Review Paper:
CRISPR/Cas gene
editing based therapy approach in clinical therapeutics
Munuru Srikanth, Manikanta Swamy Uttaravilli, Shaik Sharmila Begum, Mollety Bharati
and Geetha R.V.
Res. J. Biotech.; Vol. 20(8); 256-263;
doi: https://doi.org/10.25303/208rjbt2560263; (2025)
Abstract
Advanced inventions in genetic engineering towards clinical therapeutics have been
rising over the past decade, which has brought a new approach in treating diseases
like HIV, cancer, pulmonary diseases, genetic disorders, hereditary transferring
disorders etc. Gene editing-based therapy holds specific molecular tools i.e. ZFN,
TALENS and CRISPR/Cas9. These gene-editing techniques show precise and accurate
modification of the eukaryotic genome. The application of CRISPR/Cas had become
a trending gene-editing technique as its effectiveness in editing was comparatively
high. Nowadays, gene-editing based therapy includes gene therapy approaches i.e.
in vivo and ex vivo strategies, to reach efficient clinical treatment requirements.
A new strategy of editing was made by assigning ss-ODN along with CRISPR, TALENS
and ZFN, which can also increase the efficiency of gene editing, leading to promising
results.
In this review, we discussed advanced gene-editing techniques and few recent implementations
of genome editing to combat several diseases and disorders along with their effective
clinical strategies. We also added a few CRISPR/Cas9-developed animal models that
are used in treating human genetic disorders.
