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Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies
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Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies

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Gene Therapy for β-Hemoglobinopathies

Marina Cavazzana1, Chiara Antoniani2, Annarita Miccio2

  • 1Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hôpitaux de Paris, 75015 Paris, France; Biotherapy Clinical Investigation Center, Assistance Publique-Hôpitaux de Paris, INSERM, Groupe Hospitalier Universitaire Ouest, 75015 Paris, France; INSERM UMR 1163, Laboratory of Human Lymphohematopoiesis, 75015 Paris, France; Paris Descartes, Sorbonne Paris Cité University, Imagine Institute, 75015 Paris, France.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|April 6, 2017
PubMed

Insights

Gene therapy offers a promising alternative to hematopoietic stem cell transplantation for treating inherited blood disorders like beta-thalassemia and sickle cell disease, especially for patients lacking suitable donors.

Area of Science:

  • Hematology
  • Genetics
  • Translational Medicine

Background:

  • Beta-thalassemia and sickle cell disease are global hemoglobinopathies.
  • Allogeneic hematopoietic stem cell transplantation is the only definitive treatment.
  • Gene therapy is emerging as a viable alternative treatment.

Purpose of the Study:

  • To summarize clinical translation steps for gene therapy in hemoglobinopathies.
  • To discuss novel lentiviral and genome editing strategies.
  • To highlight gene therapy's potential for patients lacking HLA-identical donors.

Main Methods:

  • Review of current translational medicine efforts.
  • Discussion of lentiviral vector-based gene therapy.
  • Exploration of genome editing techniques.

Main Results:

  • Gene therapy is advancing towards clinical application for hemoglobinopathies.
  • Lentiviral and genome editing strategies show significant potential.
  • These advancements address limitations of current stem cell transplantations.

Conclusions:

  • Gene therapy is a promising treatment for beta-hemoglobinopathies.
  • It offers solutions for patients without HLA-identical donors.
  • Ongoing research in lentiviral and genome editing is crucial.

Keywords:
gene therapyhematopoietic stem cellhemoglobinopathiessickle cell diseasethalassemias

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