Gene
Therapy: New Therapeutic approach to Diabetes Mellitus
Shilpa
S. Borkar1*,
Seema B. Wakodkar1, Pradeep S. Raghatate2,
Debarshi Kar Mahapatra3
1Kamla
Nehru College of Pharmacy, Butibori, Nagpur-441108, Maharashtra,
India
2Department
of Pharmaceutical Chemistry Kamla Nehru College of Pharmacy,
Butibori, Nagpur-441108, Maharashtra, India
3Department
of Pharmaceutical Chemistry, Dadasaheb Balpande College of Pharmacy,
Nagpur-440037, Maharashtra, India
Correspondence:
Email- shilpa_borkar23@rediffmail.com
Abstract
In
the past decade there has been a great deal of enthusiasm and high
expectations for cell transplantation and genetic engineering. 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. One of the potential therapeutic
alternatives to treat T1DM is emerged with Gene therapy. This review
primarily focuses on the current status and the future perspectives
of gene therapy in the management of T1DM. A number of the studies
which are reported on gene therapy for the management of T1DM are
performed 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 and options
that are being investigated, notably, would be the 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.
Keywords:
Autoimmune disease, Gene therapy, Insulin, Type 1 diabetes mellitus,
Proteins