CRISPR/Cas9 Gene Editing and Ocular Disease

Eye diseases with different clinical symptoms are a highly heterogeneous set of phenotypes caused by a range of genetic variants and environmental factors. The eye is an ideal system for evaluating and validating novel gene therapies due to its anatomical location, structure, and immune privileges. Genome editing enables researchers to understand the biology behind disease mechanisms and treat a variety of health conditions, including ocular pathologies. CRISPR/Cas9-based genome editing facilitates efficient and specific genetic modification in nucleic acid sequences, resulting in permanent changes at the genome level. This approach provides a powerful, rapid and efficient means for gene therapy development for ocular diseases. It has been used to correct a large number of mutations associated with inherited retinal diseases and in some rodents with retinal diseases, which exhibits excellent therapeutic properties in vivo.

Fig. 1. Disease pathogenesis and CRISPR/Cas9 based treatment of the MYOC associated glaucoma. (Sundaresan Y, et al., 2023)

Service Overview

CRISPR/Cas, a powerful tool for studying retinal diseases and developing new treatments, offers an easy and relatively inexpensive way to create precise DNA edits. Ace Therapeutics trained scientists have developed a complete CRISPR/Cas platform to support the research projects of customers worldwide. Whether it is CRISPR/Cas-based editing or customized services, we can meet your needs.

Our scientists use genome-wide screening and CRISPR/Cas9-based gene knockout technology to study eye disease mechanisms in different species (mouse, rats, and zebrafish) and to develop novel treatments. Our CRISPR/Cas9 platform supports a wide range of genetic manipulations, including knockout, knockin, specific insertion or mutation, and transcriptional control. These capabilities, combined with the physiological relevance of cell- or animal-based models, open up endless possibilities for breakthrough ophthalmic therapeutic discovery.

Ace Therapeutics is proud to offer a comprehensive collection of CRISPR services, including the development of custom CRISPR vectors in multiple delivery formats (i.e. plasmid, viral, RNA) to efficiently deliver your CRISPR components into specific retinal cells. In addition, we focus on constructing high-quality CRISPR libraries for gene knockout, gene activation, gene suppression, etc. CRISPR screening. Our uniquely designed and well-validated genome-wide double gRNA knockout library is a powerful tool for functional screening of human and mouse genes.

Explore Our Capabilities

We establishs CRISPR/Cas9 platform from different species supports the development of drugs and new treatments for ophthalmic diseases. Our highly trained scientists can provide customized advice and services for your various gene editing functions, including but not limited to:

• Custom in vivo animal models of ocular disease based on genes of interest.
• CRISPR/Cas9-based gene knockout in mouse and rats.
• CRISPR/Cas9-based gene knockout in zebrafish.
• CRISPR-mediated gene knockout, mutant knockout cell lines.
• CRISPR knockout libraries for mouse, zebrafish, and human.
• Synthetic CRISPR sgRNA.
• CRISPR/Cas plasmid construction.
• CRISPR/Cas9-based target identification and validation.

RISPR/Cas9 is a precise gene editing technology that has emerged as a novel therapeutic tool for treating eye diseases. Ace Therapeutics has extensive experience in gene/genome editing, and we can provide systematic CRISPR solutions for global customers in the development of eye disease products. If you are interested in our services or need more detailed information, please feel free to contact us. Our experienced scientists are ready to help you!

Reference

1. Sundaresan Y, Yacoub S, Kodati B, et al. Therapeutic applications of CRISPR/Cas9 gene editing technology for the treatment of ocular diseases. FEBS J. 2023 Mar 6.

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