ReviewJournal of inherited metabolic disease2010
Therapeutic approaches for neuronopathic lysosomal storage disorders.
Review in Journal of inherited metabolic disease, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 40 citations in OpenAlex.
- Characterization of mesenchymal stem cells in mucolipidosis type II (I-cell disease).Turkish journal of biology = Turk biyoloji dergisi · 2019Article
- Expanding the Boundaries of Biotherapeutics with Bispecific Antibodies.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2018Review
- A Basic ApoE-Based Peptide Mediator to Deliver Proteins across the Blood-Brain Barrier: Long-Term Efficacy, Toxicity, and Mechanism.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Blood-brain barrier structure and function and the challenges for CNS drug delivery.Journal of inherited metabolic disease · 2013Review
- Newborn screening for lysosomal storage disorders and other neuronopathic conditions.Developmental disabilities research reviews · 2013Review
- A Dysmorphometric Analysis to Investigate Facial Phenotypic Signatures as a Foundation for Non-invasive Monitoring of Lysosomal Storage Disorders.JIMD reports · 2013Article
- Annexins as organizers of cholesterol- and sphingomyelin-enriched membrane microdomains in Niemann-Pick type C disease.Cellular and molecular life sciences : CMLS · 2012Review
- Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.Drug delivery and translational research · 2012Article
- Imaging gene delivery in a mouse model of congenital neuronal ceroid lipofuscinosis.Gene therapy · 2011Article
- Lysosomal storage causes cellular dysfunction in mucolipidosis II skin fibroblasts.The Journal of biological chemistry · 2011Article
- Lysosomal storage disorders: molecular basis and laboratory testing.Human genomics · 2011Review
- Article
Corrections and comments
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Authors and funding
1 author at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Therapy of the central nervous system (CNS) manifestations of lysosomal storage diseases (LSDs) has remained a major challenge because of its inability to deliver therapeutic agents efficiently across the intact blood-brain barrier. Non-specific therapies such as hematopoietic stem cell transplantation have been useful in globoid cell leukodystrophy (Krabbe disease) and in some mucopolysaccharidoses. Anti-inflammatory agents also show promise as adjuvant therapy. High doses of replacement therapy with native or modified enzyme show renewed promise for correction of CNS cells. Alternatively, small molecules can enter the brain relatively easily and promote reduction of accumulated substrate or function as pharmacological chaperones to enhance the level of the deficient enzyme. Gene therapy is still being developed and tested in patients. It is therefore likely that, thanks to a better understanding of disease mechanism, a variety of therapeutic approaches, used alone or in combination, will be useful to treat the devastating neurological complications of LSDs.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.