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Orphanet Journal of Rare Diseases|October 15, 2025
Recent advances in mucopolysaccharidosis IVA treatmentAndrés Felipe Leal, Harry PachajoaGene Therapy|May 17, 2022
Efficient CRISPR/Cas9 nickase-mediated genome editing in an in vitro model of mucopolysaccharidosis IVAAndrés Felipe Leal, Carlos Javier Alméciga-DíazInternational Journal of Molecular Sciences|July 12, 2025
Mesenchymal Stem Cell-Derived Extracellular Vesicles: Seeking into Cell-Free Therapies for Bone-Affected Lysosomal Storage DisordersAndrés Felipe Leal, Harry Pachajoa, Shunji TomatsuInternational Journal of Molecular Sciences|August 10, 2024
Potential Targeting Mechanisms for Bone-Directed TherapiesBetul Celik, Andrés Felipe Leal, Shunji TomatsuFrontiers in Genetics|November 27, 2025
Epigenetic landscape in lysosomal storage disorders: mechanisms and modulationAndrés Felipe Leal, Harry Pachajoa, Shunji TomatsuCells|August 13, 2025
CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9Andrés Felipe Leal, Luis Eduardo Prieto, Harry PachajoaMolecular Syndromology|February 11, 2026
Expanding the Clinical Spectrum of RERE-Related Disorders: A Case Report of Neurodevelopmental Disorder with Brain Malformations Including Chiari Type IHarry Pachajoa, Angela María Gutierrez-Obando, Andrés Felipe LealJournal of Human Genetics|June 10, 2025
Assessment of different promoters in lentiviral vectors for expression of the N-acetyl-galactosamine-6-sulfate sulfatase geneBetul Celik, Andrés Felipe Leal, Shaukat Khan, et al.International Journal of Molecular Sciences|October 27, 2022
Regulation of Molecular Targets in Osteosarcoma TreatmentBetul Celik, Kader Cicek, Andrés Felipe Leal, et al.International Journal of Molecular Sciences|November 25, 2023
Mucopolysaccharidosis IVA: Current Disease Models and DrawbacksAndrés Felipe Leal, Carlos Javier Alméciga-Díaz, Shunji TomatsuPageof 3