In:
International Journal of Molecular Sciences, MDPI AG, Vol. 23, No. 9 ( 2022-04-27), p. 4854-
Abstract:
Mucopolysaccharidosis type II (Hunter Syndrome) is a rare, x-linked recessive, progressive, multi-system, lysosomal storage disease caused by the deficiency of iduronate-2-sulfatase (IDS), which leads to the pathological storage of glycosaminoglycans in nearly all cell types, tissues and organs. The condition is clinically heterogeneous, and most patients present with a progressive, multi-system disease in their early years. This article outlines the pathology of the disorder and current treatment strategies, including a detailed review of haematopoietic stem cell transplant outcomes for MPSII. We then discuss haematopoietic stem cell gene therapy and how this can be employed for treatment of the disorder. We consider how preclinical innovations, including novel brain-targeted techniques, can be incorporated into stem cell gene therapy approaches to mitigate the neuropathological consequences of the condition.
Type of Medium:
Online Resource
ISSN:
1422-0067
DOI:
10.3390/ijms23094854
Language:
English
Publisher:
MDPI AG
Publication Date:
2022
detail.hit.zdb_id:
2019364-6
SSG:
12
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