Intrauterine Fetal Gene Therapy: Is That the Future and Is That Future Now?
| Autor: | Peddi NC; Pediatrics, PES Institute of Medical Sciences and Research, Kuppam, IND., Marasandra Ramesh H; Pediatrics, Kasturba Medical College, Mangalore, Mangalore, IND., Gude SS; Pediatrics, Guntur Medical College, Guntur, IND., Gude SS; Pediatrics, Guntur Medical College, Guntur, IND., Vuppalapati S; Pediatrics, PES Institute of Medical Sciences and Research, Kuppam, IND. |
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| Jazyk: | angličtina |
| Zdroj: | Cureus [Cureus] 2022 Feb 23; Vol. 14 (2), pp. e22521. Date of Electronic Publication: 2022 Feb 23 (Print Publication: 2022). |
| DOI: | 10.7759/cureus.22521 |
| Abstrakt: | Researchers are looking into techniques to intervene sooner and earlier in the disease process thanks to advances in disease genetics, etiologies, and prenatal diagnosis. We conducted a literature search in PubMed-indexed journals to provide an overview of the evolution of gene therapy, rationale for prenatal gene therapy, uses and risks of gene therapy, and ethical issues following the usage of gene therapy. Recent animal research has revealed that transmitting genetic material to a growing fetus through viral and non-viral vectors is conceivable besides proving how gene-editing technology is achieved by various mechanisms that utilize zinc finger nucleases, TAL effector nucleases, and clustered short palindromic repeats-Cas9 complex. This review offers an overview of the current knowledge in the field of prenatal gene therapy, as well as potential future research avenues. In addition, it weighs the risks of prenatal gene therapy, such as oncogenesis, genetic mutation transfer from mother to child, and fetal disruption, against the expected benefits, such as preventing the development of severe early-onset illness symptoms, targeting previously inaccessible organs, and establishing tolerance to the therapeutic transgenic protein, all of which lead to permanent somatic gene correction. This review discusses the scientific, ethical, legal, and sociological implications of these groundbreaking genetic disease prevention techniques, as well as the parameters that must be satisfied for a future clinical application to be considered. Competing Interests: The authors have declared that no competing interests exist. (Copyright © 2022, Peddi et al.) |
| Databáze: | MEDLINE |
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