Evolving Gene Therapy in Primary Immunodeficiency
نویسندگان
چکیده
منابع مشابه
[Gene and cell therapy for primary immunodeficiency diseases].
Primary immunodeficiency diseases (PID) represents a group of inherited diseases where mutations in certain gene lead to certain levels of defects in patient immune systems. Among them, several types of PID, including severe combined immunodeficiecny (SCID), warrented development of new types of curative treatment other than allogeneic hematopoietic stem cell transplantation, eventually culimin...
متن کاملPrevalence of Primary Immunodeficiency Diseases in Kerman, Southeast of Iran
Objective: Primary immunodeficiency diseases (PID) are rare and heterogeneous congenital diseases leading to increased unusual susceptibility to developing infections and causingsome malignancies and autoimmune diseases. This study was conducted to evaluate the epidemiological characteristics of primary these diseases in patients attending the clinic of immunodeficiency diseases in Kerman.Subje...
متن کاملTreating Immunodeficiency through HSC Gene Therapy.
Haematopoietic stem cell (HSC) gene therapy has been successfully employed as a therapeutic option to treat specific inherited immune deficiencies, including severe combined immune deficiencies (SCID) over the past two decades. Initial clinical trials using first-generation gamma-retroviral vectors to transfer corrective DNA demonstrated clinical benefit for patients, but were associated with l...
متن کاملGene Therapy for Primary Immunodeficiencies
Primary immunodeficiencies (PID) are caused by mutations in genes involved in the normal development or activity of the immune system [1, 2]. PIDs include Band T-cell defects, phagocytic disorders, and complement deficiencies with the common feature of frequent lifethreatening infections. The phenotypes vary from asymptomatic (IgA deficiency) to severe PIDs (such as Severe combined immunodefici...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2017
ISSN: 1525-0016
DOI: 10.1016/j.ymthe.2017.03.018