Herpes Simplex Virus Vector Development Service
Herpes simplex virus (HSV)-based vectors can host large amounts of foreign DNA and are suitable vectors for the development of gene therapy strategies for neurological diseases. Our company is committed to helping customers design and develop flexible, efficient, and safe herpes virus vectors for gene therapy of rare diseases, with the aim of finding suitable tools for gene delivery in the brain.
Background
HSV is a neurophilic double-stranded DNA virus that has been found to persist in the peripheral nervous system. A number of features make this herpes virus attractive as a viral vector for the treatment of rare neurological disorders, including displaying tropism for multiple cell types, the potential to carry a large payload, and the latent HSV genome remaining episomal as a closed-loop molecule without integrating into cellular DNA. Human HSV-1 is currently the most commonly used herpesvirus vector system in central nervous system experiments.
The development of HSV-based gene transfer vectors has come a long way in the last 20 years. Two main categories of HSV-based vectors have been developed including amplicons and replication-defective HSV vectors. Several studies have shown that HSV vectors with high loading capacity are applicable to the durable expression of large and/or multiple therapeutic genes in brain neurons without the expression of viral genes. As more knowledge is gained about the etiology of the disease, these vectors may play a greater role in the treatment of rare neurodegenerative diseases such as Huntington's disease (HD).
Fig. 1 Genome structures of wild-type HSV-1. (Artusi S, et al., 2018)
Our Services
Researchers at our company work to help customers develop HSV vectors for gene therapy of rare diseases, particularly neurological disorders, to efficiently deliver genes into the central and peripheral nervous systems. We focus on improving the properties of these vectors, particularly in terms of regulating the intensity and time course of transgene expression. Our services include, but are not limited to:
- Development of HSV amplicon vectors for rare disease gene therapies.
- Production of amplicon particles
- We provide two major methods, including the method based on infection with defective helper HSVs and the other method based on the transfection of HSV-1 genes.
- Improvement of HSV-1-based amplicon vectors
- We are committed to regulating and prolonging the kinetics in vivo of transgene expression induced by amplicon using different promoters and preventing transgene silencing, so as to achieve safe and lasting gene therapy in non-replicating cells.
- Development of replication-defective HSV vectors for rare disease gene therapies.
- We help customers develop different types of defective recombinant vectors, such as a non-replicating HSV-1 vector encoding for the Hex A alpha-subunit (HSV-T0alphaHex) used for gene therapy of lysosomal storage diseases with neurological involvement.
- Development of replication-competent or -attenuated vectors for rare disease gene therapies.
- We help customers develop different types of replication-competent HSV vectors, such as an HSV1716 attenuated vector used for gene therapy of Mucopolysaccharidosis (MPS) VII.
- Development of HSV-1-based viral vectors for rare skin disease gene therapies.
- Based on the unique advantage that the HSV-1 vector can enter skin cells more effectively, we provide customers with the development service of HSV-1 vector modified by editing technology to accelerate the development of several gene therapy drugs for rare skin diseases.
Applications
Our HSV vector development services are applicable to the development of gene therapies for a variety of diseases, including but not limited to:
- Huntington's disease (HD)
- Amyotrophic lateral sclerosis (ALS)
- Spinocerebellar ataxia (SCA)
- Lysosomal storage diseases (LSDs)
As an innovative biotechnology company, our company is committed to helping our customers develop appropriate gene therapy delivery tools. We provide specialist HSV vector development services aimed at addressing rare diseases such as neurological, hematological, and genetic disorders through gene therapy and accelerating the development of gene therapy products. If you are interested in our services, please contact us for more information.
Reference
- Artusi, S.; et al. Herpes simplex virus vectors for gene transfer to the central nervous system. Diseases, 2018, 6(3): 74.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.