Precision Therapy for the Treatment of Primary Immunodysregulatory Diseases.

Biologic modifiers Cytotoxic T-lymphocyte antigen 4 Hemophagocytic lymphohistiocytosis Lipopolysaccharide-responsive beige-like anchor Precision therapy Signal transducer and activator of transcription 1 gain of function Signal transducer and activator of transcription 3 gain of function

Journal

Immunology and allergy clinics of North America
ISSN: 1557-8607
Titre abrégé: Immunol Allergy Clin North Am
Pays: United States
ID NLM: 8805635

Informations de publication

Date de publication:
08 2020
Historique:
entrez: 14 7 2020
pubmed: 14 7 2020
medline: 16 6 2021
Statut: ppublish

Résumé

Precision therapy is a concept in which medical treatment is tailored to the patient's individual needs based on individual characteristics and mechanism of disease. Primary immunodysregulatory disorders are an expanding group of primary immunodeficiency diseases that are characterized by early onset autoimmunity and autoinflammation. Precision therapy allows for the alteration of the aberrant immune response leading to clinical improvement of disease related manifestations. This article reviews targeted precision-based therapy for treatment of cytotoxic T-lymphocyte antigen haploinsufficiency, lipopolysaccharide-responsive beige-like anchor deficiency, activated PI3K deficiency syndrome, signal transducer and activator of transcription- 1 and -3 - gain-of-function disorders, and disorders of inflammasome activation.

Identifiants

pubmed: 32654696
pii: S0889-8561(20)30021-7
doi: 10.1016/j.iac.2020.04.001
pii:
doi:

Substances chimiques

Biomarkers 0

Types de publication

Journal Article Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

511-526

Informations de copyright

Copyright © 2020 Elsevier Inc. All rights reserved.

Déclaration de conflit d'intérêts

Disclosure The authors have nothing to disclose.

Auteurs

Deepak Chellapandian (D)

Blood and Marrow Transplant Program, Johns Hopkins University School of Medicine, Johns Hopkins All Children's Hospital, Research and Education Building, 600 5th Street South, 4th Floor, St Petersburg, FL 33701, USA.

Maria Chitty-Lopez (M)

Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, Children's Research Institute, 601 - 4th Street South, CRI 4008, St Petersburg, FL 33701, USA.

Jennifer W Leiding (JW)

Division of Allergy and Immunology, Department of Pediatrics, University of South Florida, Children's Research Institute, 601 - 4th Street South, CRI 4008, St Petersburg, FL 33701, USA. Electronic address: jleiding@usf.edu.

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Classifications MeSH