Treating primary immunodeficiencies with defects in NK cells: from stem cell therapy to gene editing.

ABSTRACT: Primary immunodeficiency diseases (PIDs) are rare diseases that are characterized by genetic mutations that damage immunological function, defense, or both. Some of these rare diseases are caused by aberrations in the normal development of natural killer cells (NKs) or affect their lytic synapse. The pathogenesis of these types of diseases as well as the processes underlying target recognition by human NK cells is not well understood. Utilizing induced pluripotent stem cells (iPSCs) will aid in the study of human disorders, especially in the PIDs with defects in NK cells for PID disease modeling. This, together with genome editing technology, makes it possible for us to facilitate the discovery of future therapeutics and/or cell therapy treatments for these patients, because, to date, the

SUBMITTER: Eguizabal C 

PROVIDER: S-EPMC7590703 | biostudies-literature | 2020 Oct

REPOSITORIES: biostudies-literature