Gene Therapy Delivery Platforms
Download PDF
Gene therapy is developing into precise medicines that can manipulate specific genes in the treatment of serious diseases and vaccines development. To effectively function in vivo, reliable delivery systems are very important to the nucleic acids for protecting them from degradation and allowing effective cellular uptake and release. Based on profound scientific research accumulation, strong R&D strength and continuous technical support, CD Bioparticles could provide customers with one-stop customization service as:
Nucleic acid services
- Design
- Synthesis
- Modifications
Delivery systems development
- Polymer-based nanoparticles
- Liposomes
- Cell-based nanoparticles
- Viral Vectors
Characterization
- Physical characterization
- Cell characterization
- Animal studies
Nucleic acid services platform
Design
According to the name of the disease or the gene, search the database and literature for the sequence of the nucleic acid, and select the proper CDS for synthesis.
Synthesis
- In Vitro Transcription (IVT)
- Chemical synthesis for siRNA, mRNA, DNA, plasmid, ASO, sgRNA, shRNA
- The corresponding QC testing services
Modifications
5`-Terminal
Cap0/ Cap1/Cap2,Fluorescent Cap,IRES/MS2,UTR modification…
3`-Terminal
Poly(A) synthesis, UTRmodification…
Code Region:
m1ψ, m5C, 2'-OMe,Pseudo-Uridine, LNA, N6methyladenine, 5methylcytidune…
* Other modifications if needed.
Delivery system development platforms
Polymer-based nanoparticles
Due to the wide range of sources and low immunogenicity, cationic polymers, such as PEI, dendrimers, chitosan, gelatin are widely used nanoscales delivery systems for gene therapy.
Liposomes
With the similar structure of cell membrane, liposomes are considered to be a safer and more effective delivery system. Its flexibility and robustness in lipid structure, composition, ratio and preparation methods make liposomes an important artificial carrier for gene therapy. Targeting ligands, such as peptides, antibodies have been successfully applied in liposomes to achieve active targeting delivery to specified area. Furthermore, encapsulating fluorescent dyes in liposomes is very beneficial for later tracking and imaging studies.
Lipid nanoparticles (LNPs)
- High nucleic acid loading
- Stable in vivo
- Low toxicity
LNPs with target groups
- Active targeting delivery
- Tissue/cell selectivity
- High bioavailability
Commercial LNPs
- SM-102/MC3/ALC-0315 for research only
- FDA approved
- Ideal delivery model
Fluorescent liposomes
- Biological tracer
- Visual distinction
Delivery system development platforms
Cell-based nanoparticles
Exosomes are nanovesicles derived from cell membrane with natural targeting ability. CD Bioparticles provides various exosome production custom services for gene therapy research.
Viral vectors
Viral vectors are a tool commonly used in molecular biology to bring genetic material into cells. The principle is to use the molecular mechanism of viruses to transmit their genomes into other cells for infection. Its high effectiveness and tropism make viral vectors one of the most commonly used carriers for gene therapy. CD Bioparticles could provide a wild range of viral vectors for basic research and preclinical study.
Adenoviral vectors (Ads)
- Non-integrating
- Large packaging capacity
- Broad tropism
- Transduce (non-)dividing cells
- High level expression
Adeno-associated viral vectors (AAVs)
- Non-integrating
- Tissue tropisms
- Various mutants
- Low immunogenicity
- High level and long-term
Lentiviral vectors (LVs)
- S Integrating
- Stable expression
- Long-term expression
Retroviral vectors (RVs)
- Integrating
- Transduce dividing cells
Characterization Platform
Physical characterization
- DNA sequencing
- Off-target detection
- Size & PDI
- Morphology
- Zeta potential
- Lipid assay (liposomes only)
- Encapsulation efficiency & Drug loading
Cell characterization
- Cell culture
- Cell targeting
- Gene & protein expression
- Titer definition
- Functional evaluation
- Pseudovirus & plasmids creation (viral vectors only)
- Viral vectors purification (viral vectors only)
Animal studies
- Animal model establishment
- Immunizations
- Safety evaluation
- Effectiveness evaluation
