Gene Therapies for Primary Immune Deficiencies
نویسندگان
چکیده
منابع مشابه
Ten years of gene therapy for primary immune deficiencies.
Gene therapy with hematopoietic stem cells (HSC) is an attractive therapeutic strategy for several forms of primary immunodeficiencies. Current approaches are based on ex vivo gene transfer of the therapeutic gene into autologous HSC by vector-mediated gene transfer. In the past decade, substantial progress has been achieved in the treatment of severe combined immundeficiencies (SCID)-X1, adeno...
متن کاملGene therapy for primary immune deficiencies: a Canadian perspective
The use of gene therapy (GT) for the treatment of primary immune deficiencies (PID) including severe combined immune deficiency (SCID) has progressed significantly in the recent years. In particular, long-term studies have shown that adenosine deaminase (ADA) gene delivery into ADA-deficient hematopoietic stem cells that are then transplanted into the patients corrects the abnormal function of ...
متن کاملPrimary Immune Deficiencies – Principles of Care
Primary immune deficiencies (PIDs) are a growing group of over 230 different disorders caused by ineffective, absent or an increasing number of gain of function mutations in immune components, mainly cells and proteins. Once recognized, these rare disorders are treatable and in some cases curable. Otherwise untreated PIDs are often chronic, serious, or even fatal. The diagnosis of PIDs can be d...
متن کاملAdvances in neonatal screening for primary immune deficiencies
The congenital disorders of immune competence are known as primary immunodeficiencies (PID) and are mainly characterized by a pathological susceptibility to infection. These infections are mostly of time repetitive and drug resistant in nature. The number of infected infants has reached over 200 and is on the increase. Additionally, clinical severity of the disease has been confirmed to be exte...
متن کاملCord blood stem cell transplantation in primary immune deficiencies.
PURPOSE OF REVIEW Umbilical cord haematopoietic stem cell transplantation for primary immunodeficiencies is examined with other developments in treatment. Cord blood biology is reviewed, and advantages and disadvantages of umbilical cord blood stem cell transplantation for primary immunodeficiencies discussed. Clinical outcome data and future developments are reviewed. RECENT FINDINGS Cord bl...
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ژورنال
عنوان ژورنال: Frontiers in Immunology
سال: 2021
ISSN: 1664-3224
DOI: 10.3389/fimmu.2021.648951