Gene therapy: Comprehensive overview and therapeutic applications

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dc.contributor.authorSayed, Nilofer
dc.contributor.authorAllawadhi, Prince
dc.contributor.authorKhurana, Amit
dc.contributor.authorSingh, Vishakha
dc.contributor.authorNavik, Umashanker
dc.contributor.authorPasumarthi, Sravan Kumar
dc.contributor.authorKhurana, Isha
dc.contributor.authorBanothu, Anil Kumar
dc.contributor.authorWeiskirchen, Ralf
dc.contributor.authorBharani, Kala Kumar
dc.date.accessioned2024-01-21T10:55:01Z
dc.date.accessioned2024-08-14T07:44:18Z
dc.date.available2024-01-21T10:55:01Z
dc.date.available2024-08-14T07:44:18Z
dc.date.issued2022-02-03T00:00:00
dc.description.abstractGene therapy is the product of man's quest to eliminate diseases. Gene therapy has three facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where the desired gene in the form of plasmids and viral vectors, are directly administered and finally gene editing based therapy where mutations are modified using specific nucleases such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered regulatory interspaced short tandem repeats (CRISPR)/CRISPR-associated protein (Cas)-associated nucleases. Transfer of gene is either through transformation where under specific conditions the gene is directly taken up by the bacterial cells, transduction where a bacteriophage is used to transfer the genetic material and lastly transfection that involves forceful delivery of gene using either viral or non-viral vectors. The non-viral transfection methods are subdivided into physical, chemical and biological. The physical methods include electroporation, biolistic, microinjection, laser, elevated temperature, ultrasound and hydrodynamic gene transfer. The chemical methods utilize calcium- phosphate, DAE-dextran, liposomes and nanoparticles for transfection. The biological methods are increasingly using viruses for gene transfer, these viruses could either integrate within the genome of the host cell conferring a stable gene expression, whereas few other non-integrating viruses are episomal and their expression is diluted proportional to the cell division. So far, gene therapy has been wielded in a plethora of diseases. However, coherent and innocuous delivery of genes is among the major hurdles in the use of this promising therapy. Hence this review aims to highlight the current options available for gene transfer along with the advantages and limitations of every method. � 2022en_US
dc.identifier.doi10.1016/j.lfs.2022.120375
dc.identifier.issn243205
dc.identifier.urihttp://10.2.3.109/handle/32116/4309
dc.identifier.urlhttps://linkinghub.elsevier.com/retrieve/pii/S0024320522000753
dc.language.isoen_USen_US
dc.publisherElsevier Inc.en_US
dc.subjectGene deliveryen_US
dc.subjectGene therapyen_US
dc.subjectNon-viral vectorsen_US
dc.subjectTransfectionen_US
dc.subjectViral vectorsen_US
dc.titleGene therapy: Comprehensive overview and therapeutic applicationsen_US
dc.title.journalLife Sciencesen_US
dc.typeReviewen_US
dc.type.accesstypeClosed Accessen_US

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