Molecular techniques for therapeutic and diagnostic applications in Mucopolysaccharidosis IIIB and Gaucher disease
There is an unmet need to develop and test treatments for rare lysosomal disease (LD). Most LDs are present in childhood and do not currently have approved therapies. Rare diseases individually are uncommon but taken together account for a population prevalence of 3.5-5.9% worldwide. Due to their ra...
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| Format: | Others |
| Language: | English en |
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2020
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| Online Access: | http://hdl.handle.net/1828/12493 |