Targeted Gene Editing for PKD: Optimizing CRISPR / Cas9 Delivery Through Electroporation and Lipid Nanoparticles

Raksheet Reddy Gummakonda

Abstract: The state-of-the-art laboratory at hand indicates that, concerning Polycystic Kidney Disease (PKD), nothing is impossible to manage using gene-editing tools, such as CRISPR-Cas9. PKD is a disorder in which the fluid-filled sacs develop within the kidneys, enlarging them and impairing their activity. New advances in CRISPR-Cas9 editing, which allows targeting particular parts of the DNA, provide access to eliminating the mutations that lead to the development of PKD. The trick is that it is not yet locked in as far as success is concerned-it is stated that in certain studies, better results have been achieved working on the PKD1 and PKD2 genes, as compared to the others. Such incoherence portrays the emergence of new technical challenges. Experts' conversations stress the need to come to an agreement on how CRISPR/Cas9 should be used on human embryos. This paper discusses the development of CRISPR/Cas9 as well as its application to control polycystic kidney disease (PKD), and the ethical implications thereof. Going through the previous work and contemporary discussion, the paper will explain what CRISPR/Cas9 technology would offer to PKD and highlight the limitations of the technology, contributing to the overall discussion of whether gene editing has a potentially bright future in healthcare.

Keywords: Polycystic Kidney Disease (PKD), Autosomal Dominant Polycystic Kidney Disease (ADPKD), Autosomal Recessive Polycystic Kidney Disease (ARPKD), CRISPR/Cas9 gene editing, PKD 1 & PKD2 mutations, Lipid Nanoparticles (LNP) delivery, Electroporation, Gene therapy, Genetic disorders, Kidney cysts, Gene editing ethics, Off-target effects, Cyst formation reduction, Therapeutic gene delivery, In Vivo applications, Polycystin protein (PC 1, and PC2), Polycystic Kidney cell culture models, Selective gene targeting

How to Cite?: Raksheet Reddy Gummakonda, "Targeted Gene Editing for PKD: Optimizing CRISPR / Cas9 Delivery Through Electroporation and Lipid Nanoparticles", Volume 14 Issue 7, July 2025, International Journal of Science and Research (IJSR), Pages: 187-200, https://www.ijsr.net/getabstract.php?paperid=SR25630213206, DOI: https://dx.doi.org/10.21275/SR25630213206