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Meganuclease-based Therapy Development Platform

Meganucleases (MNs), also called homing endonucleases (HEs), can cleave double-stranded DNA at specific recognition sites. Our company's meganuclease-based therapy development platform can help customers develop product candidates aimed at addressing previously intractable indications using novel therapeutic modalities such as gene editing. we have the capabilities to provide professional services to ensure that we can quickly respond to the changing needs of your rare disease research projects.

Introduction to Meganuclease

Meganuclease is an endodeoxyribonuclease with a large recognition site profile. The ability of this highly specific enzyme to target individual DNA breaks in complex genomes and achieve gene modifications while avoiding random off-targeting makes it an ideal starting material for therapeutic-grade genome editing technologies. Since most genetic diseases are caused by the absence of important proteins, this technology platform is well-suited for rare disease drug development.

Meganuclease-based Therapy Development Platform-1

Fig.1 Schematic of Meganucleases (MN) technology. (Zaman, Q. U., et al., 2019)

Meganuclease-based Therapy for Rare Diseases

Meganuclease is highly advantageous for use in the development of disease therapies due to its high specificity. These enzymes can be used as scaffolds to develop custom gene-targeting tools for cell line development or gene therapy for rare diseases. For example, a gene-editing strategy based on meganuclease has been developed for the treatment of Duchene muscular dystrophy, which restores the open reading frame of mutated muscular dystrophy genes.

Our Services

To meet the broad scientific needs of our customers, our company provides customers with pre-designed meganucleases for genome editing, as well as meganuclease modification services and the re-engineering of a large number of I-CreI derivatives into custom enzymes for specific target DNA sequences of interest.

At the same time, our company uses meganuclease to achieve gene editing and treatments related to rare diseases, including gene correction approaches, targeted mutagenesis of human genes, insertion of therapeutic genes into a safe harbor, and virus clipping, thereby accelerating your research on gene therapy for rare diseases. Our meganuclease-based therapy development services include but are not limited to:

  • Meganuclease Design

To ensure the specificity of meganuclease and avoid the occurrence of toxicity, we provide customers with custom-designed meganucleases, including but not limited to:

  • Designing meganucleases that target genes involved in severe combined immunodeficiency (SCID) and other genetic diseases that can be treated with cellular therapies.
  • Designing mitochondria-targeted meganucleases to develop gene therapies for rare mitochondrial diseases.
  • Meganuclease Specificity Improvement

Designing I-CreI dimerization interfaces to produce the second generation of functionally tailored meganucleases that meet high standards of specificity and non-toxicity, and providing data to monitor meganuclease specificity.

  • Meganuclease Delivery

Help customers design lentiviral vectors for the transduction of engineered meganucleases, such as a method based on Vpr fusions.

In the area of gene therapy for rare diseases, our company helps customers with targeted gene insertion or targeted correction to replace classical gene transfer leading to random integration into the genome, with our targeting strategies including:

  • Targeted strategies based on recombinases
  • Targeted strategies based on homologous recombination
  • Targeted strategies based on transposons
  • Targeted strategies based on non-homologous end-joining

Project Workflow

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Our company is committed to providing meganuclease-based therapy development services to accelerate the development of safer and more effective gene therapies for rare diseases. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.

Reference

  • Zaman, Q. U., et al. "Genome editing opens a new era of genetic improvement in polyploid crops." The Crop Journal 7.2 (2019): 141-150.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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