Niren Murthy

Browning Lipid Nanoparticles Deliver Metabolic Benefits via Transient Conversion of White Adipose Tissue

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

Chemically modified CRISPR enzymes for multi-organ genome editing in vivo

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.

LNP-mediated BCL11A Editing Corrects Sickling Phenotypes and Preserves HSPC Fitness Compared to Electroporation

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

A lipid nanoparticle platform for high yield CRISPR-targeted homology directed repair enables fully non-viral CAR T cell generation

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.

Selective Elimination of TP53 Mutant Cells by Transcript-Activated Chromatin Shredding

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.

Swine reporter model for preclinical evaluation and characterization of gene delivery vectors

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.

CRISPR-Cas13d-mediated targeting of a context-specific essential gene enables selective elimination of uveal melanoma

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.

Efficient in vivo mammalian neuron editing using peptide-mediated CRISPR enzyme delivery

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.

Neuronal DNA repair reveals strategies to influence CRISPR editing outcomes

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

A cardiac microphysiological system for screening lipid nanoparticle/mRNA complexes predicts in vivo efficacy for heart transfection

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.

Non-viral gene editing in utero with lipid nanoparticles complexed to mRNA

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