Lysosomal storage diseases: current therapies and future alternatives
Andrés Felipe Leal, Ángela J. Espejo, Oscar F. Sánchez, Carlos Manuel Ramírez, Luis H. Reyes, Juan C. Cruz, Carlos Javier Alméciga-Díaz
Topics & Concepts
Genome editingGenetic enhancementSubstrate reduction therapyLysosomal storage disordersLysosomeEnzyme replacement therapyComputational biologyLysosomal storage diseaseENCODECRISPRCell therapyGenomeBiologyBioinformaticsTransplantationStem-cell therapyStem cellMedicineGeneGeneticsDiseaseEnzymePathologyInternal medicineBiochemistryCRISPR and Genetic EngineeringLysosomal Storage Disorders ResearchAutism Spectrum Disorder Research