana, Marina Cavazzana-Calvo, Emmanuel Payen +47 · 6 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Hemoglobinopathies and Related Disorders #RNA Interference and Gene Delivery #Virus-based gene therapy research
- Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
2018/04/18 by Alexis A. Thompson, Mark C. Walters, Janet Kwiatkowski +54 · 29 citations
Medicine · #Hemoglobinopathies and Related Disorders #Erythrocyte Function and Pathophysiology #Parvovirus B19 Infection Studies
- Gene Therapy in a Patient with Sickle Cell Disease
2017/03/01 by Jean‐Antoine Ribeil, Jean-Antoine Ribeil, Salima Hacein-Bey-Abina +33 · 30 citations
Medicine · Biochemistry, Genetics and Molecular Biology · #Hemoglobinopathies and Related Disorders #Parvovirus B19 Infection Studies #Virus-based gene therapy research
- Gene therapy for severe combined immunodeficiency: are we there yet?
2007/06/01 by Marina Cavazzana, Alain Fischer · 3 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Virus-based gene therapy research #RNA Interference and Gene Delivery #CAR-T cell therapy research
- X-SCID transgene leukaemogenicity
2006/09/01 by Adrian J. Thrasher, H. Bobby Gaspar, Christopher Baum +15 · 2 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #CAR-T cell therapy research #RNA Interference and Gene Delivery #Virus-based gene therapy research
- 20 years of gene therapy for SCID
2010/05/18 by Alain Fischer, Salima Hacein-Bey-Abina, Salima Hacein‐Bey‐Abina +2 · 4 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #CAR-T cell therapy research #RNA Interference and Gene Delivery #Virus-based gene therapy research
- The future of gene therapy
2004/02/01 by Marina Cavazzana-Calvo, Marina Cavazzana, Adrian J. Thrasher +2 · 1 citation
Biochemistry, Genetics and Molecular Biology · Engineering · Medicine · #Biomedical and Engineering Education #CAR-T cell therapy research #Virus-based gene therapy research