Bruce T. Lahn

This study evaluates the 12-month outcomes of vesemnogene lantuparvovec, an intrathecal gene therapy for SMA in children under 24 months in LMICs, showing safety and motor milestone improvements.

MuteFree eliminates ITR mutations in AAV transfer plasmids, achieving 0% mutation rates and resolving production issues in GMP manufacturing.

This study demonstrates that gene therapy using AAV vectors can rescue retinal degeneration in PROM1-IRD patient and mouse models by restoring PROM1 expression and photoreceptor structure.

miniVec is a new plasmid backbone that eliminates antibiotic resistance genes and improves plasmid yield and functionality for clinical applications.

This paper reports on a phase 1 trial of vesemnogene lantuparvovec, an affordable gene therapy for SMA, showing safety and improved motor milestones in young children.

The paper links cellular developmental potency to genomic transcriptional potency, showing that gene occlusion via chromatinization restricts lineage potential.

The paper surveys lab-made plasmids globally and finds nearly half contain errors, with 40% of transfer plasmids having mutations in ITR regions due to instability.

The paper identifies gene occlusion erasure as a defining difference between naive and primed pluripotency states, showing Esrrb-dependent deocclusion sustains naive pluripotency.