Recent Advances in Gene Therapy and Modeling of Chronic Granulomatous Disease.

Recent Advances in Gene Therapy and Modeling of Chronic Granulomatous Disease. Iran J Allergy Asthma Immunol. 2019 Apr 01;18(2):131-142 Authors: Jafarian A, Shokri G, Shokrollahi Barough M, Moin M, Pourpak Z, Soleimani M Abstract The Chronic granulomatous disease (CGD) is a primary immunodeficiency that characterized by mutations in phagocyte nicotinamide adenine dinucleotide phosphate (NADPH) oxidase, resulting in deficient antimicrobial activity of phagocytic cells and recurrent childhood infections. Hematopoietic stem cell transplantation (HSCT) is a curative option for patients with human leukocyte antigen (HLA) matched donor, when conventional cares and therapies fail. However, in many cases when the patients have not an HLA-matched donor, they need to a method to recapitulate the function of the affected gene within the patient's own cells. Gene therapy is a promising approach for CGD. While, the success of retroviral or lentiviral vectors in gene therapy for CGD has been hampered by random integration and insertional activation of proto-oncogenes. These serious adverse events led to improvement and generations of viral vectors with increased safety characteristics. Gene therapy continues to progress and the advent of new technologies, such as engineered endonucleases that have shown a great promise for the treatment of genetic disease. This review focuses on the application of gene therapy for the CGD, the limitations encountered in current clinic...
Source: Iranian Journal of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: Iran J Allergy Asthma Immunol Source Type: research

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A significant proportion of primary immunodeficiency (PID) diseases include humoral deficiency. Humoral deficiencies range in severity from complete deficiencies observed in diseases such as X-linked or autosomal recessive agammaglobulinemias and certain severe combined immune deficiencies (SCID), to milder specific antibody deficiencies. According to a recent update from the Jeffrey Modell Centers Network, disorders classified as primarily humoral deficiencies accounted for 45% of survey center PID patients worldwide, not including the many additional patients with combined immune deficiencies who also suffer from antibody deficiency.
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: CME Reviews Source Type: research
Severe Combined Immune Deficiency (SCID) is the first primary immunodeficiency amenable to population based newborn screening (NBS) and is now available in all 50 states and Puerto Rico. NBS for SCID uses real time qPCR to measure the number of copies of T cell restriction excision circles (TREC) in DNA from newborn dried blood spots (DBS) and reliably detects severe T cell lymphopenia (TCL) at birth. SCID is the primary condition detected using this assay. However, it is well known that many secondary conditions, having varying degrees of TCL, are identified through SCID NBS.
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: Perspective Source Type: research
As implied by its name, common variable immunodeficiency (CVID) is the most prevalent symptomatic primary immunodeficiency in adults and is a diagnosis frequently considered by the practicing allergist and clinical immunologist. Despite familiarity, there is disagreement about which immune deficient patients should be given the diagnosis of CVID. As the name also implies, patients with CVID often exhibit protean clinical manifestations1, 2. Reflecting this, the International Union of Immunological Societies (IUIS) Classification of Primary Immunodeficiency refers to CVID as a group of disorders with several clinical and la...
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: Review Source Type: research
CONCLUSION: Mechanisms producing these conditions are poorly understood but include cytokine and cellular inflammatory pathways, and loss of tolerance to self-antigens through the multiple signaling molecules and pathways common to tolerance and immune deficiency. PMID: 31349011 [PubMed - as supplied by publisher]
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: Ann Allergy Asthma Immunol Source Type: research
Common variable immunodeficiency (CVID) is a primary immunodeficiency that is clinically heterogeneous, characterized by both infectious and non-infectious complications. While the hallmark of disease presentation is commonly a history of recurrent sinopulmonary infections, autoimmunity and non-infectious inflammatory conditions are increasing associated with CVID.
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: Review Source Type: research
Abstract BACKGROUND: The subcutaneous immune globulin (SCIG) 20% product, Ig20Gly, was shown to be efficacious and well tolerated in two phase 2/3 North American and European studies at infusion volumes up to 60 mL/site and rates up to 60 mL/h/site in patients with primary immunodeficiency diseases. OBJECTIVE: To assess patient experience after switching to Ig20Gly with fast infusion rates and large infusion volumes/site in the North American study. METHODS: In this analysis of the open-label phase 2/3 study in which patients aged ≥2 years received weekly Ig20Gly infusions for up to approximately 1.3 ...
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Tags: Ann Allergy Asthma Immunol Source Type: research
The subcutaneous immune globulin (SCIG) 20% product, Ig20Gly, was shown to be efficacious and well tolerated in two phase 2/3 North American and European studies at infusion volumes up to 60 mL/site and rates up to 60 mL/h/site in patients with primary immunodeficiency diseases.
Source: Annals of Allergy, Asthma and Immunology - Category: Allergy & Immunology Authors: Source Type: research
DiGeorge syndrome (DGS) is a primary immunodeficiency characterized by various degrees of T-cell deficiency. In partial DGS (pDGS), other risk factors could predispose to recurrent infections, autoimmunity, and allergy. The aim of this study was to assess the effect of different factors in the development of infections, autoimmunity, and/or allergy in patients with pDGS. We studied 467 pDGS patients in follow-up at Great Ormond Street Hospital. Using a multivariate approach, we observed that palatal anomalies represent a risk factor for the development of recurrent otitis media with effusion. Gastroesophageal reflux/dyspha...
Source: Blood - Category: Hematology Authors: Tags: Pediatric Hematology, Immunobiology and Immunotherapy Source Type: research
We present a case of treatment-refractory, systemic SS as the initial manifestation in a young child with common variable immunodeficiency (CVID). We also review current literature about SS and concurrent immunodeficiencies and autoimmunity in CVID patients.Recent FindingsFew case reports exist regarding the co-occurrence of Sweet ’s syndrome and primary immunodeficiencies. SS is characterized by a pro-inflammatory state with a neutrophil predominance resulting in a spectrum of clinical manifestations. CVID is a multifactorial antibody deficiency that can be associated with autoimmunity, which some studies have propo...
Source: Current Allergy and Asthma Reports - Category: Allergy & Immunology Source Type: research
Ricardo U. Sorensen1,2,3* 1Professor Emeritus of Pediatrics, Department of Pediatrics, Louisiana State University Health Science Center, New Orleans, LA, United States 2Louisiana Primary Immunodeficiency Network, New Orleans, LA, United States 3Honorary Professor, Faculty of Medicine, University of La Frontera, Temuco, Chile In thisopinion manuscript the author postulates that the present definition of Specific Antibody Deficiency (SAD) needs to be revised and expanded. It is presently defined as a syndrome of low IgG antibody responses to purified Streptococcus pneumoniae capsular polysaccharides vaccine...
Source: Frontiers in Immunology - Category: Allergy & Immunology Source Type: research
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