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Gene therapy and type 1 diabetes mellitus

Chellappan DK 1 , Sivam NS 2 , Teoh KX 2 , Leong WP 2 , Fui TZ 2 , Chooi K 2 Show all authors , Khoo N 2 , Yi FJ 2 , Chellian J 3 , Cheng LL 3 , Dahiya R 4 , Gupta G 5 , Singhvi G 6 , Nammi S 7 , Hansbro PM 8 , Dua K 9

Affiliations 

  • 1 Department of Life Sciences, International Medical University, Kuala Lumpur, 57000, Malaysia. Electronic address: dinesh_kumar@imu.edu.my
  • 2 School of Pharmacy, International Medical University, Kuala Lumpur, 57000, Malaysia
  • 3 Department of Life Sciences, International Medical University, Kuala Lumpur, 57000, Malaysia
  • 4 Laboratory of Peptide Research and Development, School of Pharmacy, Faculty of Medical Sciences, The University of the West Indies, St. Augustine, Trinidad and Tobago
  • 5 School of Pharmaceutical Sciences, Jaipur National University, Jagatpura, 302017, Jaipur, India. Electronic address: gauravpharma25@gmail.com
  • 6 Department of Pharmacy, Birla Institute of Technology & Science (BITS), Pilani, Pilani Campus, 333031, Rajasthan, India
  • 7 School of Science and Health, Western Sydney University, NSW, 2751, Australia; NICM Health Research Institute, Western Sydney University, NSW, 2751, Australia
  • 8 Discipline of Pharmacy, Graduate School of Health, University of Technology Sydney, Ultimo NSW, 2007, Australia; School of Biomedical Sciences and Pharmacy, The University of Newcastle, Callaghan, NSW 2308, Australia & Priority Research Centre for Healthy Lungs, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia
  • 9 Discipline of Pharmacy, Graduate School of Health, University of Technology Sydney, Ultimo NSW, 2007, Australia; School of Biomedical Sciences and Pharmacy, The University of Newcastle, Callaghan, NSW 2308, Australia & Priority Research Centre for Healthy Lungs, Hunter Medical Research Institute, Newcastle, NSW, 2305, Australia; School of Pharmaceutical Sciences, Shoolini University, Solan, Himachal Pradesh, 173229, India
Biomed Pharmacother, 2018 Dec;108:1188-1200.
PMID: 30372820 DOI: 10.1016/j.biopha.2018.09.138

Abstract

BACKGROUND: Type 1 diabetes mellitus (T1DM) is an autoimmune disorder characterized by T cell-mediated self-destruction of insulin-secreting islet β cells. Management of T1DM is challenging and complicated especially with conventional medications. Gene therapy has emerged as one of the potential therapeutic alternatives to treat T1DM. This review primarily focuses on the current status and the future perspectives of gene therapy in the management of T1DM. A vast number of the studies which are reported on gene therapy for the management of T1DM are done in animal models and in preclinical studies. In addition, the safety of such therapies is yet to be established in humans. Currently, there are several gene level interventions that are being investigated, notably, overexpression of genes and proteins needed against T1DM, transplantation of cells that express the genes against T1DM, stem-cells mediated gene therapy, genetic vaccination, immunological precursor cell-mediated gene therapy and vectors.

METHODS: We searched the current literature through searchable online databases, journals and other library sources using relevant keywords and search parameters. Only relevant publications in English, between the years 2000 and 2018, with evidences and proper citations, were considered. The publications were then analyzed and segregated into several subtopics based on common words and content. A total of 126 studies were found suitable for this review.

FINDINGS: Generally, the pros and cons of each of the gene-based therapies have been discussed based on the results collected from the literature. However, there are certain interventions that require further detailed studies to ensure their effectiveness. We have also highlighted the future direction and perspectives in gene therapy, which, researchers could benefit from.

* Title and MeSH Headings from MEDLINE®/PubMed®, a database of the U.S. National Library of Medicine.

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