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Applications of AAV in Gene Therapy and Functional Studies

Adeno-associated virus (AAV) has emerged as one of the most widely used viral vectors in the field of gene therapy and in vivo functional studies, owing to its excellent biosafety profile, low immunogenicity, broad host range, and sustained gene expression. AAV is a small, non-enveloped, single-stranded DNA virus from the Parvoviridae family, first discovered in 1965 as a contaminant in adenovirus preparations. Its genome, approximately 4.7 kb in length, consists of two essential genes—Rep and Cap—flanked by two inverted terminal repeats (ITRs) that are critical for replication and packaging. Recombinant AAV (rAAV) vectors, which have had the viral genes removed to enhance safety, rely on a triple plasmid transfection system in HEK293 cells for efficient production. To meet specific tissue targeting needs, a variety of AAV serotypes—each with distinct capsid properties and cellular tropisms—can be selected, making the system highly adaptable across different organs and research models. With years of experience and platform maturity, Creative Biogene offers high-quality, customizable rAAV packaging services powered by helper-free systems, advanced purification strategies, and rigorous quality control to support a wide range of gene therapy, functional genomics, and translational applications.

Case Study 1

In the primary visual cortex (V1), inhibitory interneuron activation enhances excitatory neuron response reliability through negative feedback that narrows receptive fields (RFs). Research demonstrates that optogenetically activating parvalbumin (PV) interneurons reduces RF width and improves visual encoding by increasing signal-to-noise ratio (SNR). Researchers confirmed this by measuring significantly improved SNR and reduced RF areas in pyramidal neurons following targeted neuronal activation.

For this study, researchers utilized Creative Biogene's AAV viral production services. Creative Biogene manufactured high-concentration (1013 vg/mL) viral particles that helped enable channelrhodopsin expression in both PV and pyramidal neurons.

Case Study 1:Figure 1 illustrates the virus injections and subsequent recordings in the deep layers of the visual cortex area V1, showing labeled neurons, fluorescent dendrites, and experimental setups for analysis. (doi: 10.1038/s41598-024-73995-4)Figure 1. The researchers utilized viral injections in the deep layers of the visual cortex area V1 to label neurons. (Baranauskas G, et al., 2024)

Case Study 2

Spinal cord injury (SCI) disrupts neural circuits, causing maladaptive changes in the spinal-sympathetic reflex pathway critical for autonomic regulation. A key consequence is an immune dysfunction characterized by excessive TNFR1 activity on excitatory interneurons, leading to compromised infection responses. This dysfunction operates through NF-κB signaling in VGluT2+ interneurons—a promising therapeutic target for immune homeostasis restoration.

Researchers utilized AAV5-mediated gene delivery for cell-specific targeting in the spinal cord. Creative Biogene constructed the custom AAV5-VGluT2-Cre and AAV5-VGat-Cre vectors essential for this study's precise targeting of excitatory and inhibitory interneurons. The approach employed intraspinal microinjections at thoracic levels T10-T12, enabling localized and efficient Cre recombinase expression.

Case Study 2:Figure 2 illustrates the effect of IKKβ knockdown in VGluT2+ interneurons on improving antiviral immunity after chronic spinal cord injury, independent of lymphocyte abundance in the spleen. (doi: 10.1016/j.bbi.2024.10.021.)Figure 1. The researchers utilized AAV5-VGluT2-Cre vectors for cell-specific IKKβ knockdown in VGluT2+ interneurons to study immune response after chronic SCI. (Martynyuk T, et al., 2025)

As the demand for precise, long-term, and safe gene delivery continues to expand in both academic research and clinical development, rAAV vectors remain at the forefront of innovation. Whether for overexpression, gene knockdown, CRISPR-based gene editing, or endogenous activation, rAAV offers a robust and scalable solution. At Creative Biogene, we are committed to enabling scientific breakthroughs by providing end-to-end AAV vector solutions—delivered with reliability, scientific rigor, and tailored technical support. Together with our clients, we aim to accelerate the translation of genetic insights into real-world impact

* For research use only. Not intended for any clinical use.
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