Compositions, systems and methods for reducing low density lipoproteins by targeting gene repression

By developing an epigenetic modified DNA targeting system containing multiple DNA targeting modules, the toxicity and high cost of existing methods for treating familial hypercholesterolemia are solved, and effective reduction of LDL levels and targeted regulation of multiple genes are achieved.

CN119731321APending Publication Date: 2025-03-28TUNE THERAPEUTICS INC
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Patent Information

Application Number
CN202380060137.3
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2023-06-09
Filing Date
2023-06-23
Publication Date
2025-03-28

AI Technical Summary

Technical Problem

The existing treatments for familial hypercholesterolemia have problems of toxicity and high cost, and it is difficult to effectively reduce low-density lipoprotein (LDL) levels.

Method used

An epigenetic modified DNA targeting system was developed, which contains multiple DNA targeting modules, each module containing a fusion protein targeting multiple genes, a fusion protein binding DNA binding domain and a transcriptional repressor domain, which is used to repress and regulate gene transcription of LDL.

Benefits of technology

Through this system, LDL levels can be effectively reduced without introducing gene disruption or DNA breakage, targeted regulation of multiple genes and enhanced the effectiveness and safety of treatment.

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Abstract

In some aspects, an epigenetically modified DNA targeting system, such as a CRISPR-Cas / guide RNA (gRNA) system, is provided for transcriptional repression of a gene to promote cell phenotypes that lead to reduction of low density lipoprotein (LDL). In some embodiments, the epigenetically modified DNA targeting system binds to or targets a target site that modulates at least one gene or a regulatory element thereof that modulates LDL. In some embodiments, the system is a multiplex system that binds to or targets target sites in at least two genes or regulatory elements thereof. Also provided herein are methods and uses associated with the provided epigenetically modified DNA targeting systems associated with the treatment of cardiovascular diseases and familial hypercholesterolemia.
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Citation Information

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