Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.
Sign Up to like & getrecommendations! Published in 2022 at "Nature medicine"
DOI: 10.1038/s41591-021-01650-w
Abstract: Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are the most prevalent monogenic disorders worldwide. Trial HGB-205 ( NCT02151526 ) aimed at evaluating gene therapy by autologous CD34+ cells transduced ex vivo with lentiviral vector… read more here.
Keywords: long term; gene therapy; trial; hgb 205 ... See more keywords
Analysis of RBC Properties in Patients with SCD Treated with Lentiglobin Gene Therapy
Sign Up to like & getrecommendations! Published in 2018 at "Blood"
DOI: 10.1182/blood-2018-99-115973
Abstract: Introduction: Gene therapy is a highly promising therapeutic strategy in sickle cell disease (SCD). The Phase 1/2 HGB-205 (NCT02151526) clinical study in France is evaluating the safety and efficacy of LentiGlobin gene therapy, which consists… read more here.
Keywords: lentiglobin gene; gene therapy; hgb 205; patients scd ... See more keywords