Targeted Therapy with Biologicals and Small Molecules in Primary Immunodeficiencies.
Med Princ Pract
; 29(2): 101-112, 2020.
Article
en En
| MEDLINE
| ID: mdl-31597133
ABSTRACT
Primary immunodeficiencies are disorders resulting from mutations in genes involved in immune defense and immune regulation. These conditions are characterized by various combinations of recurrent infections, autoimmunity, lymphoproliferation, inflammatory manifestations, and malignancy. In the last 20 years, newborn screening programs and next generation sequencing techniques have increased the ability to diagnose primary immunodeficiencies. Furthermore, an advanced understanding of the molecular basis of these inherited disorders has led to the implementation of targeted therapies that utilize small molecules and biologics to modulate the activity of impaired intracellular pathways. This article will discuss selected primary immunodeficiencies, the genetic defects of which have been recently studied and are amenable to targeted therapy as a reflection of the potential of precision medicine in the future.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Productos Biológicos
/
Enfermedades de Inmunodeficiencia Primaria
Límite:
Humans
Idioma:
En
Revista:
Med Princ Pract
Asunto de la revista:
EDUCACAO
Año:
2020
Tipo del documento:
Article
País de afiliación:
Estados Unidos