Applications and Research of Gene Therapy

Gene therapy refers to transfer a normal exogenous gene into a target cell to correct or compensate the diseases caused by gene defects and abnormal expression in achieving therapeutic purposes. Broadly speaking, gene therapy also includes measures and new technologies for treating certain diseases at the DNA level. Traditional gene therapy generally uses a harmless virus or other vectors to copy normal genes into cells to replace the disease-causing defect genes. The CRISPR technology corrects defective genes by excising damaged DNA and replacing it with the correct gene sequence directly. This method eliminates the risk of inserting a exogenous gene into the wrong position and activating the oncogene. The corrected gene is still regulated by the promoter of the gene itself, so the protein in the cell will maintain normal level. The development and maturity of CRISPR technology has opened up new ideas and new methods for gene therapy of a large variety of diseases.

Research Strategy

pgRNA/sgRNA design
and synthesis
  • Genomic or panel-wide gene retrieval and pgRNA/sgRNA design
sgRNA synthesis
and infection
  • Ready-to-use sgRNA synthesis (injected directly)
  • Syno® 2.0 gene synthesis and vector construction
  • Vector design of polytypic species
  • Lentivirus packaging and transfection
In vitro functional verification and analysis
  • SSA activity detection
  • In vitro cutting activity verification
  • Endogenous active cutting
In vitro experiment
  • Stable cell line construction
  • Animal model construction
  • Cell or animal level phenotype detection


  • One-stop Solution
  • Synbio Technologies provides a total solution from sgRNA design, sgRNA synthesis, lentiviral packaging to in vitro functional verification and in vivo validation.

  • Patented Technology Platform
  • Patented software for sgRNA design can provide efficient and accurate multi-species sequence design.

  • Ready-to-use sgRNA
  • Inject directly or transfect cells for gene editing, 20 μg can be delivered in at least 3 business days.

  • Animal Model Construction
  • Multi-pattern species including zebrafish, mice and rats are available.