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Your search keyword '"Cholesteryl ester storage disease"' showing total 1 results
Start Over Descriptor "Cholesteryl ester storage disease" Publication Type Magazines
1 results on '"Cholesteryl ester storage disease"'

1. Patent Issued for Methods and compositions for treatment of cholesteryl ester storage disease (USPTO 12060558).

Subjects
PEPTIDE nucleic acids, ESTERS, NON-coding RNA
Abstract

The article focuses on a newly issued patent for methods and compositions designed to treat Cholesteryl Ester Storage Disease (CESD). Topics include the use of antisense oligomers to modulate the expression of lysosomal acid lipase (LAL) protein, the impact of LAL deficiency on CESD, and the approach of targeting skippable-exon-containing pre-mRNA to address this rare chronic liver disease.

Published
2024

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