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Justice, B.

Publications and source records attributed to Justice, B..

2 recordsLinked to original sources

The Non-Human Primate Sample Size Calculator: a design tool for longitudinal research projects

Nonhuman primates (NHP) are crucial models of human health and disease and offer the opportunity to bridge the gap between basic research and the clinic. Given the ethical considerations, high costs, and logistical constraints associated with NHP studies, careful planning is essential to maximize scientific rigor while minimizing animal use. Statistical power calculations, to determine optimal sample size, require knowing the expected number of animals at each timepoint, a challenge for longitudinal studies that must account for natural deaths or disease during a study. Here, we leveraged data from a recent study detailing NHP life- and healthspans to develop a web-based NHP study design tool, available at midas.wakehealth.edu. For 11 NHP species relevant to biomedical research, users can provide study details to generate estimates of sample counts based on natural healthspan trajectories. We envision this tool being used by colony managers and investigators for both study design and monitoring colony health over the course of a study.

physiology↗

Compact adenine base editors to enable therapeutic rescue of Duchenne muscular dystrophy

Adenine base editors (ABEs) have emerged as a powerful gene-editing technology enabling precise and programmable adenine-to-guanine substitutions across the genome. However, their translation into in vivo therapeutics is limited by delivery challenges, as their size exceeds the packaging capacity of adeno-associated virus (AAV). Here, we report the discovery, structural characterization, and engineering of two compact, highly active ABEs built on novel deaminases and Cas9d nucleases, enabling all-in-one, single-vector AAV delivery. Applying these compact ABEs to Duchenne muscular dystrophy (DMD), we demonstrate efficient disruption of conserved splice-acceptor sites at dystrophin exons 45 and 51 in human skeletal muscle cells, enabling therapeutically relevant exon skipping. Together, these ABEs help expand the therapeutic reach of base editing towards diverse tissue types and disease targets.

bioengineering↗