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Published in 2017 at "Gene Therapy"
DOI: 10.1038/gt.2017.21
Abstract: Ex vivo gene transfer to the graft before transplantation is an attractive option for circumventing systemic side effects of chronic antirejection therapy. Gene delivery of the immunomodulatory protein cytotoxic T-lymphocyte-associated protein 4–immunoglobulin (CTLA4-Ig) prevented chronic…
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Keywords:
non human;
kidney non;
mediated engineering;
human primates ... See more keywords
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Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2022.162
Abstract: The mucopolysaccharidoses (MPS) are a group of recessively inherited conditions caused by deficiency of lysosomal enzymes essential to the catabolism of glycosaminoglycans. MPS I is caused by deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA), while…
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Keywords:
mps mps;
gene transfer;
aav9 mediated;
gene ... See more keywords