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Total 130 results found since Jan 2013.

Chronic Granulomatous Disease: a Comprehensive Review
AbstractChronic granulomatous disease (CGD) is a primary immunodeficiency of phagocyte function due to defective NADPH oxidase (phox). Compared with the common types ofCYBB/gp91phox,NCF1/p47phox, andCYBA/p22phox deficiency,NCF4/p40phox deficiency is a mild and atypical form of CGD without invasive bacterial or fungal infections. It can be diagnosed using serum-opsonizedE.coli as a stimulus in dihydrorhodamine (DHR) assay. Patients withCYBC1/Eros deficiency, a new and rare form of CGD, present as loss of respiratory burst and gp91phox expression in phagocytes. Neutrophils from patients with CGD are deficient in neutrophil e...
Source: Clinical Reviews in Allergy and Immunology - June 9, 2020 Category: Allergy & Immunology Source Type: research

Gene therapy for X-linked severe combined immunodeficiency: historical outcomes and current status
Source: Journal of Allergy and Clinical Immunology - June 15, 2020 Category: Allergy & Immunology Authors: Sung-Yun Pai, Adrian J. Thrasher Source Type: research

Gene Therapy for Wiskott-Aldrich Syndrome: History, New Vectors, Future Directions
Source: Journal of Allergy and Clinical Immunology - July 1, 2020 Category: Allergy & Immunology Authors: Francesca Ferrua, Francesco Marangoni, Alessandro Aiuti, Maria Grazia Roncarolo Source Type: research

Overview of the current status of gene therapy for primary immune deficiencies (PIDs)
Source: Journal of Allergy and Clinical Immunology - July 31, 2020 Category: Allergy & Immunology Tags: Continuing Medical Education examination Source Type: research

Outcomes of 3 Patients with Adenosine Deaminase Deficiency on Long-Term Enzyme Replacement Therapy (ERT)
Adenosine deaminase (ADA) deficiency is an autosomal recessive severe combined immunodeficiency that typically presents within the first year with severe infections. Other features of ADA deficiency include: autoimmunity, myeloid dysplasia, pulmonary disease, neurodevelopmental delay, skeletal dysplasia, hepatic dysfunction, and lymphoma.1 Hematopoietic stem cell transplant (HSCT) and gene therapy (GT) have been successful in the treatment of ADA deficiency.2,3 Enzyme replacement therapy (ERT) has traditionally been used as a bridge to HSCT or GT and not as first-line long-standing treatment.
Source: Annals of Allergy, Asthma and Immunology - December 21, 2020 Category: Allergy & Immunology Authors: Melissa D. Gans, Larry Bernstein, Jenny Shliozberg, Tatyana Gavrilova, Arye Rubinstein Tags: Letters Source Type: research

Current and Prospective Targets of Pharmacologic Treatment of Hereditary Angioedema Types 1 and 2
In conclusion, an increasing shift towards the use of highly effective long-term prophylaxis is anticipated, which should drastically abate the burden on patients with hereditary angioedema.
Source: Clinical Reviews in Allergy and Immunology - January 9, 2021 Category: Allergy & Immunology Source Type: research

Outcomes of 3 patients with adenosine deaminase deficiency on long-term enzyme replacement therapy
Adenosine deaminase (ADA) deficiency is an autosomal recessive severe combined immunodeficiency that typically presents within the first year with severe infections. Other features of ADA deficiency include the following: autoimmunity, myeloid dysplasia, pulmonary disease, neurodevelopmental delay, skeletal dysplasia, hepatic dysfunction, and lymphoma.1 Hematopoietic stem cell transplant (HSCT) and gene therapy (GT) have been successful in the treatment of ADA deficiency.2,3 Enzyme replacement therapy (ERT) has traditionally been used as a bridge to HSCT or GT and not as first-line, long-standing treatment.
Source: Annals of Allergy, Asthma and Immunology - December 21, 2020 Category: Allergy & Immunology Authors: Melissa D. Gans, Larry Bernstein, Jenny Shliozberg, Tatyana Gavrilova, Arye Rubinstein Tags: Letters Source Type: research

Gene therapy offers potential cure to children born without an immune system
An experimental form of gene therapy developed by a team of researchers from UCLA and Great Ormond Street Hospital in London has successfully treated 48 of 50 children born with a rare and deadly inherited disorder that leaves them without an immune system.Severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID, is caused by mutations in theADA gene that creates the enzyme adenosine deaminase, which is essential to a functioning immune system.For children with the condition, even day-to-day activities like going to school or playing with friends can lead to dangerous, life-threatening infections...
Source: UCLA Newsroom: Health Sciences - May 11, 2021 Category: Universities & Medical Training Source Type: news

What Types of New Therapies are Available for Cystic Fibrosis?
Discussion Cystic fibrosis (CF) was identified in 1938 by Dr. Dorothy Andersen who described 49 patients with pancreatic insufficiency. Since that time significant achievements in the knowledge about the disease and treatments for patients have changed the mortality from a few months to patients living into middle adulthood or even later. Quality of life for patients and their families and friends has also markedly improved. CF is the classic Mendelian autosomal recessive genetic disorder which is a worldwide disorder but affects people of north European ancestry more often where the main mutation is more common. There are...
Source: PediatricEducation.org - June 28, 2021 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

M201 severe combined immunodeficiency with difficulties in genetic testing: implications for therapy
SCID genetic testing directly impacts therapy. For example, gene therapy for Artemis-deficient SCID is available for affected infants lacking a matched sibling donor.
Source: Annals of Allergy, Asthma and Immunology - November 1, 2021 Category: Allergy & Immunology Authors: F. Murshed, F. Young, M. Kan, M. Dorsey, J. Puck Source Type: research

Late Breaking Abstract - Delivery of anti-inflammatory and anti-oxidant synthetic mRNA to a rodent ARDS model by vibrating mesh nebulisation
Acute respiratory distress syndrome (ARDS) has diverse aetiologies, of which pathogen induced pneumonia is amongst the most debilitating. Anti-inflammatory and anti-oxidant therapeutic proteins have previously shown promise in pre-clinical ARDS models (Devaney, J. et al. Crit Care 2013; 17(2): R82). However, many gene therapy approaches have failed due to poor targeting or slow expression of the transgene. A synthetic mRNA vector for extremely rapid production of protein, coupled with direct aerosolised delivery to the injured lung could overcome these limitations. Having previously observed efficacious results in in vitro...
Source: European Respiratory Journal - November 25, 2021 Category: Respiratory Medicine Authors: McCarthy, S., Angel, M., Rohde, C., Masterson, C., Macloughlin, R., Laffey, J., OToole, D. Tags: Allergy and immunology Source Type: research

Future of Therapy for Inborn Errors of Immunity
This article aims to review the future of therapy of PI/IEI (referred to IEI throughout this paper). Historically, immune deficiencies have been characterized as monogenic disorders resulting in immune deficiencies affecting T cells, B cells, combination of T and B cells, or innate immune disorders. More recently, immunologists are also recognizing a variety of phenotypes associated with one genotype or similar phenotypes across genotypes and a role for incomplete penetrance or variable expressivity of some genes causing inborn errors of immunity [3]. The IUIS classification of immune deficiencies (IEIs) has evolved over t...
Source: Clinical Reviews in Allergy and Immunology - January 12, 2022 Category: Allergy & Immunology Source Type: research

The Treatment of Primary Immune Deficiencies: Lessons Learned and Future Opportunities
AbstractPrimary immunodeficiency is a group of disorders associated with susceptibility to infectious agents and the development of various comorbidities. Many primary immunodeficiencies are complicated by immune dysregulation, autoinflammation, or autoimmunity which impacts multiple organ systems. Major advances in the treatment of these disorders have occurred over the last half-century, and deeper molecular understanding of many disorders combined with clinically available genetic testing is allowing for use of precision therapy for several primary immunodeficiencies. Patients with antibody deficiencies who rely on immu...
Source: Clinical Reviews in Allergy and Immunology - July 1, 2022 Category: Allergy & Immunology Source Type: research

News at a glance: New gene therapy, Europe ’s drought, and a black hole’s photon ring
ARCHAEOLOGY Drought exposes ‘Spanish Stonehenge’ for study Scientists are rushing to examine a 7000-year-old stone circle in central Spain that had been drowned by a reservoir for decades and was uncovered after the drought plaguing Europe lowered water levels. Nicknamed the “Spanish Stonehenge”—although 2000 years older than the U.K. stone circle—the Dolmen of Guadalperal (above) was described by archaeologists in the 1920s. The approximately 100 standing stones, up to 1.8 meters tall and arranged around an oval open space, were submerged in the Valdecañas reservoir after the construction of a ...
Source: Science of Aging Knowledge Environment - August 25, 2022 Category: Geriatrics Source Type: research