Intrauterine Fetal Gene Therapy: Is That the Future and Is That Future Now?

Nikhil Chowdary Peddi,Sravya Vuppalapati,Sai Sreeya Gude,Harshita Marasandra Ramesh,Sai Sravya Gude

doi:10.7759/cureus.22521

Abstract

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.

Highlights

BackgroundGene therapy involves delivering genetic material to a cell to elicit a therapeutic effect, such as correcting an error or giving cells a new function
Recent animal research has revealed that transmitting genetic material to a growing fetus through viral and nonviral 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

Summary

Introduction

Gene therapy involves delivering genetic material to a cell to elicit a therapeutic effect, such as correcting an error or giving cells a new function. An adult patient with severe b-thalassemia who had been reliant on monthly transfusions because infancy recently showed the possibility of an ex vivo gene transfer technique for the treatment of thalassemia He was transfusion-free for at least 21 months after autologous transplantation of gene-modified hematopoietic stem cells using a lentivirus expressing b-globin. The most important risks of IUGT include the ability to disrupt normal fetal development [24]; carry an elevated risk of germline transfer of genetic modification [24]; and induce genotoxicity and/or oncogenesis [24] These are the primary identified or inferred specific dangers of prenatal gene therapy that generate ethical concerns. Because these off-target evaluations for in utero gene editing were conducted in a biased manner, future robust and unbiased investigations are needed [10]

Conclusions

Findings

Disclosures