Gene Therapy

Gene therapy modifies genetic material, offering long-term or curative outcomes for diseases.

Benefits of Using AAV
for Gene Therapy

Low Immune Response

AAV vectors are ideal for sensitive applications due to their low likelihood of triggering strong immune reactions.

Stable Gene Delivery

Unlike retroviral vectors, AAV provides non-integrating gene delivery, reducing risks of mutagenesis.

Scalable Production

High-quality production ensures a reliable supply for research and clinical trials, from small to large scale.

Wide Therapeutic Applications

Versatility makes AAV ideal for gene therapy targeting organs like the brain, liver, and retina.

How Vectors Work

for Gene Therapy

Vectors are essential tools in gene therapy, acting as delivery vehicles to transport genetic material into target cells. These engineered viral vectors, such as AAV and Adenovirus, carry therapeutic genes into the cell's nucleus, where the genetic instructions can be expressed to address a specific disease or condition. 

By leveraging the natural ability of viruses to deliver DNA or RNA, vectors enable precise and efficient gene delivery while minimizing the risks associated with wild-type viruses.

AAV for Gene Therapy Disease modeling

Key Applications

of AAV in Gene Therapy

Monogenic Disorders

Address conditions caused by single-gene mutations, such as Cystic Fibrosis, Hemophilia, and Sickle Cell Anemia.

Neuronal Disease

Revolutionizing neurological treatment with targeted AAV gene delivery. 

Rare Genetic Disorders

Provide hope for conditions like Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy.

Ocular Disease

Advancing vision restoration with targeted AAV-based gene therapy.
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High Efficiency of Transduction

Deliver therapeutic genes effectively to target cells, ensuring robust gene expression.
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Enhanced Safety Profiles

AAV vectors are non-pathogenic and exhibit low immunogenicity, minimizing adverse reactions.
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Expertise and Support

Tailored to meet the specific needs of diverse research and therapeutic applications.
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Broad Targeting Capability

AAV can transduce a wide range of cell types, including dividing and non-dividing cells.
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Long-Term Gene Expression

Ideal for sustained therapeutic effects in conditions requiring prolonged intervention.
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Proven Safety

Our AAV vectors are non-pathogenic and trigger minimal immune responses, making them safe for clinical use.

Use Case

A pharmaceutical company needed AAV vectors encoding 2 neuroprotective factors to inject into brain cells for Parkinson’s disease research. Vector Biolabs helped the customer come up with the best design, cloned both genes into our AAV backbone, and produced the ready for injection AAV virus. Every stage was monitored with quality control (QC) assays to ensure a high-quality virus for advancing therapeutic development.

The final turnaround time from cloning to final QC was between 4 to 5 weeks.