Drug intelligence / Profile preview

SosCas12f

Development stage
Preclinical
Lead developer
Virginia Commonwealth University
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
Administration
Intracerebral, Intravenous
01

Overview

SosCas12f is a hypercompact CRISPR-based gene editing system consisting of an engineered *Oryza sativa* Cas12f (enOsCas12f) nuclease fused to the non-specific double-stranded DNA-binding protein Sso7d. Developed by researchers at Virginia Commonwealth University and the Texas Biomedical Research Institute, this system is designed to overcome the low editing efficiency typically associated with compact Type V-F CRISPR effectors at endogenous genomic targets. The small size of the SosCas12f construct allows for "all-in-one" packaging into a single adeno-associated virus (AAV) vector, such as AAV-PhP.eB, along with multiplex single-guide RNAs (sgRNAs). Preclinical studies have demonstrated that SosCas12f significantly improves editing efficiency in vitro and in vivo, targeting clinically relevant loci such as TTR and VEGFA in human iPSC-derived brain organoids and mouse models. It has also been evaluated in SIV reporter assays, suggesting potential applications in treating viral infections like HIV.

Other names
Sso7d-enhanced hypercompact enOsCas12fSso-7d-enhanced hypercompact enOsCas12fSso 7d-enhanced hypercompact enOsCas12fSso7d-enOsCas12fSso-7d-enOsCas12fSso 7d-enOsCas12f
02

Targets

DNA

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