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Brommel, C.

Publications and source records attributed to Brommel, C..

2 recordsLinked to original sources

Sensitive Chromosomal Translocation Quantitation from Amplicon Sequencing Using Primer-Anchored Statistical Translocation Analysis (PASTA)

Chromosomal translocations are rare structural rearrangement outcomes of genome editing, requiring analytical frameworks that combine high quantitative accuracy with performant sensitivity and specificity. Amplicon sequencing offers a scalable means to detect rare rearrangements with ultra-deep targeted sequencing, but existing methods often rely on heuristic thresholds or ad hoc normalization steps that limit reproducibility and have unknown analytical performance. Here, we present a computational tool we call PASTA (Primer-Anchored Statistical Translocation Analysis), using a count-based differential-event statistical framework to quantify and statistically confirm translocation junctions from targeted amplicon sequencing data. Comparison of this method to ddPCR demonstrates that quantitation is highly accurate, and outperforms other NGS-based methods even when randomized adapter chemistry is not present in amplicon sequencing structures. To measure analytical performance, we create a benchmarking dataset for measuring chromosomal translocation analysis performance with frequencies ranging from 1% to sub-0.01%, and demonstrate that the method can detect frequencies down to 0.01% with >75% sensitivity when sufficient read depth is present. Taken together, this work demonstrates using amplicon sequencing with PASTA as a bioinformatics analysis tool is a solution for translocation detection in amplicon sequencing genotoxicity assessments, enabling identification of rare genome rearrangements in both research and preclinical applications

bioinformatics↗

High ionic strength vector formulations enhance gene transfer to airway epithelia

A fundamental challenge for cystic fibrosis (CF) gene therapy is ensuring sufficient ransduction of airway epithelia to achieve therapeutic correction. Hypertonic saline (HTS) is frequently administered to people with CF to enhance mucus clearance. HTS transiently disrupts epithelial cell tight unctions, but its ability to improve gene transfer has not been investigated. Here we asked if increasing the concentration of NaCl enhances the transduction efficiency of three gene therapy vectors: adenovirus, AAV, and lentiviral vectors. Vectors formulated with 3-7% NaCl exhibited markedly increased transduction for all hree platforms, leading to anion channel correction in primary cultures of human CF epithelial cells and enhanced gene transfer in mouse and pig airways in vivo. The mechanism of transduction enhancement nvolved tonicity but not osmolarity or pH. Formulating vectors with a high ionic strength solution is a simple strategy to greatly enhance efficacy and immediately improve preclinical or clinical applications. One Sentence SummaryFormulating adenoviral, AAV, and lentiviral vectors with hypertonic saline remarkably enhances lung gene transfer. (114 characters, including spaces)

molecular biology↗