Lysosomal storage disease: Gene therapy on both sides of the blood–brain barrier
| Autor: | Perry B. Hackett, Elena L. Aronovich |
|---|---|
| Rok vydání: | 2015 |
| Předmět: |
Nervous system
Mucopolysaccharidosis I Mucopolysaccharidosis Genetic enhancement medicine.medical_treatment Endocrinology Diabetes and Metabolism Genetic Vectors Hematopoietic stem cell transplantation Biology Blood–brain barrier Bioinformatics Biochemistry Article Mucopolysaccharidosis III 03 medical and health sciences 0302 clinical medicine Endocrinology medicine Lysosomal storage disease Genetics Animals Humans Molecular Biology 030304 developmental biology 0303 health sciences Genetic Therapy Enzyme replacement therapy medicine.disease 3. Good health Lysosomal Storage Diseases Disease Models Animal medicine.anatomical_structure Blood-Brain Barrier Immunology Lysosomes 030217 neurology & neurosurgery |
| Zdroj: | Molecular Genetics and Metabolism. 114(2):83-93 |
| ISSN: | 1096-7192 |
| DOI: | 10.1016/j.ymgme.2014.09.011 |
| Popis: | Most lysosomal storage disorders affect the nervous system as well as other tissues and organs of the body. Previously, the complexities of these diseases, particularly in treating neurologic abnormalities, were too great to surmount. However, based on recent developments there are realistic expectations that effective therapies are coming soon. Gene therapy offers the possibility of affordable, comprehensive treatment associated with these diseases currently not provided by standards of care. With a focus on correction of neurologic disease by systemic gene therapy of mucopolysaccharidoses types I and IIIA, we review some of the major recent advances in viral and non-viral vectors, methods of their delivery and strategies leading to correction of both the nervous and somatic tissues as well as evaluation of functional correction of neurologic manifestations in animal models. We discuss two questions: what systemic gene therapy strategies work best for correction of both somatic and neurologic abnormalities in a lysosomal storage disorder and is there evidence that targeting peripheral tissues (e.g., in the liver) has a future for ameliorating neurologic disease in patients? |
| Databáze: | OpenAIRE |
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