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Author

O. Ryzhkova

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#gene editing Open access Sep 2026

Comparison of Prime Editing and CRISPR-Cas Mediated HDR in Correcting CFTR-F508del Mutation in Patient iPSC-Derived Airway Basal Cells

Functional analysis evaluating the effectiveness of gene editing revealed that prime editing of the F508del mutation in the CFTR gene—using the pegRNA4 combined with the PE2-NG variant—restores CFTR channel conductance, comparable to triple therapy with CFTR modulators.

O. Volodina, A. Demchenko, E. Kondrateva et al. · 0 citations
Open access Aug 2026

Exploring the clinical and mutational spectrum of MORC2-associated disorders.

It is demonstrated that early-onset MORC2-associated disorders segregate into two principal neurological phenotypes: a predominantly neuromuscular form and a central nervous system-predominant form.

A. Murtazina, Eugenii Tatarsky, I. Viakhireva et al. · 0 citations

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