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Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities.
Gene Therapy for Mucopolysaccharidosis Type II-A Review of the Current Possibilities.
- Source :
-
International journal of molecular sciences [Int J Mol Sci] 2021 May 23; Vol. 22 (11). Date of Electronic Publication: 2021 May 23. - Publication Year :
- 2021
-
Abstract
- Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder based on a mutation in the IDS gene that encodes iduronate 2-sulphatase. As a result, there is an accumulation of glycosaminoglycans-heparan sulphate and dermatan sulphate-in almost all body tissues, which leads to their dysfunction. Currently, the primary treatment is enzyme replacement therapy, which improves the course of the disease by reducing somatic symptoms, including hepatomegaly and splenomegaly. The enzyme, however, does not cross the blood-brain barrier, and no improvement in the function of the central nervous system has been observed in patients with the severe form of the disease. An alternative method of treatment that solves typical problems of enzyme replacement therapy is gene therapy, i.e., delivery of the correct gene to target cells through an appropriate vector. Much progress has been made in applying gene therapy for MPS II, from cellular models to human clinical trials. In this article, we briefly present the history and basics of gene therapy and discuss the current state of knowledge about the methods of this therapy in mucopolysaccharidosis type II.
- Subjects :
- Adolescent
Animals
Blood-Brain Barrier
CRISPR-Cas Systems
Child
Child, Preschool
Clinical Trials as Topic
Dependovirus genetics
Disease Models, Animal
Drug Carriers
Electroporation
Enzyme Replacement Therapy methods
Gene Editing
Genetic Therapy
Genetic Vectors adverse effects
Genetic Vectors therapeutic use
Glycoproteins pharmacokinetics
Glycoproteins therapeutic use
Hematopoietic Stem Cell Transplantation
Humans
Immunoconjugates administration & dosage
Immunoconjugates therapeutic use
Infant
Injections, Intraventricular
Injections, Spinal
Lentivirus genetics
Mice
Mucopolysaccharidosis II genetics
Multicenter Studies as Topic
Recombinant Proteins administration & dosage
Recombinant Proteins therapeutic use
Retroviridae genetics
Transcription Activator-Like Effector Nucleases
Glycoproteins genetics
Mucopolysaccharidosis II therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1422-0067
- Volume :
- 22
- Issue :
- 11
- Database :
- MEDLINE
- Journal :
- International journal of molecular sciences
- Publication Type :
- Academic Journal
- Accession number :
- 34070997
- Full Text :
- https://doi.org/10.3390/ijms22115490