New genome editing technologies in the treatment of X-linked adrenoleukodystrophy
X-linked adrenoleukodystrophy is a severe progressive neurological disease that is predominantly found in male patients and caused by mutations in the X-linked ABCD1 gene encoding peroxisome transport protein. The disease is clinically characterized by two main phenotypes: the most severe infant cer...
| Main Authors: | , , |
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| Format: | Article |
| Language: | Russian |
| Published: |
Ltd. “The National Academy of Pediatric Science and Innovation”
2020-05-01
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| Series: | Rossijskij Vestnik Perinatologii i Pediatrii |
| Subjects: | |
| Online Access: | https://www.ped-perinatology.ru/jour/article/view/1117 |