BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy
Oculopharyngeal muscular dystrophy (OPMD) is a rare autosomal dominant disease that results from an alanine expansion in the N-terminal domain of Poly-A Binding Protein Nuclear-1 (PABPN1). We have recently demonstrated that a two-vector gene therapy strategy significantly ameliorated the pathology i...
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| Main Authors: | , , , , , , , , , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2021-06-01T00:00:00Z.
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| Subjects: | |
| Online Access: | Connect to this object online. |
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