Gene therapy leaves a vicious cycle

Reena Goswami, Gayatri Subramanian, Liliya Silayeva, Isabelle Newkirk, Deborah Doctor, Karan Chawla, Saurabh Chattopadhyay, Dhyan Chandra, Nageswararao Chilukuri, Venkaiah Betapudi

Research output: Contribution to journalReview articlepeer-review

235 Scopus citations

Abstract

The human genetic code encrypted in thousands of genes holds the secret for synthesis of proteins that drive all biological processes necessary for normal life and death. Though the genetic ciphering remains unchanged through generations, some genes get disrupted, deleted and or mutated, manifesting diseases, and or disorders. Current treatment options-chemotherapy, protein therapy, radiotherapy, and surgery available for no more than 500 diseases-neither cure nor prevent genetic errors but often cause many side effects. However, gene therapy, colloquially called “living drug,” provides a one-time treatment option by rewriting or fixing errors in the natural genetic ciphering. Since gene therapy is predominantly a viral vector-based medicine, it has met with a fair bit of skepticism from both the science fraternity and patients. Now, thanks to advancements in gene editing and recombinant viral vector development, the interest of clinicians and pharmaceutical industries has been rekindled. With the advent of more than 12 different gene therapy drugs for curing cancer, blindness, immune, and neuronal disorders, this emerging experimental medicine has yet again come in the limelight. The present review article delves into the popular viral vectors used in gene therapy, advances, challenges, and perspectives.

Original languageEnglish
Article number297
JournalFrontiers in Oncology
Volume9
Issue numberAPR
DOIs
StatePublished - 2019

Bibliographical note

Publisher Copyright:
Copyright © 2019 Goswami, Subramanian, Silayeva, Newkirk, Doctor, Chawla, Chattopadhyay, Chandra, Chilukuri and Betapudi. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

Keywords

  • Clinical trials
  • Diseases and disorders
  • Gene therapy
  • Modern medicines
  • Viral vectors

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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