Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Experiment Videos

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
Summary

Related Concept Videos

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the <i>HBG1/2</i> promoters.

Molecular therapy. Nucleic acids·2026
Same author

Nationwide Centralized Manufacturing and Fresh Distribution of TCRαβ/CD19-DepletedGrafts: A Real-World French Experience.

Transplantation and cellular therapy·2026
Same author

CleanFinder: a scalable framework for comprehensive genome editing analysis.

Trends in biotechnology·2026
Same author

Tracking HIV-1 DNA fate from cell culture to humanized mice tissues.

Molecular therapy. Advances·2026
Same author

Splenectomy in β-thalassemia patients: Practices and risks in a nationwide study.

HemaSphere·2026
Same author

Clinical application of base editing for treating β-thalassaemia.

Nature·2026

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.
Keywords:
gene therapyhematopoietic stem cellhemoglobinopathiessickle cell diseasethalassemias

Related Experiment Videos

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.