Google
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
+myBinderRelated Content
-
Management of infections in SCD patients treated with HU in the framework of Escort HUBackground and method: This study aims t...
-
New York State Sickle Cell Advocacy Network (NYSSCAN)New York State Sickle Cell Advocacy Netw...
-
The first South American case of pre-implantation genetic diagnosis to select compatible embryo for cord blood trans...Sickle cell anemia is an inherited syste...
-
Sickle Cell Treatment ‘Life-Changing’ for Brockton BrothersEmmanuel “Manny” Johnson, 21, of Bro...
-
Sickle Cell Pulmonary Clinic, Connecticut Children’s Medical CenterConnecticut Children’s is one of the f...
-
Amy E. Sobota, MD, MPHAmy E. Sobota is a pediatric hematologis...
-
Sophie Lanzkron, MD, MHSDr. Sophie Lanzkron is an associate prof...
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.
Powered by
Support for this site is provided by
This platform is made possible through a partnership with the Sickle Cell Disease Association of America, Inc. (SCDAA) and its member organizations. SCDAA's mission is to advocate for people affected by sickle cell conditions and empower community-based organizations to maximize quality of life and raise public consciousness while advancing the search for a universal cure.