Autologous Ex Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Favorable Safety Profile

Journal of Human Immunity 2026 Open access

Read full text
Authors: Claire Booth, Julián Sevilla, Elena Almarza, Caroline Y. Kuo, Josune Zubicaray, Maria Chitty-Lopez, Susan Manganaro, Haojun Ouyang, Bitha Narayanan, Janel Long‐Boyle, Augustine O. Fernandes, Kritika Chetty
+13 more Satiro DeOliveira, Crystal Banuelos, Jinhua Xu‐Bayford, Phillip John-Neek, Connie Jackson, T Moore, Kimberly Gilmour, Axel Schambach, Michael Rothe, Adrian James Thrasher, Juan Antonio Bueren, Jonathan D. Schwartz, Donald B. Kohn
DOI: 10.70962/cis2026abstract.4
Cited by: 1

Abstract

Background Severe leukocyte adhesion deficiency-I (LAD-I) results from biallelic deleterious ITGB2 variants leading to deficient/defective CD18 leukocyte expression and impaired endothelial adhesion and extravasation. Children with

Fields of Study & Subjects

Hematopoietic Stem Cell Transplantation Microfluidic and Bio-sensing Technologies Cell Adhesion Molecules Research

Publication details

Journal / source
Journal of Human Immunity
Publisher
Rockefeller University Press
Volume
2
Issue
CIS2026
Publication date
1 May 2026
Language
EN