Author

B. Pattnaik

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Open access Aug 2026

Reprogramming translation for rare disease therapy: challenges posed by large genes

This study offers the most compelling clinical evidence so far that gene replacement therapy can safely restore signi ﬁ cant hearing in children with OTOF-related deafness, with bene ﬁ ts lasting up to 2.5 years, setting a new standard for treating inherited sensory disorders.

B. Pattnaik, Jose-Alein Sahel · Signal Transduction and Targ... · 0 citations

Mar 2025

Genome-Wide CRISPR Screening Identifies Cellular Factors Controlling Nonviral Genome Editing Efficiency

A novel genome-wide CRISPR screening strategy that will facilitate the systematic engineering of novel nonviral genome editing delivery methods, where the identified novel gene hits can be further used to increase editing efficiency for other therapeutically relevant cell types.

Shivani Saxena, Meha Kabra, Amr A. Abdeen et al. · bioRxiv · 2 citations

Open access Aug 2026

Genome-wide CRISPR screening identifies cellular factors controlling nonviral genome editing efficiency

To systematically map cellular factors constraining nonviral genome editing, influencing uptake and intracellular trafficking, we develop a genome-wide CRISPR screening platform linking perturbation of 19,114 genes to editing outcomes in human cells. We identify six negative regulators of delivery whose depletion incre...

Shivani Saxena, Meha Kabra, Amr A. Abdeen et al. · Nature Communications · 0 citations