Area of research
Genetics · Pediatrics, Perinatology and Child Health
Research interest
Research interests include Hemoglobinopathies and Related Disorders, Prenatal Screening and Diagnostics, Virus-based gene therapy research, and Acute Lymphoblastic Leukemia research.
Prime Editing for p47 <sup>phox</sup> -Deficient Chronic Granulomatous Disease
Cedar Trial in Progress: A First in Human, Phase 1/2 Study of the Correction of a Single Nucleotide Mutation in Autologous HSCs (GPH101) to Convert HbS to HbA for Treating Severe SCD
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia
Lentiglobin Gene Therapy for Transfusion-Dependent β-Thalassemia: Update from the Northstar Hgb-204 Phase 1/2 Clinical Study
Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product)
Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βΑ-T87Q -Globin Vector (LentiGlobin BB305 Drug Product)