Review
Version 1
Preserved in Portico This version is not peer-reviewed
Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy
Version 1
: Received: 11 December 2023 / Approved: 12 December 2023 / Online: 12 December 2023 (16:11:02 CET)
A peer-reviewed article of this Preprint also exists.
Hasselbeck, S.; Cheng, X. Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy. Pharmaceuticals 2024, 17, 41. Hasselbeck, S.; Cheng, X. Molecular Marvels: Small Molecules Paving the Way for Enhanced Gene Therapy. Pharmaceuticals 2024, 17, 41.
Abstract
In the rapidly evolving landscape of genetic engineering, the advent of CRISPR-Cas technologies has catalyzed a paradigm shift, empowering scientists to manipulate the genetic code with unprece-dented accuracy and efficiency. Despite the remarkable capabilities inherent to CRISPR-Cas sys-tems, recent advancements have witnessed the integration of small molecule to augment their functionality, introducing new dimensions to the precision and versatility of gene editing applica-tions. This review delves into the synergy between CRISPR-Cas technologies and small molecule drugs, elucidating the pivotal role of chemicals in optimizing target specificity and editing efficiency. By examining a diverse array of applications, ranging from therapeutic interventions to agricultural advancements, we explore how the judicious use of chemicals enhances the precision of CRISPR-Cas-mediated genetic modifications. In this review we emphasize the significance of small molecule drugs in fine-tuning the CRISPR-Cas machinery, which allows researchers to exert meticulous con-trol over the editing process. We delve into the mechanisms through which these chemicals bolster target specificity, mitigate off-target effects, and contribute to the overall refinement of gene edit-ing outcomes. Additionally, we discuss the potential of chemical integration in expanding the scope of CRISPR-Cas technologies, enabling tailored solutions for diverse genetic manipulation challenges. As CRISPR-Cas technologies continue to evolve, the integration of small molecule drugs emerges as a crucial avenue for advancing the precision and applicability of gene editing techniques. This re-view not only synthesizes current knowledge but also highlights future prospects, paving the way for a deeper understanding of the synergistic interplay between CRISPR-Cas systems and chemical modulators in the pursuit of more controlled and efficient genetic modifications.
Keywords
Cas9; Small Molecules; Genome Editing
Subject
Chemistry and Materials Science, Medicinal Chemistry
Copyright: This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Comments (0)
We encourage comments and feedback from a broad range of readers. See criteria for comments and our Diversity statement.
Leave a public commentSend a private comment to the author(s)
* All users must log in before leaving a comment