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Gene Therapy for Severe Combined Immunodeficiency [PDF]

open access: possibleAnnual Review of Medicine, 2005
Studies of severe combined immunodeficiency (SCID), a group of rare monogenic disorders, have provided key findings about the physiology of immune system development. The common characteristic of these diseases is the occurrence of a block in T cell differentiation, always associated with a direct or indirect impairment of B cell immunity.
Marina Cavazzana-Calvo   +3 more
openaire   +2 more sources

Gene therapy of severe combined immunodeficiencies

The Journal of Gene Medicine, 2001
Recent advances in gene transfer in human hematopoietic cells, combined with a better understanding of the genetic aspects of several immunodeficiencies, has offered new opportunities in the domain of gene therapy. Severe combined immunodeficiency (SCID) appear to represent a good model for the application of gene therapy, combining an expected ...
Le Deist F   +4 more
openaire   +7 more sources

Severe Combined Immunodeficiency Disorders

Immunology and Allergy Clinics of North America, 2015
Severe combined immunodeficiency disorders represent pediatric emergencies due to absence of adaptive immune responses to infections. The conditions result from either intrinsic defects in T-cell development (ie, severe combined immunodeficiency disease [SCID]) or congenital athymia (eg, complete DiGeorge anomaly).
William T. Shearer, Ivan K. Chinn
openaire   +3 more sources

Severe Combined Immunodeficiency Diseases

Springer Seminars in Immunopathology, 1978
The most severe, and also a relatively common immunodeficiency syndrome involves both antibody- and cell-mediated immune mechanisms. In more modern terms the ‘severe combined immunodeficiency’ syndromes (SCID) are defined as all diseases resulting from marked and longlasting functional impairment of both the T- and B-cell system.
W. H. Hitzig, L. J. Dooren, J. M. Vossen
openaire   +2 more sources

Newborn Screening for Severe Combined Immunodeficiency

Pediatric Clinics of North America, 2019
The T-cell receptor excision circle (TREC) assay is an effective screening tool for severe combined immunodeficiency (SCID). The TREC assay was designed to detect typical SCID and leaky SCID, but any condition causing low naïve T-cell counts will also be detected.
Tayaba Miah   +2 more
openaire   +4 more sources

Severe Combined Immunodeficiencies in Humans [PDF]

open access: possible, 1993
Severe combined immunodeficiencies (SCID) form a heterogeneous group of genetic disorders characterized by intrinsic defects in T cell differentiation and function causing profound abnormalities of cellular and humoral immunity (Table 1) (Arnaiz-Villena 1992b, Conley 1992, Fischer 1991, De Saint Basile 1991b).
B. Lisowska-Grospierre   +10 more
openaire   +1 more source

Gene Therapy for Severe Combined Immunodeficiencies [PDF]

open access: possible, 1999
Severe combined immunodeficiency diseases (SCIDs) are a group of primary immunodeficiencies characterized by profoundly impaired cell-mediated and humoral responses [1, 2]. Affected children typically fail to thrive and become ill with recurrent infections caused by bacteria, viruses, and opportunistic pathogens.
AIUTI , ALESSANDRO, BORDIGNON , CLAUDIO
openaire   +2 more sources

Gene therapy for severe combined immunodeficiencies

Expert Opinion on Biological Therapy, 2005
Severe combined immune deficiencies (SCIDs) are a group of monogenic diseases resulting in profound disturbances of lymphocyte development and function. Affected individuals are prone to life-threatening infections and without treatment do not survive beyond the first year of life.
Adrian J. Thrasher, H. Bobby Gaspar
openaire   +2 more sources

Gastroesophageal reflux and severe combined immunodeficiency☆, ☆☆, ★, ★★

Journal of Allergy and Clinical Immunology, 1997
Gastrointestinal and respiratory symptoms and failure to thrive not associated with infections or medications were noted in patients with severe combined immunodeficiency.The aim of our study was to determine the frequency of gastroesophageal reflux in patients with severe combined immunodeficiency.We studied the case histories of 73 pediatric patients
Andreas Boeck   +2 more
openaire   +3 more sources

Newborn Screening for Severe Combined Immunodeficiency

Current Allergy and Asthma Reports, 2018
This review provides a brief history of newborn screening (NBS) for severe combined immunodeficiency (SCID), discusses the theoretical basis for the T cell receptor excision circle (TREC) assay, highlights the results of recent studies using the TREC, and provides practical advice for the evaluation of infants with an abnormal TREC assay.Currently, all
John M. Routes, James W. Verbsky
openaire   +3 more sources

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