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Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
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机译:用于治疗眼咽肌营养不良症的试剂及其用途
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摘要
The present disclosure relates to RNA interference (RNAi) reagents, such as short hairpin microRNA (shmiR) and short hairpin RNA (shRNA), for treatment of oculopharyngeal muscular dystrophy (OPMD), compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto. The present disclosure also relates to the use of the RNAi reagents in combination with PABPN1 replacement reagents, such as constructs which encode functional PABPN1 protein, for treatment of OPMD, compositions comprising same, and use thereof to treat individuals suffering from OPMD or which are predisposed thereto.
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