In-depth investigation of the mechanisms of specific diseases and identification of key gene targets associated with the diseases. Through a comprehensive analysis of the pathophysiological process of the disease, we can determine which gene abnormalities are the key factors leading to the disease, and provide a basis for the subsequent development of therapeutic strategies.

· Standard gene synthesis

· SegmentGene gene synthesis

· siRNA/miRNA/ASO synthesis service

· Eukaryotic/prokaryotic transcriptome sequencing

· CRISOPR-sgRNA library construction

· Stabilized cell line construction

Genes with therapeutic potential are carefully selected based on identified targets. It can be a normally functioning gene to replace a defective gene or a gene that can modulate a disease-related biological pathway to correct the abnormality in the disease state.

·Adeno-associated virus packaging

The design of suitable gene vectors ensures that therapeutic genes can be delivered efficiently and safely into the patient's cells. Common vectors include viral vectors (e.g., adeno-associated viruses, lentiviruses, etc.) and non-viral vectors (e.g., lipid nanoparticles, etc.). Optimizing the structure and properties of vectors improves their targeting and reduces immunogenicity and potential toxicity.

· Gene Therapy NGS Safety Evaluation

· Adeno-associated virus packaging

Tests are performed on in vitro cultured cell models to validate the efficacy and safety of therapeutic genes and vectors. Observe the expression of the genes and their effects on cell function and detect any adverse side effects.

· Gene Therapy NGS Safety Evaluation