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91 : Editing the LRF Repressor Binding Site in the γ-Globin Promoters Induces Therapeutically Relevant Fetal Hemoglobin Levels for the Treatment of β-Hemoglobinopathies

Researchers

Presenter

  • Giacomo Frati

Principal Investigators

  • Giulia Hardouin

  • Bochra Mlaya

  • Leslie Weber

  • Tristan Felix

  • Antonio Casini

  • Marion Rosello

  • Anne Chalumeau

  • Mario Amendola

  • Adrian J. Thrasher

  • Giandomenico Turchiano

  • Filippo Del Bene

  • Wassim El Nemer

  • Anna Cereseto

  • Jean-Paul Concordet

  • Carine Giovannangeli

  • Marina Cavazzana

  • Annarita Miccio

Medical Centers

  • Imagine Institute, Laboratory of chromatin and gene regulation during development, INSERM UMR1163, Paris, France

  • University Paris-Sud, Paris, France

  • Imagine Institute, INSERM UMR1163, Paris, France, Paris, France

  • Imagine Institute, Paris, FRA

  • Curie Institute, Paris, France

  • University of Trento, Trento, Italy

  • Institut de lavision, Paris, France

  • R&D, Genethon, Evry, France

  • Great Ormond Street Institute of Child Health, UCL, London, United Kingdom

  • UCL GOS Institute of Child Health Molecular and Cellular Immunology, LOndon, United Kingdom

  • Integrated Biology of the Red Cell, INSERM Unit 1134, Paris, France

  • Laboratoire dexcellence Gr-Ex, Paris, France

  • INSERM U565, CNRS UMR7196, Museum National d'Histoire Naturelle, Paris, France, Paris, France

  • INSERM U565, CNRS UMR7196, Museum National d'Histoire Naturelle, Paris, France, Paris, France

  • Biotherapy Department, Hopital Necker Enfants Malades, Paris, France

  • INSERM U1016, IMAGINE Institute, Universite Paris Descartes, Sorbonne Paris Cite, Paris, France

Locations

  • France

  • Italy

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Blood/haematological Diseases

Disease

  • Sickle cell anemia

  • Thalassemia

Biomarkers

  • Red Blood Cells

Drug/Treatment

  • N/A

Outcome

  • N/A

Study Design

  • N/A

Phase

  • NA

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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