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Updated: Dec 15, 2025

Development of Stem Cell-derived Antigen-specific Regulatory T Cells Against Autoimmunity
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Precision Therapy for the Treatment of Primary Immunodysregulatory Diseases

Deepak Chellapandian1, Maria Chitty-Lopez2, Jennifer W Leiding2

  • 1Blood 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.

Immunology and Allergy Clinics of North America
|July 14, 2020
PubMed

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.

Keywords:
Biologic modifiersCytotoxic T-lymphocyte antigen 4Hemophagocytic lymphohistiocytosisLipopolysaccharide-responsive beige-like anchorPrecision therapySignal transducer and activator of transcription 1 gain of functionSignal transducer and activator of transcription 3 gain of function

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