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MicroLesterol

Development stage: Preclinical
Lead developer: MicroCrispr
Modality: CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies

Overview

MicroLesterol is an experimental *in vivo* CRISPR-based gene therapy candidate being developed by MicroCrispr for the treatment of familial hypercholesterolemia. The therapy is designed to utilize CRISPR-mediated gene editing to target and permanently silence genes involved in cholesterol regulation, such as Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) or other LDL-regulating genes. By disrupting these targets in the liver, MicroLesterol aims to achieve a durable reduction in low-density lipoprotein (LDL) cholesterol levels. The program is currently in the preclinical stage of development.

Targets

PCSK9 (Proprotein convertase subtilisin/kexin type 9)

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