Gene therapy for rare haematological and neurometabolic paediatric diseases
Nowadays, gene therapy hast the potential to cure an increasingly greater number of monogenic inherited disorders with absent or limited treatment options, and radically change their natural history. Hematopoietic stem cells (HSCs) represent one of the preferred targets for gene therapy, as genetic...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-09-01
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| Series: | Global Pediatrics |
| Subjects: | |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2667009724000642 |