Niren Murthy

This study develops lipid nanoparticles (BLNPs) that deliver siRNAs to induce browning of white adipose tissue, improving metabolic health in mice.

This paper introduces PERCEPT, a CRISPR delivery platform that enables multi-organ genome editing in vivo by chemically modifying CRISPR enzymes for targeted and efficient delivery.

This study compares LNP and electroporation for delivering CRISPR to HSPCs, finding LNPs provide better cell viability and HbF induction with lower toxicity.

This paper develops a lipid nanoparticle (LNP) platform for non-viral CRISPR-mediated homology directed repair (HDR) to generate CAR T cells with improved efficiency and reduced toxicity compared to electroporation.

This paper develops a method to selectively kill cancer cells with TP53 mutations by using RNA-guided Cas12a2 to induce chromatin shredding through targeting cancer-specific transcripts.

This paper develops a swine reporter model to evaluate gene delivery vectors in a large animal system and demonstrates its utility for assessing biodistribution, safety, and administration methods.

The paper identifies RASGRP3 as a context-specific essential gene in uveal melanoma and develops a CRISPR-Cas13d RNA-targeting therapeutic that selectively eliminates uveal melanoma cells.

This study develops Neuro-PERC, a peptide-mediated CRISPR delivery method, enabling efficient in vivo neuronal editing in mice and pigs, and extending survival in a Huntington's disease model.

The paper investigates DNA repair in postmitotic neurons and shows that manipulating repair pathways can influence CRISPR editing outcomes.

The paper develops a cardiac microphysiological system to screen lipid nanoparticle/mRNA complexes and identifies a formulation with improved transfection efficiency and reduced off-target liver uptake.

This study demonstrates that lipid nanoparticle-delivered thermostable Cas9 RNPs achieve high-efficiency genome editing in mouse liver and lung tissues.

This study demonstrates that lipid nanoparticles can deliver mRNA for gene editing enzymes in utero, effectively editing multiple organs with low toxicity.