Trusted Resources: Evidence & Education
Scientific literature and patient education texts
Gene Therapy in a Patient with Sickle Cell Disease
source: The New England Journal of Medicine
year: 2017
authors: Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O1, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M
summary/abstract:Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this disorder is complicated by the complex cellular abnormalities and challenges in achieving effective, persistent inhibition of polymerization of hemoglobin S. We describe our first patient treated with lentiviral vector-mediated addition of an antisickling β-globin gene into autologous hematopoietic stem cells. Adverse events were consistent with busulfan conditioning. Fifteen months after treatment, the level of therapeutic antisickling β-globin remained high (approximately 50% of β-like-globin chains) without recurrence of sickle crises and with correction of the biologic hallmarks of the disease.
DOI: 10.1056/NEJMoa1609677read more full text
Related Content
-
Sickle Cell Disease – Children’s Hospital of PhiladelphiaSickle cell disease is the name of a gro...
-
Association Between Vaso-Occlusive Crises and Opioid Prescriptions Among Patients With Sickle Cell Disease: A Retros...Background/Objectives: Among sickle cel...
-
Virtual Reality Helps Reduce Pain Among Patients With Sickle Cell DiseaseImmersive virtual reality appeared effec...
-
Adulting is especially difficult for youth with special health care needsThere are approximately 4.3 million adol...
-
Amy E. Sobota, MD, MPHAmy E. Sobota is a pediatric hematologis...
-
SCDAA 2023 National Abstract CompetitionIf you are a researcher, community-based...
-
The Sickle Cell Infusion Center at Johns HopkinsThe Sickle Cell Center for Adults is ded...
To improve your experience on this site, we use cookies. This includes cookies essential for the basic functioning of our website, cookies for analytics purposes, and cookies enabling us to personalize site content. By clicking on 'Accept' or any content on this site, you agree that cookies can be placed. You may adjust your browser's cookie settings to suit your preferences. More Information
The cookie settings on this website are set to "allow cookies" to give you the best browsing experience possible. If you continue to use this website without changing your cookie settings or you click "Accept" below then you are consenting to this.