Virus-Based Gene Delivery System Development for Stroke

With advances in molecular biology, new genes continue to be discovered, making gene therapy a novel and innovative approach to treating stroke. This approach uses DNA or RNA as pharmacological agents to manipulate target genes or treat disease using specific vectors, particularly adeno-associated viruses (AAVs). AAV-mediated gene therapies have been approved for several genetic diseases and cancers. Although clinical trial reports on AAV gene therapy for ischemic stroke are scarce, the growing number of preclinical studies on AAV-mediated gene therapy and significant advances in genetic engineering are expected to provide hope for the use of AAV vectors in stroke treatment.

Fig. 1. AAV vectors applying in preclinical stroke models. (Wang et al., 2023)

Our Virus-Based Gene Delivery System Development Services

The design of the gene delivery system is the key to gene therapy for stroke. As a leading service provider in the field of stroke, Ace Therapeutics offers reliable virus-based gene delivery system development services to help clients accelerate the development of gene therapy for stroke. Our team of experts utilizes molecular biology, viral vector design techniques, RNA sequencing, and bioinformatics to design and develop virus-based gene delivery systems to meet the unique needs of pharmaceutical companies.

From viral vector design to preclinical evaluation, Ace Therapeutics offers a full range of services to assist clients at every stage of the virus-based gene delivery system development process.

Viral Vector Design

Our team of experienced scientists carefully assists clients in the selection and design of optimal lentiviral and AAV vectors to ensure that the viruses are non-pathogenic, do not induce neurotoxicity, and can facilitate targeted drug delivery.

• Replacement of viral genomes with genomic therapeutic cassettes of interest (anti-inflammatory, anti-apoptotic, pro-angiogenic, neuro-regenerative genes, and other genes for post-ischemic molecular regulation).
• Synthesis or addition of complementary stranded viral vector motifs.
• Deletion of relevant genes from the genome to reduce the risk of pathogenicity during viral replication.
• AAV capsid engineering.
• Custom promoter design.

Viral Vectors Construction

We use a variety of tools (targeting markers, chemical genetic tools, and programmable genome editing tools) to construct viral vectors carrying a variety of stroke-related target genes, such as neurotrophic factors, transcription factors, inflammatory vesicles, lncRNAs, cytokines, neuroplasticity regulators.

Preclinical Studies of Virus-Based Gene Delivery System in Animal Models of Stroke

We provide in vivo models of stroke to evaluate the efficacy and safety of virus-based gene delivery systems. Our researchers can select stereotactic coordinates based on the anatomical features of the animals and perform local injection of targeted stroke therapy. We also offer ocular and nasal routes of administration. Specific routes of administration, viral titers, injection doses, and injection depths can be customized to meet customer needs.

• Monitoring of physiologic parameters during stroke
• Neurobehavioral assessment
• Histological analysis
• Measurement of infarct volume
• BBB permeability assessment

Ace Therapeutics helps clients advance preclinical studies of virus-based gene delivery systems. Working with Ace Therapeutics, we can help you have more flexibility in designing viral vectors with improved engineering properties, specific targeting vectors, optimized manufacturing processes and delivery strategies, and reduced side effects, making them an effective tool to aid in the treatment of stroke. If you are interested in our services, please do not hesitate to contact us! 

1. Wang, J., et al. (2023). Gene therapy of adeno-associated virus (AAV) vectors in preclinical models of ischemic stroke. CNS Neuroscience & Therapeutics, 29(12), 3725-3740.