Clinical Reviews in Allergy & Immunology

, Volume 49, Issue 2, pp 163–176

Gene Therapy for Autoimmune Disease

  • Shang-An Shu
  • Jinjun Wang
  • Mi-Hua Tao
  • Patrick S. C. Leung
Article

DOI: 10.1007/s12016-014-8451-x

Cite this article as:
Shu, SA., Wang, J., Tao, MH. et al. Clinic Rev Allerg Immunol (2015) 49: 163. doi:10.1007/s12016-014-8451-x

Abstract

Advances in understanding the immunological and molecular basis of autoimmune diseases have made gene therapy a promising approach to treat the affected patients. Gene therapy for autoimmune diseases aims to regulate the levels of proinflammatory cytokines or molecules and the infiltration of lymphocytes to the effected sites through successful delivery and expression of therapeutic genes in appropriate cells. The ultimate goal of gene therapy is to restore and maintain the immune tolerance to the relevant autoantigens and improve clinical outcomes for patients. Here, we summarize the recent progress in identifying genes responsible for autoimmune diseases and present examples where gene therapy has been applied as treatments or prevention in autoimmune diseases both in animal models and the clinical trials. Discussion on the advantages and pitfalls of gene therapy strategies employed is provided. The intent of this review is to inspire further studies toward the development of new strategies for successful treatment of autoimmune diseases.

Keywords

Gene therapyAutoimmune diseaseAnimal modelClinical trialDelivery vectors

Abbreviations

AAV

Adenoassociated virus

APL

Altered peptide ligand

APN

Adiponectin

BAFF

B cell-activating factor belonging to the TNF family

BDNF

Brain-derived neurotrophic factor

BMSCs

Bone marrow stem cells

CIA

Collagen induced arthritis

cPLA2α

Cytosolic phospholipase A2

CNS

Central nervous system

DRP

DNase-resistant particles

EAE

Experimental autoimmune encephalomyelitis

EDC

1-Ethyl-3-(3-dimethylaminopropyl)-carbodiimide

GA

Glatiramer acetate

MOG

Myelin oligodendrocyte glycoprotein

MBP

Myelin basic protein

MS

Multiple sclerosis

PBMC

Peripheral blood monocyte

PLGA

Poly(lactic-co-glycolic acid)

PLP

Proteolipid protein

RA

Rheumatoid arthritis

shRNA

Small hairpin RNA

siRNA

Small interfering RNA

TLR

Toll-like receptor

Tregs

Regulatory T cells

VEGF

Vascular endothelial growth factor

Copyright information

© Springer Science+Business Media New York 2014

Authors and Affiliations

  • Shang-An Shu
    • 1
  • Jinjun Wang
    • 1
  • Mi-Hua Tao
    • 2
  • Patrick S. C. Leung
    • 1
  1. 1.Division of Rheumatology/Allergy and Clinical Immunology, School of MedicineUniversity of CaliforniaDavisUSA
  2. 2.Institute of Biomedical SciencesAcademia SinicaTaipeiTaiwan