Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities
Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder based on a mutation in the <i>IDS</i> 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 lead...
| Main Authors: | , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
MDPI AG
2021-05-01
|
| Series: | International Journal of Molecular Sciences |
| Subjects: | |
| Online Access: | https://www.mdpi.com/1422-0067/22/11/5490 |
| _version_ | 1797533020560818176 |
|---|---|
| author | Paweł Zapolnik Antoni Pyrkosz |
| author_facet | Paweł Zapolnik Antoni Pyrkosz |
| author_sort | Paweł Zapolnik |
| collection | DOAJ |
| description | Mucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder based on a mutation in the <i>IDS</i> 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. |
| first_indexed | 2024-03-10T11:08:40Z |
| format | Article |
| id | doaj.art-c30dba310f2b4c50986473f73ec048ab |
| institution | Directory Open Access Journal |
| issn | 1661-6596 1422-0067 |
| language | English |
| last_indexed | 2024-03-10T11:08:40Z |
| publishDate | 2021-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | International Journal of Molecular Sciences |
| spelling | doaj.art-c30dba310f2b4c50986473f73ec048ab2023-11-21T20:59:36ZengMDPI AGInternational Journal of Molecular Sciences1661-65961422-00672021-05-012211549010.3390/ijms22115490Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current PossibilitiesPaweł Zapolnik0Antoni Pyrkosz1Students’ Scientific Association of Clinical Genetics, Department of Clinical Genetics, Medical College, University of Rzeszów, 35-959 Rzeszów, PolandDepartment of Clinical Genetics, Medical College, University of Rzeszów, 35-959 Rzeszów, PolandMucopolysaccharidosis type II (MPS II) is a lysosomal storage disorder based on a mutation in the <i>IDS</i> 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.https://www.mdpi.com/1422-0067/22/11/5490mucopolysaccharidosis IIHunter syndromeadeno-associated virusesgenetic therapygene editingreview |
| spellingShingle | Paweł Zapolnik Antoni Pyrkosz Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities International Journal of Molecular Sciences mucopolysaccharidosis II Hunter syndrome adeno-associated viruses genetic therapy gene editing review |
| title | Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities |
| title_full | Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities |
| title_fullStr | Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities |
| title_full_unstemmed | Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities |
| title_short | Gene Therapy for Mucopolysaccharidosis Type II—A Review of the Current Possibilities |
| title_sort | gene therapy for mucopolysaccharidosis type ii a review of the current possibilities |
| topic | mucopolysaccharidosis II Hunter syndrome adeno-associated viruses genetic therapy gene editing review |
| url | https://www.mdpi.com/1422-0067/22/11/5490 |
| work_keys_str_mv | AT pawełzapolnik genetherapyformucopolysaccharidosistypeiiareviewofthecurrentpossibilities AT antonipyrkosz genetherapyformucopolysaccharidosistypeiiareviewofthecurrentpossibilities |