Possible strategies to cross the blood-brain barrier
The mucopolysaccharidoses (MPS) are a heterogeneous group of in-born metabolic conditions caused by genetic defects that result in the absence or severe deficiency of one of the lysosomal hydrolases responsible for the degradation of glycosaminoglycans (GAGs). Such enzyme deficiency causes accumulat...
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| Published in | Italian journal of pediatrics Vol. 44; no. Suppl 2; p. 131 |
|---|---|
| Main Authors | , |
| Format | Journal Article |
| Language | English |
| Published |
England
BioMed Central
16.11.2018
BMC |
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| Abstract | The mucopolysaccharidoses (MPS) are a heterogeneous group of in-born metabolic conditions caused by genetic defects that result in the absence or severe deficiency of one of the lysosomal hydrolases responsible for the degradation of glycosaminoglycans (GAGs). Such enzyme deficiency causes accumulation of GAGs that begins in infancy and progressively worsens, often affecting several organs including the central nervous system (CNS) inducing mental retardation, progressive neurodegeneration, and premature death. Over the last years, enormous progress has been made in the treatment of many MPS types, and available treatments are efficacious for many of them. Nevertheless, treatment of MPS with CNS involvement is limited mostly because of delivery impediments related to the presence of the blood-brain barrier (BBB). This chapter presents an overview of the BBB and of the different strategies that have been developed to overcome the problem of drug transport at the BBB, assuring efficient delivery of therapeutic agents to the brain. |
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| AbstractList | The mucopolysaccharidoses (MPS) are a heterogeneous group of in-born metabolic conditions caused by genetic defects that result in the absence or severe deficiency of one of the lysosomal hydrolases responsible for the degradation of glycosaminoglycans (GAGs). Such enzyme deficiency causes accumulation of GAGs that begins in infancy and progressively worsens, often affecting several organs including the central nervous system (CNS) inducing mental retardation, progressive neurodegeneration, and premature death. Over the last years, enormous progress has been made in the treatment of many MPS types, and available treatments are efficacious for many of them. Nevertheless, treatment of MPS with CNS involvement is limited mostly because of delivery impediments related to the presence of the blood–brain barrier (BBB). This chapter presents an overview of the BBB and of the different strategies that have been developed to overcome the problem of drug transport at the BBB, assuring efficient delivery of therapeutic agents to the brain. Abstract The mucopolysaccharidoses (MPS) are a heterogeneous group of in-born metabolic conditions caused by genetic defects that result in the absence or severe deficiency of one of the lysosomal hydrolases responsible for the degradation of glycosaminoglycans (GAGs). Such enzyme deficiency causes accumulation of GAGs that begins in infancy and progressively worsens, often affecting several organs including the central nervous system (CNS) inducing mental retardation, progressive neurodegeneration, and premature death. Over the last years, enormous progress has been made in the treatment of many MPS types, and available treatments are efficacious for many of them. Nevertheless, treatment of MPS with CNS involvement is limited mostly because of delivery impediments related to the presence of the blood–brain barrier (BBB). This chapter presents an overview of the BBB and of the different strategies that have been developed to overcome the problem of drug transport at the BBB, assuring efficient delivery of therapeutic agents to the brain. |
| ArticleNumber | 131 |
| Author | Scarpa, Maurizio Bellettato, Cinzia M |
| Author_xml | – sequence: 1 givenname: Cinzia M surname: Bellettato fullname: Bellettato, Cinzia M organization: European Reference Network for Hereditary Metabolic Diseases, MetabERN, Wiesbaden, Germany – sequence: 2 givenname: Maurizio surname: Scarpa fullname: Scarpa, Maurizio email: maurizio.scarpa@metab.ern-net.eu, maurizio.scarpa@metab.ern-net.eu, maurizio.scarpa@metab.ern-net.eu, maurizio.scarpa@metab.ern-net.eu organization: Department for the Woman and Child Health, University of Padova, Padova, Italy. maurizio.scarpa@metab.ern-net.eu |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/30442184$$D View this record in MEDLINE/PubMed |
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| Keywords | Drug delivery Mucopolysaccharidoses Central nervous system Blood–brain barrier |
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| SubjectTerms | Blood-Brain Barrier Central nervous system Central Nervous System Diseases - etiology Central Nervous System Diseases - therapy Disease Drug delivery Drug delivery systems Endothelium Enzyme Replacement Therapy - methods Enzymes Gene therapy Glycosaminoglycans Humans Intellectual disabilities Metabolism Molecular weight Mucopolysaccharidoses Mucopolysaccharidoses - complications Mucopolysaccharidoses - therapy Mucopolysaccharidosis Nanoparticles Nervous system Neurodegeneration Review |
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| Title | Possible strategies to cross the blood-brain barrier |
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