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Gene Therapy Development Platforms

Gene therapy refers to the replacement or supplementation of defective genes in patients with functional genes to treat diseases. Our company offers comprehensive gene therapy development services and technical support covering every stage of rare disease therapy research, designed to help our clients address possible barriers and challenges in the development of gene therapies for rare diseases.

Gene Engineering for Gene Therapy

Gene therapies usually use gene engineering technology to introduce exogenous normal genes into target cells to correct or compensate for diseases caused by defective and abnormal genes to achieve therapeutic purposes. Gene engineering, also known as genetic engineering, genetic modification, or genetic manipulation, is a technology that uses biotechnology to directly manipulate the genome of an organism to change the genetic material of cells. Gene engineering technologies commonly used in gene therapy include CRISPR/Cas9, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALEN), and meganucleases.

CRISPR-Cas9 ZFNs TALENs Meganucleases
Recognized Length 22 bp + PAM sequence 9-18 bp 30-40 bp ~14-40bp
Recognized Mechanism DNA-RNA interaction via Watson-Crick base pairing DNA-protein interaction DNA-protein interaction Protein-DNA
Advantages Simple and convenient to build and low cost. High specificity, avoids immune response. Small off-target effects, easy to construct and use, and low price. Low toxicity in mammalian cells.
Disadvantages PAM recognition sequence is limited and easy to miss the target. Easily off-target, leading to cell death or additional mutations. A long process of assembling TALEN encoding plasmids and a large workload. The possibility of imprecisely binding to highly similar targets at different locations in the genome.

The rapid development of gene engineering technology has improved the efficiency and accuracy of gene editing. It can not only be used for the study of expression regulation and gene function, the construction of cell and animal models, the screening of oncogenes and drug targets, but also for gene therapy. Since 2006, genetic engineering techniques have been extensively developed for gene therapy.

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Fig.1 Application of gene engineering technology in gene therapy. (Danaeifar, Mohsen, 2023)

Gene engineering technology provides the gene editing tools and genetic materials required for the development of gene therapy, and therefore also provides tremendous support for gene therapy research and development for rare diseases. There have been some related cases for the treatment of rare diseases, such as:

  • Duchenne muscular dystrophy
  • Cystic fibrosis
  • Sickle cell anemia
  • Down syndrome
  • Fanconi anemia
  • Hemophilia

Our Services

Since about 80% of rare diseases are caused by genetic mutations, gene therapy can be used to the greatest extent in the research of rare diseases. Our company's gene therapy development platforms combined with our unique gene therapy solutions provide you with comprehensive gene editing tools and materials needed for gene therapy, we can provide you with customized services to support your gene therapy research and development for rare diseases. Our services include but are not limited to:

Our Advantages

  • Cutting-edge technology and equipment
  • Professional technical support
  • Stringent quality control and testing
  • Careful design and transparent operation process
  • Innovative and flexible solutions
  • Reliable and trusted partner

Project Workflow

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Our company is committed to providing gene 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.


  • Danaeifar, Mohsen. "Recent advances in gene therapy: genetic bullets to the root of the problem." Clinical and Experimental Medicine 23.4 (2023): 1107-1121.

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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