What is CRISPR/cas9?

CRISPR/Cas9 is an easy-to-use and highly specific gene editing tool that has revolutionized molecular biology, providing an easy and relatively inexpensive way to create precise DNA edits. CRISPR/Cas9-based engineering can help accelerate drug discovery, reduce high attrition rates in drug development, and facilitate the development of cell and gene therapies. For example, when viral vectors such as adeno-associated virus are delivered to diseased tissues, direct genome editing can be effectively achieved in vivo to treat different ophthalmic diseases. Today, CRISPR/Cas9 has been actively explored as a strategy for the treatment of inherited retinal diseases and is widely used in translational research and small molecule drug development due to its robustness, precision, and ease of use.

Fig. 1. Characteristics and mechanism of several CRISPR/Cas9 gene editing tools.(Pulman J, et al., 2022)

Service Overview

At Ace Therapeutics, with the help of the CRISPR/Cas9 platform, our scientists can edit one or more genes to create cell line and animal (mice, rats, zebrafish, etc.) human eye disease models to facilitate small molecule drug discovery in ophthalmology and verification. Our CRISPR/Cas9 platform supports a wide range of genetic manipulations, including knockout, knockin, specific insertion or mutation, and control of transcription. These capabilities, combined with the physiological relevance of cell- or animal-based models, create nearly limitless possibilities for breakthrough ophthalmic drug discovery. Our implementation is primarily deployed at the cellular and organismal level through target discovery/validation (most practical in cells) and direct screening in gene-edited animals.

Based on our CRISPR/Cas9 editing platform, we can provide customers with a wide range of CRISPR/Cas9 editing services, including but not limited to.

• Genome-wide screening for target identification and validation.
• CRISPR Knockout Libraries for Mouse, Zebrafish, and Human.
• Provide cell lines with RISPR-mediated gene knockout, mutation knockout.
• Customized in vivo animal models based on genes of interest.
• CRISPR/Cas9-based gene knockout in mouse, rat.
• CRISPR/Cas9-based gene knockout in zebrafish.
• Gene therapy.

CRISPR/Cas9 and Target Screening & Validation

Advantages of Ace Therapeutics' CRISPR/Cas9-Based Target Screening & Validation

• Provide a hybrid array for high-throughput screening.
• CRISPR technology can be applied to any species, especially mice, rats, and zebrafish.
• Efficient and flexible, it can edit multiple genes at the same time.
• The CRISPR lentiviral system can infect both dividing and non-dividing cells.

Ace Therapeutics is committed to providing a full range of technical support for your Ophthalmology research. We are pleased to provide you with efficient and high-quality target identification and verification services using our advanced CRISPR/Cas9 platform. If you are interested in our services or have any special needs, please feel free to contact us. We look forward to working with you in the future.

Reference

1. Pulman J, Sahel JA, Dalkara D. New Editing Tools for Gene Therapy in Inherited Retinal Dystrophies. CRISPR J. 2022 ;5(3):377-388.

• RNAi-Based Target Screening
• Computer-Aided Target Identification and Verification
• Gene Expression Profiling-Based Screening
• Genetic Engineering-Based Target Screening
• Label-Free Target ID
• Mechanism of Action (MOA) Study
• Target Functional Annotation and ID
• Target Structure Determination

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