Gene therapy: An updated overview on the promising success stories
- Author:
Roshaidie Abdul Rashid
1
Author Information
1. Human Genome Centre, School of Medical Sciences, Universiti Sains Malaysia, Health Campus, Kubang Kerian, Kelantan, Malaysia
- Collective Name:Ravindran Ankathil
- Publication Type:Review
- Keywords:
gene therapy;
overview;
transgene;
update;
vector
- From:The Malaysian Journal of Pathology
2020;42(2):171-185
- CountryMalaysia
- Language:English
-
Abstract:
Gene therapy is a method of treatment of disease aimed at its molecular level. The progress of gene
therapy, however, was as promising as it was tardy mainly due to the limitations in the resources
and financial part of its development as well as owing to the rarity of most diseases it can offer
its benefits to. The methods of gene therapy can vary depending on factors such as the physiology
of tissue of interest, affinity of vectors to a certain type of cells, depth and accessibility of the
tissue of interest, and size of the gene to be replaced or edited. The concept behind gene therapy
has inspired scientists and clinicians alike leading to a rapid expansion of its clinical utility that
has become so widespread to not only include diseases of monogenic origin, but also polygenic
diseases, albeit not so commonly. This article delves into notable success stories of gene therapy
which has been regarded as the beacon of medical novelty expected to blossom in the near future
to provide a holistic, targeted, precise, and individualistic personalised-medicine as well as laying
out the future hopes of gene therapy in the treatment of debilitating diseases such as solid tumours,
AIDS, Tuberculosis, Diabetes Mellitus, psychiatric illnesses, which are still at a standstill, from a
gene therapy point of view
- Full text:5.2020my01059.pdf
