Drug intelligence / Profile preview

CFH-specific CRISPR-CAS9

Development stage
Preclinical
Lead developer
Loyola University Chicago
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies
01

Overview

CFH-specific CRISPR-CAS9 is a gene-editing tool designed to genetically deplete tumor cell-derived complement factor H (CFH). Developed by researchers at Loyola University Chicago, this CRISPR-Cas9 system targets the CFH gene to suppress glioblastoma (GBM) development. In preclinical models, transduction of human glioma cells with lentiviral particles encoding CFH-specific CRISPR-Cas9 successfully depleted CFH, leading to reduced in vitro cell proliferation and significantly increased survival in murine models of glioblastoma.

02

Targets

CFH (Complement factor H)

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