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Gene Therapy
Gene therapy uses viral vectors to deliver therapeutic genes into patient cells, correcting or compensating for genetic defects. Adeno-associated virus (AAV) is the leading platform for in vivo gene therapy due to its strong safety profile, low immunogenicity, and ability to drive long-term expression in non-dividing tissues such as the brain, muscle, liver, and retina. Its main limitation is a small cargo capacity (~4.7 kb), requiring compact transgenes or split-vector strategies.
Adenovirus (AdV) offers a much larger packaging capacity and high transduction efficiency, making it suitable for delivering large or complex payloads (e.g., CRISPR systems or therapeutic cDNAs). Because AdV does not integrate into the host genome, expression is typically transient—useful for short-term therapies like cancer gene therapy or vaccines—but its higher immunogenicity must be managed.
Lentivirus (LV) enables stable, long-term gene expression through genome integration and is widely used for ex vivo gene therapy, particularly in hematopoietic stem cells and CAR-T therapies. While this allows durable correction of genetic disorders, careful design is required to minimize insertional mutagenesis.
Together, AAV, adenovirus, and lentivirus form a complementary toolkit: AAV for safe, long-term in vivo delivery; adenovirus for high-capacity, transient expression; and lentivirus for stable genetic modification in regenerative medicine and immunotherapy.
SignaGen Laboratories provides a comprehensive portfolio of viral vectors—including AAV, adenovirus, and lentivirus—supporting gene therapy research with both pre-made products and custom vector design and production services.
In practice, these platforms serve distinct roles. Lentivirus underpins ex vivo therapies, where patient cells are modified and reinfused, as in CAR-T treatments. AAV dominates in vivo applications, enabling long-term gene expression in tissues such as the brain and retina for diseases like spinal muscular atrophy and inherited retinal disorders. Adenovirus, while less suited for permanent gene replacement, is valuable for cancer therapy and vaccines due to its strong immunogenicity and large payload capacity (~36 kb).
Together, these systems enable a broad range of gene therapy strategies, from stable cell engineering to durable gene replacement and immune-based treatments.
| Feature | Lentivirus (LV) | AAV | Adenovirus (AdV) |
| Strategy | Ex Vivo (Modified cells) | In Vivo (Direct injection) | Vaccines / Oncology |
| Integration | Yes (Permanent) | No (Episomal) | No (Transient) |
| Typical Target | Dividing cells (Blood/Stem) | Non-dividing (Muscle/Nerve) | Immune cells / Tumors |
| Safety Profile | Risk of insertional mutagenesis | Highest safety rating | High immunogenicity |
| Clinical Examples | CAR-T (Kymriah) | Zolgensma (SMA), Luxturna | COVID-19 Vaccines, Gendicine |
Viral Vector Production Options for Pre-clinical Gene Theraphy Applications
End-to-end viral vector production supporting construct development, plasmid preparation, viral packaging, purification, and quality control. Flexible service options enable efficient generation of high-quality vectors for in vitro, in vivo, and preclinical research applications
Custom adeno-associated virus (AAV) vectors designed for precise gene delivery and expression. Supports applications including transgene expression, RNAi-mediated knockdown, CRISPR genome editing, and tissue-specific gene modulation in preclinical and in vivo studies.
Packaging of customer-supplied AAV transfer plasmids into purified viral particles. Production includes packaging, purification, and QC to deliver high-quality AAV suitable for in vivo experiments and functional studies.
Lentiviral vector development and production enabling stable gene delivery in dividing and non-dividing cells. Supports transgene overexpression, shRNA or miRNA knockdown, CRISPR genome editing, and HDR-based genome modification.
Conversion of validated lentiviral transfer plasmids into high-titer viral particles for experimental use. Packaging and purification workflows produce lentivirus suitable for reliable transduction and stable gene expression studies.
Recombinant adenoviral vectors for high-efficiency transient gene expression without genomic integration. Commonly used for functional genomics, pathway studies, and genome editing applications.
High-yield adenovirus amplification using the Ad.MAX™ helper-virus–free system. Designed for efficient viral rescue and scalable production for research applications.
Need custom viral vectors for your pre-clinical gene therapy?
Accelerate your gene therapy program with tailored viral vector solutions—from construct design and plasmid preparation to production, purification, and QC—across AAV, adenovirus, and lentivirus platforms. Our team optimizes vector design, titer, and performance to deliver reliable, high-quality vectors for in vitro and in vivo pre-clinical studies.
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