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.
Highlights
The human genome contains ∼25,000 genes that encode a wide variety of proteins colloquially called the building blocks and workhorses of the cell to drive every biological process necessary for life and death [1,2,3,4]
According to the Genetic and Rare Diseases Information Center (GARD) and Global Genes R, the leading rare disease patient advocacy organization in the world, dysfunctional genes account for 80% of the total 7,136 diseases reported to date
While the idea of gene therapy has been around for the past 80 years, Professor William Szybalski’s demonstration in 1962 on correcting a genetic defect by delivering foreign DNA into mammalian cells is regarded as its birth [33]
Summary
Reena Goswami 1, Gayatri Subramanian 2, Liliya Silayeva 1, Isabelle Newkirk 1, Deborah Doctor 1, Karan Chawla 2, Saurabh Chattopadhyay 2, Dhyan Chandra 3, Nageswararao Chilukuri 1 and Venkaiah Betapudi 1,4*. 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. 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. 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. Specialty section: This article was submitted to Cancer Molecular Targets and Therapeutics, a section of the journal
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