Abstract
Gene therapy technique alters cellular gene expression for therapeutic purposes through diverse methods. Different vectors are used for the transfer of gene into either somatic or germline human cells. Viral vectors are used commonly for this transfer. Adeno-, Herpes-, and Lente-viruses are the most common experimented vectors. Genes are transferred into the cells by in vivo or ex vivo mode of administrations. Non-viral vectors (particle- or chemical-based DNA transfer) are also employed for the same purpose. Gene therapy has various applications in the treatment of malignancy, cardiovascular diseases, SCID, Alzheimer’s disease, and many others. Though there are a number of limitations for this mode of treatment, several gene therapy products are being approved recently by regulatory authorities. Of late, the approved drugs are talimogene laherparepvec, voretigene neparvovec, gendicine, sipuleucel-T, tisagenlecleucel, and axicabtagene ciloleucel. Several other drugs are in clinical trials and awaiting regulatory approval.
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Yadav, A.C., MG, G. (2019). Gene Therapy. In: Raj, G., Raveendran, R. (eds) Introduction to Basics of Pharmacology and Toxicology. Springer, Singapore. https://doi.org/10.1007/978-981-32-9779-1_23
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DOI: https://doi.org/10.1007/978-981-32-9779-1_23
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