Lysosomal storage diseases: from pathophysiology to therapy.
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Sanfilippo syndrome: causes, consequences, and treatmentsCheckpoints to the Brain: Directing Myeloid Cell Migration to the Central Nervous SystemNew therapeutic approaches for Krabbe disease: The potential of pharmacological chaperonesTFEB at a glanceLysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular ToxicityIdentification of Small Molecule Compounds for Pharmacological Chaperone Therapy of AspartylglucosaminuriaBioinformatic Analysis of the Human Recombinant Iduronate 2-Sulfate Sulfatase.Induced pluripotent stem cell models of lysosomal storage disorders.Newborn Screening for Lysosomal Storage Diseases: A Concise Review of the Literature on Screening Methods, Therapeutic Possibilities and Regional ProgramsGene therapy for lysosomal storage disorders: a good start.Heat shock protein-based therapy as a potential candidate for treating the sphingolipidoses.Azasugar inhibitors as pharmacological chaperones for Krabbe diseaseMucopolysaccharidosis VI in cats - clarification regarding genetic testingMutations in ARSB in MPS VI patients in India.Lysosomes relax in the cellular suburbs.Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease.The lysosome as a command-and-control center for cellular metabolism.Disease models for the development of therapies for lysosomal storage diseases.Disease manifestations and burden of illness in patients with acid sphingomyelinase deficiency (ASMD)Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.Pharmacological Chaperone Therapy: Preclinical Development, Clinical Translation, and Prospects for the Treatment of Lysosomal Storage Disorders.Gene therapy for monogenic disorders of the bone marrow.Recent advances in gene therapy for lysosomal storage disorders.Progress toward improved therapies for inborn errors of metabolism.Lysosomal targeting strategies for design and delivery of bioactive for therapeutic interventions.Lysosomal enzyme replacement therapies: Historical development, clinical outcomes, and future perspectives.Glycomimetic-based pharmacological chaperones for lysosomal storage disorders: lessons from Gaucher, GM1-gangliosidosis and Fabry diseases.Treatment for Krabbe's disease: Finding the combination.Perspective on innovative therapies for globoid cell leukodystrophy.Mechanisms and functions of lysosome positioning.Modifying blood-brain barrier transport to bring hope for patients with lysosomal storage diseases.Drug delivery in overcoming the blood-brain barrier: role of nasal mucosal graftingFrom Lysosomal Storage Diseases to NKT Cell Activation and BackTargeting and delivery of therapeutic enzymes.Exacerbating and reversing lysosomal storage diseases: from yeast to humans.Signalling Pathways Controlling Cellular Actin Organization.Lysoplex: An efficient toolkit to detect DNA sequence variations in the autophagy-lysosomal pathway.The Underexploited Role of Non-Coding RNAs in Lysosomal Storage Diseases.Molecular Imaging of Hydrolytic Enzymes Using PET and SPECT.Recombinant production and affinity purification of the FraC pore forming toxin using hexa-His tag and pET expression cassette.
P2860
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P2860
Lysosomal storage diseases: from pathophysiology to therapy.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年学术文章
@wuu
2015年学术文章
@zh-cn
2015年学术文章
@zh-hans
2015年学术文章
@zh-my
2015年学术文章
@zh-sg
2015年學術文章
@yue
2015年學術文章
@zh
2015年學術文章
@zh-hant
name
Lysosomal storage diseases: from pathophysiology to therapy.
@en
type
label
Lysosomal storage diseases: from pathophysiology to therapy.
@en
prefLabel
Lysosomal storage diseases: from pathophysiology to therapy.
@en
P1476
Lysosomal storage diseases: from pathophysiology to therapy
@en
P2093
Generoso Andria
P304
P356
10.1146/ANNUREV-MED-122313-085916
P577
2015-01-01T00:00:00Z