AAV Gene Therapy Development Services

AAV gene therapy for brain tumors uses adeno-associated virus vectors to deliver therapeutic genes directly into brain tumor cells, aiming to inhibit tumor growth or induce tumor cell death with high specificity and minimal side effects. Alfa Cytology provides AAV gene therapy development services for brain tumors, utilizing advanced gene editing and delivery technologies to develop effective and targeted treatments.

Introduction to AAV Gene Therapy for Brain Tumors

AAV (adeno-associated virus) gene therapy for brain tumors is an innovative treatment approach that utilizes engineered AAV vectors to deliver therapeutic genes directly into brain tumor cells. These vectors are designed to cross the blood-brain barrier and specifically target tumor cells without affecting healthy brain tissue. By introducing genes that can inhibit tumor growth, induce apoptosis, or enhance the immune response against tumor cells, this therapy aims to halt tumor progression.

Fig 1. Treatment of mouse gliomas with AAV-LIGHT therapy. (RAMACHANDRAN M, et al., 2023)

Key Mechanisms of AAV Gene Therapy for Brain Tumors

Direct Restoration or Compensation of Tumor Suppressor Genes

Through AAV vectors, functional tumor suppressor genes (e.g., TP53, PTEN) can be delivered to restore or enhance cell cycle regulators such as p53 and RB. This approach reduces tumor cell proliferation and induces apoptosis.

Expression of Pro-Apoptotic or Cytotoxic Molecules

By delivering toxin genes, suicide genes (e.g., HSV-TK), or pro-apoptotic factors (e.g., Bax, TRAIL, Caspases), AAV vectors selectively kill or induce apoptosis in tumor cells within the tumor microenvironment, enabling precise tumor eradication.

Modulation of the Tumor Microenvironment and Angiogenesis

AAV vectors can deliver anti-angiogenic agents (e.g., anti-VEGF) or regulatory genes (e.g., MMP inhibitors) to weaken the tumor's blood supply and improve its microenvironment. This helps slow tumor growth and curb invasion or metastasis.

Immunomodulation and Combination with Immunotherapy

AAV-mediated expression of cytokines or immune co-stimulatory molecules (such as IFN, IL-2, IL-12, CD40L) boosts local immune responses in the tumor. AAV can also deliver genes that block immunosuppressive pathways (e.g., inhibiting PD-L1).

Our Services

Alfa Cytology possesses the expertise and advanced capabilities to research AAV gene therapy for brain tumors. With extensive experience in gene editing and viral vector technologies, we are well-equipped to develop innovative treatments targeting brain tumors using AAV-mediated gene delivery.

Workflow of AAV Gene Therapy Development

Vector Design and Optimization

Leveraging AI-assisted AAV serotype screening and capsid engineering, we customize highly targeted, low-immunogenicity vectors for central nervous system (CNS) applications-such as penetrating the blood-brain barrier-or for tissue-specific delivery.

Viral Vector Production and Purification

We provide scalable AAV production services, encompassing plasmid construction, triple plasmid co-transfection, and suspension cell culture. Using gradient purification methods-such as iodixanol density gradient centrifugation and affinity chromatography-we ensure high purity (>90%) and high titers (≥1E13 vg/mL), supporting production needs for preclinical studies.

In Vitro and In Vivo Functional Validation

Alfa Cytology verify AAV delivery efficiency, target specificity, and therapeutic gene function using primary cells, 3D organoids, and animal models (e.g., orthotopic brain tumor models).

Safety and Immunogenicity Evaluation

We evaluate the immunogenicity and potential risks of AAV vectors using ELISPOT assays, neutralizing antibody detection, and off-target analysis through next-generation whole-genome sequencing (NGS).

Modeling Services for AAV Gene Therapy Development

Alfa Cytology's advanced in vivo and in vitro modeling technologies provide deep insights into AAV gene therapy efficacy and safety, empowering you to develop more forward-thinking gene therapy solutions.

In Vitro Models

In Vivo Models

Alfa Cytology boasts advanced laboratory and R&D capabilities, offering comprehensive, one-stop development services in AAV gene therapy for brain tumors. If you're interested in our work or potential collaborations, please feel free to contact us at any time.

Reference

RAMACHANDRAN M, VACCARO A, VAN DE WALLE T, et al. Tailoring vascular phenotype through AAV therapy promotes anti-tumor immunity in glioma [J]. Cancer cell, 2023, 41(6): 1134-51.e10.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.