P. Hariharan*1, G. Hemalatha2, J. Karthi3, S. Swarnalatha4
2 developments in the corpus
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International Journal of Medical & Pharmaceutical Sciences
First-in-human cell culture demonstration of CRISPR-mediated uricase gene restoration with functional enzyme activity, but lacking animal models, toxicology data, or any human in vivo evidence needed for clinical translation.
International Journal of Medical & Pharmaceutical Sciences
This is a mechanistic proof-of-concept study in cultured cells demonstrating CRISPR-mediated gene integration and enzymatic activity, not a clinical trial or in vivo validation; it raises the question of whether this approach could work therapeutically rather than answering it.