Future of Care for Patients With Chronic Granulomatous Disease: Gene Therapy and Targeted Molecular Medicine

Overview

Journal J Pediatric Infect Dis Soc

Publisher Oxford University Press

Specialties Infectious Diseases
Pediatrics

Date 2018 May 11

PMID 29746676

Citations 10

Authors

Michael D Keller

Luigi D Notarangelo

Harry L Malech

Affiliations

Soon will be listed here.

Abstract

Chronic granulomatous disease is a rare and potentially fatal disorder of neutrophil function. Beyond current medical management and hematopoietic stem cell transplantation, new methods of gene therapy that use lentiviral vectors or gene editing might extend curative therapies to patients who lack a suitable transplantation donor while eliminating the risk of graft-versus-host disease. Furthermore, new therapies focused on altering the biology of phagolysosomes might offer novel targeted treatments for inflammatory complications in patients with chronic granulomatous disease.

Citing Articles

Clinical presentation, diagnosis, and treatment of chronic granulomatous disease.

Staudacher O, von Bernuth H Front Pediatr. 2024; 12:1384550.

PMID: 39005504 PMC: 11239527. DOI: 10.3389/fped.2024.1384550.

Chronic Granulomatous Disease: an Updated Experience, with Emphasis on Newly Recognized Features.

Oikonomopoulou Z, Shulman S, Mets M, Katz B J Clin Immunol. 2022; 42(7):1411-1419.

PMID: 35696001 PMC: 9674739. DOI: 10.1007/s10875-022-01294-6.

Effects of voriconazole on population pharmacokinetics and optimization of the initial dose of tacrolimus in children with chronic granulomatous disease undergoing hematopoietic stem cell transplantation.

Chen X, Wang D, Lan J, Wang G, Zhu L, Xu X Ann Transl Med. 2021; 9(18):1477.

PMID: 34734029 PMC: 8506700. DOI: 10.21037/atm-21-4124.

Characterization of dental pulp stem cells isolated from a patient diagnosed with Crouzon syndrome.

Torii D, Kobayashi T, Horie T, W Tsutsui T J Cell Physiol. 2021; 236(7):5317-5324.

PMID: 33386632 PMC: 8048801. DOI: 10.1002/jcp.30241.

Geographic Variability and Pathogen-Specific Considerations in the Diagnosis and Management of Chronic Granulomatous Disease.

Prince B, Thielen B, Williams K, Kellner E, Arnold D, Cosme-Blanco W Pediatric Health Med Ther. 2020; 11:257-268.

PMID: 32801991 PMC: 7383027. DOI: 10.2147/PHMT.S254253.

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