· Methods in molecular biology· Vol 3075, pp.
41-57
· 0 citations
Medicine
TL;DR
Significant challenges remain for its broad applicability as a potential curative therapy for human genetic diseases, mainly related to ensuring efficient and safe delivery to target tissues.
Abstract
CRISPR tools are revolutionizing the landscape of genetic therapies, with the potential to cure a range of previously untreatable diseases. Among all the available genome editing technologies, prime editing is an especially versatile tool that enables precise genetic modifications, including point mutations, insertions, and deletions, without inducing double-strand breaks or requiring a donor DNA template. Through structural modifications and the development of novel systems with additional functionalities, prime editing has expanded its applicability with improved precision, efficacy, and safety. It is already being tested in clinical trials for chronic granulomatous disease, and many preclinical studies are underway. However, significant challenges remain for its broad applicability as a potential curative therapy for human genetic diseases, mainly related to ensuring efficient and safe delivery to target tissues.
This review systematically summarizes the key improvements and evolutionary progress in the prime editor design and its updated delivery systems, with a particular focus on innovative modifications that have successfully overcome technical barriers.
Shu-Ran Zhang, Leong Chang, Ya-Min Kong et al.· Current Gene Therapy· 0 citations
This review starts by examining the advantages and limitations of various physical methods for delivering Cas9, and highlights key applications of CRISPR systems in epigenetic modifications, and explores the use of CRISPR-Cas9 technology in genome editing, with a particular focus on base editing and prime editing.
Suhail Ahmad Sheikh, W. Shah, Azka Khan et al.· Journal of Population Therap...· 0 citations
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
The advent of CRISPR-Cas9 technology has revolutionized genome editing, enabling precise modifications to the human genome with unprecedented accuracy and sequence specificity. This review examines current mechanistic insights, translational advances, and clinical developments in gene editing, focusing on applications...
Abraham E. Ubhenin, F. Adamude, D. O. Ochalefu et al.· Nigerian Medical Journal· 0 citations
A multidimensional framework is proposed in which gene therapy platforms are evaluated according to three intrinsic properties—genetic precision, temporal control, and dosage tunability—while delivery, clinical maturity, and disease context act as major translational constraints.
Jared Wieland, Peyton Jackson, William Penrod et al.· Cells· 0 citations
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