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Julen Torrens-Baile

ORCID: 0009-0005-5541-6462
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About
Contact & Profiles
A5093659537
Research Areas
  • Porphyrin Metabolism and Disorders
  • CRISPR and Genetic Engineering
  • Genetic and Kidney Cyst Diseases
  • Biomedical Research and Pathophysiology
  • Virus-based gene therapy research

Universidad de Navarra
 2024

 Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1
OPENALEX - Publications 
Laura Torella Julia Klermund Martin Bilbao‐Arribas Ibón Tamayo Geoffroy Andrieux and 14 more Kay O. Chmielewski África Vales Cristina Olagüe Daniel Moreno Ivan Raimondi Amaya Abad Julen Torrens-Baile Eduardo Salido Maite Huarte Mikel Hernáez Melanie Boerries Toni Cathomen Nerea Zabaleta Gloria González‐Aseguinolaza

The therapeutic use of adeno-associated viral vector (AAV)-mediated gene disruption using CRISPR-Cas9 is limited by potential off-target modifications and the risk uncontrolled integration genomes into CRISPR-mediated double-strand breaks. To address these concerns, we explored AAV-delivered paired Staphylococcus aureus nickases (D10ASaCas9) to target Hao1 for treatment primary hyperoxaluria type 1 (PH1). Our study demonstrated effective disruption, a significant decrease in glycolate...

10.1038/s44321-023-00008-8 article EN cc-by EMBO Molecular Medicine 2024-01-05
 FRI-152-YI Gene editing to treat liver metabolic disorders: harnessing paired Cas9-Nickases for efficient and safe treatment of primary hyperoxaluria type 1
OPENALEX - Publications 
Laura Torella Martin Bilbao‐Arribas Julia Klermund Geoffroy Andrieux África Vales Aranguren and 6 more Cristina Olague Micheltorena Julen Torrens-Baile Eduardo Salido Toni Cathomen Nerea Zabaleta Gloria González‐Aseguinolaza

10.1016/s0168-8278(24)01975-5 article EN Journal of Hepatology 2024-06-01
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