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Publication
Development of a U1 snRNA-adapted gene therapeutic strategy to correct 5’ splicing defects in lysosomal storage disorders
| Name: | Description: | Size: | Format: | |
|---|---|---|---|---|
| 1.3 MB | Adobe PDF |
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Keywords
Doenças Genéticas U1snRNA therapy Lysosomal Storage Disorders MPS III C ML III alpha/beta MPS I