Lymphoblastoid and <i>Jurkat</i> cell lines are useful surrogate in developing a CRISPR-Cas9 method to correct leukocyte adhesion deficiency genomic defect.

ABSTRACT: Introduction: Leukocyte adhesion deficiency type 1 (LAD1) is a severe inborn error of immunity caused by mutations in the ITGB2 gene, which encodes the beta-2 integrin subunit (CD18). These mutations lead to the absence or deficiency of CD18/CD11a, b, and c heterodimers, crucial for leukocyte adhesion and immune function. CRISPR-Cas9 Gene editing technology represents a promising approach for correcting these genomic defects restore the stable expression of CD18 and reverse the disease. Methods: We developed a CRISPR-Cas9-based gene correction strategy using Jurkat cells and patient-derived lymphoblastoid cell lines as surrogates for hematopoietic progenitor cells. Three candidate gRNAs were first predicted in silico using CRISPOR and experimentally tested in wild-type

SUBMITTER: Ramadan AR 

PROVIDER: S-EPMC11968696 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature