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Filtering by: PPMO Academic Affiliation College of Veterinary Medicine

In Vivo Delivery of Morpholino Oligos by Cell-Penetrating Peptides

Creator:

Moulton, Hong M.

Abstract:

Morpholino oligos (Morpholinos) are widely used tools for knocking down gene expression and are currently in a clinical trial for treatment of Duchene muscular dystrophy. A Morpholino analog has been in a clinical trial as a potential anti-bioterrorism agent for inhibiting replication of deadly Marburg viral infection. The cellular uptake...

Resource Type:

Article

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-Penetrating Peptides Running title: In Vivo Delivery of PPMO Hong M. Moulton Biomedical Sciences
Long-Term Rescue of Dystrophin Expression and Improvement in Muscle Pathology and Function in Dystrophic Mdx Mice by Peptide-Conjugated Morpholino

Creator:

Wu, Bo, Lu, Peijuan, Cloer, Caryn, Shaban, Mona, Grewal, Snimar, Milazi, Stephanie, Shah, Sapana N., Moulton, Hong M., and Lu, Qi Long

Abstract:

Exon skipping is capable of correcting frame-shift and nonsense mutations of Duchenne Muscular Dystrophy (DMD). Phase II clinical trials in UK and Netherlands have reported induction of dystrophin expression in muscles of DMD patients by systemic administrations of both phosphorodiamidate morpholino oligomers (PMO) and 2’O methyl phosphorothioate. Peptide-conjugated PMO (PPMO)...

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Article

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-conjugated PMO (PPMO) offers significantly higher efficiency than PMO with the ability to induce near