Title : mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5.

Pub. Date : 2019 Dec 13

PMID : 31828178






3 Functional Relationships(s)
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Protein Name
Organism
1 Lack of CYP7B1 leads to an accumulation of its oxysterol substrates, in particular 25-hydroxycholesterol and 27-hydroxycholesterol that are able to cross the blood-brain barrier and have neurotoxic properties. arthrofactin cytochrome P450 family 7 subfamily B member 1 Homo sapiens
2 A single-dose injection of either mouse or human CYP7B1 mRNA led to a pronounced degradation of oxysterols in liver and serum within 2 days of treatment. arthrofactin cytochrome P450 family 7 subfamily B member 1 Homo sapiens
3 Pharmacokinetics indicate a single injection of human CYP7B1 mRNA to be effective in reducing oxysterols for at least 5 days. arthrofactin cytochrome P450 family 7 subfamily B member 1 Homo sapiens