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International Journal of Molecular Sciences|July 12, 2025
CRISPRa-Mediated Increase of OPA1 Expression in Dominant Optic AtrophyGiada Becchi, Michael Whitehead, Joshua P Harvey, et al.
Human Molecular Genetics|June 2, 2022
Modelling autosomal dominant optic atrophy associated with OPA1 variants in iPSC-derived retinal ganglion cellsPaul E Sladen, Katarina Jovanovic, Rosellina Guarascio, et al.
International Journal of Molecular Sciences|February 10, 2024
AAV-RPGR Gene Therapy Rescues Opsin Mislocalisation in a Human Retinal Organoid Model of RPGR-Associated X-Linked Retinitis PigmentosaPaul E Sladen, Arifa Naeem, Toyin Adefila-Ideozu, et al.
Molecular Therapy. Nucleic Acids|September 30, 2021
CRISPR-Cas9 correction of OPA1 c.1334G>A: p.R445H restores mitochondrial homeostasis in dominant optic atrophy patient-derived iPSCsPaul E Sladen, Pedro R L Perdigão, Grace Salsbury, et al.
Cells|June 28, 2023
Eupatilin Improves Cilia Defects in Human CEP290 Ciliopathy ModelsJulio C Corral-Serrano, Paul E Sladen, Daniele Ottaviani, et al.
Acta Neuropathologica Communications|February 13, 2025
Disruption of mitochondrial homeostasis and permeability transition pore opening in OPA1 iPSC-derived retinal ganglion cellsMichael Whitehead, Joshua P Harvey, Paul E Sladen, et al.
International Journal of Molecular Sciences|January 10, 2026
KCNV2-Deficient Retinal Organoid Model of Cone Dystrophy-In Vitro Screening for AAV Gene Replacement TherapySophie L Busson, Arifa Naeem, Silvia Ferrara, et al.
Molecular Therapy. Nucleic Acids|December 17, 2025
Adenine base editor correction of pathogenic variations associated with inherited retinal dystrophy in patient iPSC and retinal organoidsAmy Leung, Pedro R L Perdigão, Almudena Sacristan-Reviriego, et al.
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