Lentiviral and genome-editing strategies for the treatment of β-hemoglobinopathies

Author:

Magrin Elisa12ORCID,Miccio Annarita34ORCID,Cavazzana Marina1245ORCID

Affiliation:

1. Biotherapy Department, Necker Children’s Hospital, Assistance Publique–Hôpitaux de Paris, Paris, France;

2. Biotherapy Clinical Investigation Center, Groupe Hospitalier Universitaire Ouest, Assistance Publique–Hôpitaux de Paris, INSERM CIC 1416, Paris, France;

3. Laboratory of Chromatin and Gene Regulation during Development, INSERM UMR_S1163, Paris, France;

4. Paris Descartes–Sorbonne Paris Cité University, Imagine Institute, Paris, France; and

5. Laboratory of Human Lymphohematopoiesis, INSERM UMR_S1163, Paris, France

Abstract

Abstract This review article summarizes the clinical outcomes and genotoxicity issues of gene therapy for hemoglobinopathies based on lentiviral vectors carrying a β-globin–like gene or genome editing to correct the erythropoietic defect.

Publisher

American Society of Hematology

Subject

Cell Biology,Hematology,Immunology,Biochemistry

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