scholarly journals Haematopoietic stem cell transplantation for severe sickle cell disease in childhood: a single centre experience of 50 patients

2014 ◽  
Vol 165 (3) ◽  
pp. 402-408 ◽  
Author(s):  
Laurence Dedeken ◽  
Phu Q. Lê ◽  
Nadira Azzi ◽  
Cécile Brachet ◽  
Catherine Heijmans ◽  
...  
Keyword(s):  
Stem Cell ◽  
Sickle Cell Disease ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
Haematopoietic Stem Cell Transplantation ◽  
Haematopoietic Stem Cell ◽  
Cell Disease ◽  
Single Centre ◽  
Single Centre Experience

scholarly journals In utero Therapy for the Treatment of Sickle Cell Disease: Taking Advantage of the Fetal Immune System

2021 ◽  
Vol 8 ◽  
Author(s):  
Alba Saenz de Villaverde Cortabarria ◽  
Laura Makhoul ◽  
John Strouboulis ◽  
Giovanna Lombardi ◽  
Eugene Oteng-Ntim ◽  
...  
Keyword(s):  
Stem Cell ◽  
Immune System ◽  
Sickle Cell Disease ◽  
Stem Cell Transplantation ◽  
Cell Transplantation ◽  
Sickle Cell ◽  
In Utero ◽  
Haematopoietic Stem Cell ◽  
Cell Disease ◽  
Fetal Immune System

Sickle Cell Disease (SCD) is an autosomal recessive disorder resulting from a β-globin gene missense mutation and is among the most prevalent severe monogenic disorders worldwide. Haematopoietic stem cell transplantation remains the only curative option for the disease, as most management options focus solely on symptom control. Progress in prenatal diagnosis and fetal therapeutic intervention raises the possibility of in utero treatment. SCD can be diagnosed prenatally in high-risk patients using chorionic villus sampling. Among the possible prenatal treatments, in utero stem cell transplantation (IUSCT) shows the most promise. IUSCT is a non-myeloablative, non-immunosuppressive alternative conferring various unique advantages and may also offer safer postnatal management. Fetal immunologic immaturity could allow engraftment of allogeneic cells before fetal immune system maturation, donor-specific tolerance and lifelong chimerism. In this review, we will discuss SCD, screening and current treatments. We will present the therapeutic rationale for IUSCT, examine the early experimental work and initial human experience, as well as consider primary barriers of clinically implementing IUSCT and the promising approaches to address them.

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