Specific Promoter Driven Targeting Service for AAV Vector

Introduction

For developing therapies for neurological disorders and other hard-to-reach target diseases, Creative Biolabs' Specific Promoter Driven Targeting of AAV Vector Service delivers unparalleled gene delivery precision and efficiency via advanced promoter engineering and viral vector technology. It offers a clear, step-by-step pathway to ensure therapeutic genes are delivered with optimal specificity and efficacy, minimizing off-target effects and maximizing therapeutic potential.

Specific Promoter Driven Targeting of AAV Vector

Fig.1 By combining different promoters, the targeting of AAV to microglia is enhanced.¹

Brain resilience relies on neurogenesis, but early-life stress induces chronic neuroinflammation, depleting neural stem cells and impairing brain function. Specific promoter-driven AAV vectors offer a solution: they control gene expression at the cellular level, precisely delivering therapeutic genes to restore neurogenesis, counter inflammation, and aid complex neurological disorders.

AAV Applications Driven by Tissue/Cell-Specific Promoters

• Gene Therapy Field
• Liver Disease Treatment: TBG/hAAT promoter-driven AAV targets hepatocytes, delivering therapeutic genes to express deficient proteins (e.g., coagulation factors) for hereditary liver diseases like hemophilia.
• Cardiac Disease Treatment: cTnT promoter targets cardiomyocytes, delivering genes to repair function (e.g., promoting regeneration, improving contractility) for conditions like myocardial infarction.
• Neurological Disease Treatment: SYN promoter targets neurons (delivering neuroprotective/repair genes for Parkinson's, Alzheimer's); GFAP promoter targets astrocytes for astrocyte-related neurological disorders.
• Diabetes Treatment: Insulin promoter targets pancreatic β-cells, delivering genes to regulate insulin secretion or repair β-cell function.
• Disease Model Construction Field
• Cardiac Disease Models: ANF promoter-driven AAV9-ANF-Cre mediates atrial-specific gene recombination/knockdown, constructing models for studying atrial disease pathogenesis and therapies.
• Neurological Disease Models: Neuron/glia-specific promoters drive target gene overexpression/knockout, building models to study disease onset and progression.
• Basic Research Field
• Cell Function Studies: Specific promoters drive reporter genes to label target cells, aiding observation of cell behavior under physiological/pathological conditions.
• Gene Regulation Mechanism Studies: Genes linked to specific promoters are introduced into target cells to study their expression regulation and impacts on cellular function/phenotype.

Workflow

1. Required Starting Materials: To initiate the service, clients typically provide the following:
   • The complete sequence of the therapeutic gene or a clear description of the protein of interest.
   • Information on the specific target cell type (e.g., neurons, astrocytes, etc.) and tissue.
   • A concise summary of the disease model or therapeutic objective.
2. Promoter Engineering and Vector Construction: We engineer custom AAV vectors using our diverse cell-specific promoter library or novel designs, ensuring therapeutic genes are expressed exclusively in target cells to prevent systemic toxicity and ensure efficacy.
3. Vector Production and Quality Control: AAV vectors are produced in our advanced facility, with rigorous QC checks (titer, purity, integrity) to meet top standards for research and preclinical use.
4. In Vitroand In Vivo Validation (Optional): Optional validation includes dose-response studies and expression analysis in cell cultures or animal models to confirm vector specificity and function.
5. Final Deliverables: Upon project completion, you will receive a comprehensive package.
   • The purified, ready-to-use AAV vector.
   • Detailed quality control data and a Certificate of Analysis.
   • A full report on the vector construction, promoter details, and validation data.
6. Estimated Timeframe: The typical timeframe for this service ranges from 8 to 12 weeks, depending on the complexity of the promoter engineering and the scope of optional validation studies.

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What We Can Offer

Customer Reviews

"Using Creative Biolabs' Specific Promoter Driven Targeting of AAV Vector Service in our research has significantly improved the specificity of our gene delivery, allowing us to pinpoint neuronal subtypes in the hippocampus and more accurately model neurogenesis after early-life stress."

- Dr. Grace Sullivan, Lead Researcher, BioMed Institute.

"The ability to precisely target specific glial cells with Creative Biolabs' AAV vectors was a game-changer for our neuroinflammation studies. It enabled us to achieve therapeutic gene expression while avoiding the pleiotropic effects that we saw with other less-specific delivery methods."

- Dr. James Foster, Neuroscience Lab Director.

"We compared Creative Biolabs' targeted AAV vectors to three other commercially available options and found that their vectors consistently provided a higher expression level in our target cell type with virtually no expression in neighboring tissues, which was a critical advantage for our preclinical safety studies."

– Dr. Sarah Voss, Director of Preclinical Development.

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FAQs

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Reference

1. Stamataki, Maria, et al. "Microglia targeting by adeno-associated viral vectors." Frontiers in Immunology 15 (2024): 1425892. https://doi.org/10.3389/fimmu.2024.1425892. Distributed under Open Access license CC BY 4.0, without modification.