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Gene therapy

Saini, D (2020) Gene therapy. [Book Chapter]

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Official URL: https://doi.org/10.1016/B978-0-12-820595-2.00011-4


Genes, composed of DNA, are templates for protein production. The altering gene causes changes in proteins and leads to diseases. A viable method to treat these genetic diseases is gene therapy, involving vectors to transfer healthy genes into the patient. Viral vectors are transferred into host cells, where they alter the nuclear DNA. Somatic gene therapy involving the transfer of genes into somatic cells can also be done. Germline therapy, transfer of genes into germ cells, ensures that all alterations are heritable. In Gene Augmentation Technique, when cells contain nonfunctional DNA, functional DNA is inserted. In Gene Inhibition Therapy, a gene is inserted, which suppresses or overrides the faulty gene. Hurdles for Gene therapy to be effective: Immune responses, multigenetic disorders, cost, short-lived natures, and side effects. There are ethical complications as to what is normal versus undesirable traits.

Item Type: Book Chapter
Publication: Advances in Animal Genomics
Publisher: Elsevier
Additional Information: The copyright for this article belongs to the Authors.
Keywords: CRISPR Gene editingGene therapySomatic and germline therapyViral and nonviral vectors
Department/Centre: Division of Biological Sciences > Biochemistry
Date Deposited: 21 Feb 2023 05:27
Last Modified: 21 Feb 2023 05:27
URI: https://eprints.iisc.ac.in/id/eprint/79959

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