Lentiviral vectors for delivery of
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.
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| Format: | Patent |
| Sprache: | eng |
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| Zusammenfassung: | The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency. |
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