Sickle-cell disease: the first clinical trials soon?

Publié le : 9 July 2013

 Researchers from the University of California at Los Angeles (UCLA) have "successfully established the foundation for using hematopoietic (blood-producing) stem cells from the bone marrow of patients with sickle cell disease to treat the disease." In concrete terms, the researchers introduced an anti-sickling gene into the defective hematopoietic stem cells using lentiviruses. According to the results of the experiment, carried out in vitro, the number of red globules obtained was significant enough to "eventually lead to a significant improvement in the patient’s clinical condition." The results of this research were published in the Journal of Clinical Investigation. According to the researchers, the first clinical trials could be launched in early 2014.            

Currently, the only curative treatment available for sickle cell disease is the use of implants of stem cells derived from a relative’s bone marrow or from blood from the umbilical cord. 

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