Aug 2026· Trends in Biotechnology· 0 citations· 82 references
Medicine
TL;DR
It is argued that as CRISPR biotechnologies advance beyond the relative simplicity of in vitro applications, the understanding must also evolve to address the challenges of optimizing “on-target” and “off-target” mutational activities across the diverse contexts in which they occur.
Abstract
Recent FDA-approved gene editing therapies illustrate not only the transformative potential of CRISPR biotechnologies in treating a broad range of diseases but also the spectrum of possible molecular variations CRISPR therapeutics can adopt. These include exagamglogene autotemcel, an ex vivo Cas9-mediated therapy for hemoglobinopathies, and kayjayguran abengcemeran, an in vivo therapy using a PAM-altered, base-editing Cas9 variant for an ultra-rare metabolic disorder. Together, these therapies underscore how far CRISPR has advanced beyond its original use as a tool in biological/biomedical research. Here, we argue that as CRISPR biotechnologies advance beyond the relative simplicity of in vitro applications, our understanding must also evolve to address the challenges of optimizing “on-target” and “off-target” mutational activities across the diverse contexts in which they occur.
The mechanisms of CRISPR-Cas9 gene editing, recent technological advancements, and prospects for cancer management are explored and insights into strategies for precise delivery, improved targeting accuracy, and the regulatory considerations surrounding CRISPR-Cas9 for oncological applications are provided.
Nikhil Rawal, Shruti Batra, Tanu Sharma et al.· Current pharmaceutical desig...· 0 citations
The success of CRISPR-Cas9 in monogenic diseases represents the prelude to precision medicine, whereas conquering complex diseases will require systematic leaps in target discovery, delivery technology, and safety profiles.
This review summarizes recent progress in applying CRISPR systems in vivo for cancer immunotherapy, focusing on approaches that target cancer cells and the tumor microenvironment, as well as those that directly engineer immune cell populations themselves.
Cole W. Christopher, Xiaoyu Zhou· Frontiers in Immunology· 0 citations
How CRISPR-enabled functional genomics approaches can reveal unexpected cancer dependencies and resistance mechanisms is outlined, and emerging applications of CRISPR-based diagnostics in oncology that convert precise nucleic acid sequence recognition into rapid mutation detection are discussed.
S. Grigg, Carolyn Shembrey, M. Fareh et al.· Nature Reviews Clinical Onco...· 1 citation
CRISPR has emerged as a next-generation gene-editing tool with the potential to target the molecular pathways associated with ageing and related disorders. It functions through RNA-guided Cas nucleases, directing DNA cleavage and utilizing the native DNA repair machinery for genetic manipulations. Advances in CRISPR te...
Sakshi Rathore, Akash Gupta, Kamal Shah et al.· Ageing Research Reviews· 0 citations
A major focus of this review is the inclusion of recent hybrid systems (VLPs, SORT-LNPs) and the recognition that chemical modification of guide RNAs is a critical parameter for therapeutic success and that hybrid systems and stimuli-responsive nanoparticles are poised to dominate the next 5 years of clinical developme...
M. Rezaee, F. Izadi, Saeed Nobaharian et al.· Beni-Suef University Journal...· 0 citations
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