CRISPR/Cas9-Based Therapies Development Services for Liver Disease

Ace Therapeutics aims to provide the most satisfactory service to liver disease researchers worldwide. We have extensive research experience in the field of CRISPR/Cas9-based liver disease therapy development to escort your therapy development.

About 30 years ago, the first human clinical trial of gene therapy was successfully completed, which means that gene therapy can have obvious clinical benefits and is a potential way to treat diseases. Because some liver diseases are caused by mutations in a single gene, gene therapy is a powerful way to cure the disease. CRISPR/Cas9 is a versatile and efficient RNA-guided gene editing platform capable of modifying any gene of interest. Based on our expertise and technology platform, we provide an integrated service for the development of liver disease therapeutics based on CRISPR/Cas9 to meet the needs of different researchers.

What Can We Do?

CRISPR/Cas9-based gene therapy has made significant progress in the treatment of liver diseases. Nevertheless, the clinical translation of this approach still faces many challenges. Ace Therapeutics offers comprehensive development services for CRISPR/Cas9-based gene therapy for liver disease.

• We have different experimental cells and experimental animals to build liver disease models for different research needs.
• We have the perfect CRISPR/Cas9 technology to customize appropriate liver disease models.
• We have sophisticated platforms that can examine physiological and pathophysiological processes in different diseases to assess the efficacy of therapies.
• With extensive hands-on experience, our scientists adjust experimental protocols in real time to obtain optimal experimental results based on the specifics of the development process of gene therapies targeting liver diseases.

Given that alpha-1 antitrypsin deficiency is caused by mutations in the SERPINA1 gene which in turn leads to the accumulation of defective AAT proteins to abnormal levels in the liver, resulting in liver damage. We provide comprehensive research services for the development of CRISPR/Cas9-based gene therapies for the treatment of alpha-1 antitrypsin deficiency.

Given that hereditary tyrosinemia type 1 (HT-1) is the result of mutations in the fumarate acetylhydrolase (Fah) gene, we will treat HT-1 using Sleeping Beauty transposon-encoding mRNA, intravenous facial injection of AAV vectors expressing the wild-type allele, and injecting sgRNA, Cas9, and single-stranded DNA template hydrodynamically and other methods.

Based on the fact that CRISPR/Cas9 has also shown significant therapeutic effects in suppressing infection by human viral pathogens, we will suppress hepatitis virus infection by lentiviral transduction of the Cas9 gene for the treatment of hepatitis virus infection.

Ace Therapeutics has rich practical experience in the field of developing liver disease therapeutics based on CRISPR/Cas9 technology. Based on our professional knowledge and technology platform, we are willing to provide you with the most professional and appropriate scientific research services. If you would like to learn more about our services, please feel free to contact us.

Reference

1. Aravalli, R. N. Steer, C. J. Crispr/cas9 therapeutics for liver diseases. J Cell Biochem. 2018. 119(6): p. 4265-4278.

Our products and services are for research use only and can not be used for diagnostic or other purposes.