Recent insights into lysosomal acid lipase deficiency
Melanie Korbelius, Katharina B. Kuentzel, I Bradić, Nemanja Vujić, Dagmar Kratky
Topics & Concepts
Enzyme replacement therapyDyslipidemiaLysosomeGenetic enhancementLysosomal storage diseaseHematopoietic stem cell transplantationLipaseBiologySubstrate reduction therapyStem cellEnzymeImmunologyMedicineBiochemistryDiseaseGeneInternal medicineCell biologyLysosomal Storage Disorders ResearchCalcium signaling and nucleotide metabolismAutophagy in Disease and Therapy