Publicaciones en colaboración con investigadores/as de Centro de Investigación Médica Aplicada (4)

2024

  1. Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1

    EMBO molecular medicine, Vol. 16, Núm. 1, pp. 112-131

  2. Gene editing in liver diseases

    FEBS Letters

2020

  1. In Vitro and In Vivo Genetic Disease Modeling via NHEJ-Precise Deletions Using CRISPR-Cas9

    Molecular Therapy - Methods and Clinical Development, Vol. 19, pp. 426-437

2018

  1. CRISPR/Cas9-mediated glycolate oxidase disruption is an efficacious and safe treatment for primary hyperoxaluria type I

    Nature Communications, Vol. 9, Núm. 1