ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS?
Lysosomal storage disorders are an agglomeration of genetic disorders such as Fabry disease, Gaucher disease, Pompe disease, Krabbe’s disease and mucopolysaccharidosis that typically impairs the prime orangs of humans, including brain, heart, musculoskeletal system, spleen, eye, and lungs. Patients...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Gomal Medical College, D.I.Khan, Pakistan
2021-12-01
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| Series: | Gomal Journal of Medical Sciences |
| Subjects: | |
| Online Access: | http://gjms.com.pk/index.php/journal/article/view/999 |
| _version_ | 1798014875930198016 |
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| author | Taimoor Hassan Xu Huadong |
| author_facet | Taimoor Hassan Xu Huadong |
| author_sort | Taimoor Hassan |
| collection | DOAJ |
| description | Lysosomal storage disorders are an agglomeration of genetic disorders such as Fabry disease, Gaucher disease, Pompe disease, Krabbe’s disease and mucopolysaccharidosis that typically impairs the prime orangs of humans, including brain, heart, musculoskeletal system, spleen, eye, and lungs. Patients with lysosomal storage disorders face mild to severe complications and even death. In order to address these health concerns, scientists are working by dint off, various therapies are introduced such as gene therapy, typical oral medicines, organ/ cell transplantation etc. However, hematopoietic stem cell transplantation and enzyme replacement therapy came out as best stakeholders to treat aforementioned disorders. Nonetheless, according to suggested data, it is concluded that presently enzyme replacement therapies are somehow ineffective for many lysosomal storage disorders till today. But we believe that in near future, as more and more research will be progressed, the ultimate therapy to these disorders will be developed. |
| first_indexed | 2024-04-11T15:25:23Z |
| format | Article |
| id | doaj.art-18b34f6c777446a7bf8d81f94a15cc74 |
| institution | Directory Open Access Journal |
| issn | 1819-7973 1997-2067 |
| language | English |
| last_indexed | 2024-04-11T15:25:23Z |
| publishDate | 2021-12-01 |
| publisher | Gomal Medical College, D.I.Khan, Pakistan |
| record_format | Article |
| series | Gomal Journal of Medical Sciences |
| spelling | doaj.art-18b34f6c777446a7bf8d81f94a15cc742022-12-22T04:16:16ZengGomal Medical College, D.I.Khan, PakistanGomal Journal of Medical Sciences1819-79731997-20672021-12-01192737710.46903/gjms/19.02.999847ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS?Taimoor Hassan0Xu Huadong1School of Pharmacy & School of Medicine, Changzhou University, Jiangsu, ChinaSchool of Pharmacy & School of Medicine, Changzhou University, Jiangsu, ChinaLysosomal storage disorders are an agglomeration of genetic disorders such as Fabry disease, Gaucher disease, Pompe disease, Krabbe’s disease and mucopolysaccharidosis that typically impairs the prime orangs of humans, including brain, heart, musculoskeletal system, spleen, eye, and lungs. Patients with lysosomal storage disorders face mild to severe complications and even death. In order to address these health concerns, scientists are working by dint off, various therapies are introduced such as gene therapy, typical oral medicines, organ/ cell transplantation etc. However, hematopoietic stem cell transplantation and enzyme replacement therapy came out as best stakeholders to treat aforementioned disorders. Nonetheless, according to suggested data, it is concluded that presently enzyme replacement therapies are somehow ineffective for many lysosomal storage disorders till today. But we believe that in near future, as more and more research will be progressed, the ultimate therapy to these disorders will be developed.http://gjms.com.pk/index.php/journal/article/view/999drug delivery systemsenzyme replacement therapyfabry diseaselysosomal enzyme disorderslysosomal storage disordersgaucher diseasepompe disease. |
| spellingShingle | Taimoor Hassan Xu Huadong ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS? Gomal Journal of Medical Sciences drug delivery systems enzyme replacement therapy fabry disease lysosomal enzyme disorders lysosomal storage disorders gaucher disease pompe disease. |
| title | ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS? |
| title_full | ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS? |
| title_fullStr | ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS? |
| title_full_unstemmed | ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS? |
| title_short | ARE ENZYME REPLACEMENT THERAPIES EFFECTIVE AGAINST LYSOSOMAL STORAGE DISORDERS? |
| title_sort | are enzyme replacement therapies effective against lysosomal storage disorders |
| topic | drug delivery systems enzyme replacement therapy fabry disease lysosomal enzyme disorders lysosomal storage disorders gaucher disease pompe disease. |
| url | http://gjms.com.pk/index.php/journal/article/view/999 |
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