Bikash Ranjan Pattnaik

DHA deficiency drives neural oxidized-lipid stress and microglial activation

This study shows that DHA deficiency causes lipid remodeling and microglial activation in mice, leading to oxidative stress and visual dysfunction, with APOE-dependent lipid disposal and galectin-3 involvement.

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

The paper uses CRISPR screening to identify genes that enhance nonviral genome editing efficiency in human cells, finding six genes that increase editing efficiency by up to five-fold.

Mitochondrial fusion controls the development of specialized mitochondrial structure and metabolism in rod photoreceptor cells

This study shows that mitochondrial fusion via MFN1 and MFN2 is essential for developing specialized mitochondrial structures and maintaining energy metabolism in rod photoreceptor cells.

Prevenying vision loss in a mouse model of Leber Congenital Amaurosis by engineered tRNA

This study demonstrates that engineered tRNA can restore vision in a mouse model of Leber Congenital Amaurosis by suppressing a premature termination codon.

Evaluation of subretinally delivered Cas9 ribonucleoproteins in murine and porcine animal models highlights key considerations for therapeutic translation of genetic medicines

This study evaluates subretinal delivery of Cas9 ribonucleoproteins in mice and pigs, showing variable editing efficiency and highlighting species-specific challenges for therapeutic translation.

Nonviral base editing of KCNJ13 mutation preserves vision in an inherited retinal channelopathy

This paper demonstrates nonviral base editing of a KCNJ13 mutation in a mouse model of retinal degeneration, preserving vision through functional rescue of Kir7.1 channels.

Comprehensive Analysis of CRISPR Base Editing Outcomes for Multimeric Protein

The paper evaluates CRISPR base editors for correcting a KCNJ13 mutation causing LCA16, finding high on-target efficiency but negligible channel function due to bystander edits and mRNA structural issues.

Human iPSC modeling reveals mutation-specific responses to gene therapy in Best disease

This study uses iPSC-RPE models to evaluate gene augmentation and editing for Best disease, finding that some mutations respond to gene therapy while others require genome editing.

Gene augmentation and read-through rescue channelopathy in an iPSC-RPE model of congenital blindness

The paper tests read-through drugs and gene therapy in patient-derived iPSC-RPE cells with a KCNJ13 mutation to restore Kir7.1 channel function and rescue channelopathy in LCA16.