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Gene Therapy Approaches to Hemoglobinopathies.
Giuliana Ferrari1, Marina Cavazzana2, Fulvio Mavilio3
1San Raffaele-Telethon Institute for Gene Therapy (SR-TIGET), Istituto Scientifico Ospedale San Raffaele, Via Olgettina 58, Milan 20132, Italy; Vita-Salute San Raffaele University, Milan, Italy.
Hematology/Oncology Clinics of North America
|September 13, 2017
Summary
Gene therapy using lentiviral vectors shows promise for hemoglobinopathies like beta-thalassemia and sickle cell disease. Overcoming bone marrow microenvironment challenges is crucial for widespread clinical application.
Area of Science:
- * Hematology
- * Gene Therapy
- * Molecular Biology
Background:
- * Current gene therapy for hemoglobinopathies involves autologous hematopoietic stem cell transplantation.
- * Lentiviral vectors are used to genetically modify stem cells, expressing therapeutic globin genes.
- * This approach has demonstrated safety and potential efficacy in preclinical and early clinical studies.
Purpose of the Study:
- * To review the current state of gene therapy for hemoglobinopathies.
- * To identify existing hurdles limiting the general application of this treatment.
- * To highlight the impact of the bone marrow microenvironment on therapeutic efficiency.
Main Methods:
- * Review of preclinical and early clinical studies on gene therapy for hemoglobinopathies.
- * Analysis of lentiviral vector technology and globin gene regulation.
- * Examination of the bone marrow microenvironment's role in stem cell transplantation.
Main Results:
- * Gene therapy shows safety and potential efficacy for treating hemoglobinopathies.
- * Hurdles remain, preventing widespread clinical use.
- * An altered bone marrow microenvironment negatively impacts stem cell harvesting and engraftment in beta-thalassemia and sickle cell disease.
Conclusions:
- * Gene therapy holds significant potential for hemoglobinopathies.
- * Addressing challenges related to stem cell mobilization and engraftment is essential.
- * Improving the bone marrow microenvironment is critical for successful gene therapy application.
Keywords:
Gene transferGlobin gene regulationHematopoiesisLentiviral vectorsRetroviral vectorsSickle cell diseaseStem cell transplantationThalassemiaMore Related Videos
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