Leah C. Byrne

Ocular Safety and Efficacy of AAV-mediated Tyrosinase Gene Augmentation in a Nonhuman Primate Model

This study evaluates the safety and efficacy of AAV-mediated TYR gene augmentation in nonhuman primates for OCA1 treatment, showing localized hypermelanosis and RPE transduction without severe adverse effects.

Systemic, RPE-directed AAV-Tyrosinase therapy restores ocular pigmentation in an OCA1 mouse model

This study demonstrates that systemic delivery of an RPE-directed AAV-Tyr vector restores ocular pigmentation and reduces photophobia in an OCA1 mouse model.

Structural dynamics insights into principles underlying the fitness of new broadly potent AAVs

The paper develops a new AAV engineering workflow, scAAVengr-Hunt, to create potent vectors and identifies a structural mechanism underlying their broad species and tissue potency.

Cell Type Specific Enhancers for Dorsolateral Prefrontal Cortex.

The paper identifies cell type-specific enhancers for the DLPFC in NHPs using single nucleus RNA-Seq and ATAC-Seq, validates them with AAV injections, and demonstrates functional expression in specific neuron types.

Machine learning sequence prioritization for cell type-specific enhancer design

The paper introduces SNAIL, a virus-based method using machine learning to design cell type-specific enhancer probes for targeted nuclear isolation and characterization.

Targeting ON-bipolar cells by AAV gene therapy stably reverses LRIT3-congenital stationary night blindness

This paper demonstrates that AAV gene therapy targeting ON-bipolar cells in a canine model of LRIT3-CSNB significantly improves night vision and restores LRIT3 function.

Parallel functional testing identifies enhancers active in early postnatal mouse brain

The paper identifies and validates enhancers active in early postnatal mouse brain using a modified MPRA approach and rAAV delivery.

Transcriptional Diversity of Medium Spiny Neurons in the Primate Striatum

This study identifies molecularly distinct medium spiny neuron types in the primate striatum, revealing new subtypes associated with specific anatomical compartments and functional roles.

In vivo directed evolution of AAV in the primate retina

This study uses directed evolution to create AAV vectors with enhanced gene delivery to the outer retina in primates.