Andres F. Leal
Bogota, DC, Colombia
Last Updated: 09/26/2026
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Found 30 publications
Tay-Sachs disease models: From cellular and animal models to treatment perspectives.
Journal: Molecular genetics and metabolism
Published: January 15, 2026
Expanding the Clinical Spectrum of RERE-Related Disorders: A Case Report of Neurodevelopmental Disorder with Brain Malformations Including Chiari Type I.
Journal: Molecular syndromology
Published: October 30, 2025
Variable Expressivity in Type 2 Familial Partial Lipodystrophy Related to a Pathogenic LMNA Variant R482: Maternal Transmission to Non-Identical Twins.
Journal: The application of clinical genetics
Published: September 26, 2025
Mucopolysaccharidosis VI: Therapeutic strategies and perspectives.
Journal: Molecular genetics and metabolism
Published: August 26, 2025
Epigenetic landscape in lysosomal storage disorders: mechanisms and modulation.
Journal: Frontiers in genetics
Published: August 05, 2025
Recent advances in mucopolysaccharidosis IVA treatment.
Journal: Orphanet journal of rare diseases
Published: June 05, 2025
Mesenchymal Stem Cell-Derived Extracellular Vesicles: Seeking into Cell-Free Therapies for Bone-Affected Lysosomal Storage Disorders.
Journal: International journal of molecular sciences
Published: June 03, 2025
CRISPR/Cas-Based Ex Vivo Gene Therapy and Lysosomal Storage Disorders: A Perspective Beyond Cas9.
Journal: Cells
Published: June 02, 2025
Yeast-Produced Human Recombinant Lysosomal β-Hexosaminidase Efficiently Rescues GM2 Ganglioside Accumulation in Tay-Sachs Disease.
Journal: Journal of personalized medicine
Published: March 23, 2025
Evaluation of the PP6D5 Polymer as a Novel Non-Viral Vector in the Development of a CRISPR/nCas9-Based Gene Therapy for Tay-Sachs Disease.
Journal: Pharmaceutics
Published: March 21, 2025
CRISPR/nCas9-Edited CD34+ Cells Rescue Mucopolysaccharidosis IVA Fibroblasts Phenotype.
Journal: International journal of molecular sciences
Published: March 19, 2025
Assessment of different promoters in lentiviral vectors for expression of the N-acetyl-galactosamine-6-sulfate sulfatase gene.
Journal: Journal of human genetics
Published: March 13, 2025
Last Updated: 09/26/2026