Jeffrey Matthijn Beekman

This study evaluates ELX-02's effectiveness in restoring CFTR function across various nonsense variants using patient-derived intestinal organoids, finding limited and variable rescue effects.

This study identifies spike and host glycan determinants of HKU1 airway tropism by showing preferential binding to 9-O-acetylated 2,8-linked disialosides and structural insights into ligand recognition.

UDIST separates shape and texture in microscopy images into independent latent subspaces, enabling unbiased phenotypic analysis across biological scales.

The paper reports the structure of the mammalian ciliary transition zone microtubule doublet using cryo-electron tomography, revealing unique proteins and their roles in microtubule stabilization and transport regulation.

This study develops a personalized multi-assay platform using nasal epithelial cells to assess ciliary function and therapeutic response in primary ciliary dyskinesia.

The paper uses expansion microscopy to map the 3D microtubule organization in human airway multiciliated cells, revealing the role of NEDD1 in basal foot-dependent microtubule organization.

This study develops a method to differentiate human nasal epithelial cells into airway-like tissues using small molecules, creating disease models for respiratory conditions.

This study uses expansion microscopy to show SARS-CoV-2 exits cells via Rab27-dependent amphisome fusion, identifying a potential therapeutic target.

The paper describes the immortalization of human nasal and bronchial airway epithelial cells using hTERT overexpression, enabling long-term genome editing studies.

This study examines how different TTC7A missense mutations in pediatric IBD patients lead to distinct intestinal epithelial phenotypes.

This study identifies SSNA1 as a centriolar protein in the distal lumen that promotes ciliogenesis without microtubule association, challenging previous views of its function.

The paper predicts and validates responses to CFTR modulator combinations in patients with rare mutations using structural data and functional assays.

The paper demonstrates that ACE-tRNAs can suppress nonsense mutations in CF by rescuing CFTR transcript abundance and channel function in multiple models.

The paper screens 1400 FDA-approved drugs in CF patient-derived intestinal organoids using a functional assay to identify potential repurposing for rare CF genotypes.

This paper introduces a fluorescent reporter system for evaluating and enhancing prime editing efficiency in genomic targets.

This study evaluates prime editing for modeling cancer mutations and repairing CFTR in intestinal organoids, showing its potential but also highlighting challenges in efficiency and off-target effects.