Gene therapy and genome editing for primary immunodeficiency diseases
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...
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Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the...
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Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by loss of function of the dystrophin gene on the X chromosome. Gene augmentation of dystrophin is challenging due to the large size of the dystrophin cDNA. Emerging genome editing technologies, such as TALEN and CRISPR-Cas9 systems, open a new erain the restoration of functional dystrophin and are a hallmark of bona fide gen...
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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...
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Introduction: Primary immunodeficiencies (PID) are rare heterogeneous disorders with defects in which one or more components of the immune system are malfunctioning. Clinical presentations of the patients according to type of immunodeficiency are variable. The majority of these patients are susceptible to infections depending on the type of disorder. In these patients, one of the most important...
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ژورنال
عنوان ژورنال: Genes & Diseases
سال: 2020
ISSN: 2352-3042
DOI: 10.1016/j.gendis.2019.07.007