CRISPR/Cas9 technology in the modeling of and treatment of mucopolysaccharidosis
Mucopolysaccharidosis (MPS) syndromes are a group of heterogeneous genetic disorders in terms of genetic basis and clinical manifestations, ranging from mild to fatal forms. There are a number of applied or prospective treatment modalities for MPS, including bone marrow transplantation, enzyme repla...
| Published in: | Biochemistry and Biophysics Reports |
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| Main Authors: | , |
| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-09-01
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| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2405580824001353 |