Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy. [electronic resource]
Producer: 20180503Description: 130-143 p. digitalISSN:- 2159-3345
- Animals
- Animals, Newborn
- Apolipoproteins E -- chemical synthesis
- Biomarkers -- blood
- Blood-Brain Barrier -- chemistry
- Brain -- cytology
- Cell Survival -- drug effects
- Disease Models, Animal
- Exons
- Fibroblasts -- drug effects
- Hepatocytes -- drug effects
- Humans
- Kidney -- chemistry
- Mice
- Morpholinos -- chemistry
- Muscular Atrophy, Spinal -- drug therapy
- Nanoconjugates -- analysis
- Peptides -- chemical synthesis
- Phenotype
- Quadriceps Muscle -- chemistry
- Survival of Motor Neuron 2 Protein -- drug effects
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Publication Type: Journal Article
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