Retroviral Vector Development Service
Retroviral vector-mediated gene transfer is central to the development of gene therapy. Our company is committed to providing customers with comprehensive retroviral vector development services, including the design of safer vectors, the establishment of transduction protocols, and the evaluation of the efficacy and safety of gene therapy vectors, accelerating the development of retroviral-based gene therapies for rare diseases.
Background
Retroviruses are single-stranded RNA viruses capable of converting RNA to cDNA by the action of reverse transcriptase, which is then amplified by the action of proteases such as DNA replicase, transcriptase, and translatorase. Researchers usually design replication-defective viruses based on certain properties of retroviruses to produce expression vectors capable of carrying specific target genes, known as retroviral vectors.
Retroviral vectors are currently the most widely used method for human gene transfer due to their relatively high efficiency of gene transfer and their ability to randomly insert and stably integrate exogenous genes into the host cell genome for sustained expression. Among these, lentiviral vectors and gamma-retroviral vectors have been widely used in gene therapy, for example, to introduce Chimeric Antigen Receptor (CAR) genes into T cells for the targeted killing of tumors by CAR-T cells. Gene therapy has gradually replaced currently available therapies for rare genetic diseases, such as enzyme replacement therapies, autologous transplantation, and antibody therapies.
Fig. 1 Genetic organization and morphology of retroviruses. (Maier P, et al., 2010)
Our Retroviral Vector Development Services
Based on the extensive expertise of our researchers in the complexities of retrovirus-mediated gene transfer, including cell targeting and control of genomic insertion sites, we are confident that we can provide our customers with development services of safer vectors and therapeutic protocols. Our services include, but are not limited to:
- Design of replication-defective vectors for rare disease gene therapies.
We replace most or all of the coding region of the retrovirus with the gene or sequence element to be transferred to avoid the vector making the proteins needed for additional cyclic replication. - Design of retroviral vectors for protein expression. We offer a variety of strategies, such as using promoters in retroviral long terminal repeats (LTRs) to control the expression of cDNAs encoding target proteins, to help customers design retroviral vectors that express one or more proteins.
- Construction of self-inactivating vectors.
- Optimization of the retroviral vector genome architecture.
- Production of high-titer vectors and development of methods to facilitate vector infection of cells to maximize the efficiency of gene transfer.
- Improvement of transfer and targeting of retroviral vectors by glycoprotein engineering.
We help our customers develop more effective engineering targeted envelope proteins to improve retroviral vector gene therapies. - Design of retroviral packaging cells and production of vectors.
- Characterization of retroviral vector insertion sites and biosafety assays.
We help customers assess the role of vector elements to further better understand the potential side effects of gene therapies using techniques to characterize retroviral vector insertion sites, such as in vitro immortalization (IVIM) assays.
Applications
Our retroviral vector development services are suitable for the development of gene therapies for a wide range of rare monogenic diseases. Examples of common applications are below:
- Development of gene therapies based on gammaretroviral vectors for severe combined immunodeficiency (SCID)
- Development of gene therapies for Wiskott–Aldrich syndrome
- Development of gene therapies for junctional epidermolysis bullosa (JEB)
Our company has a well-established portfolio of products, services, and solutions in the field of gene therapy research to accelerate your retrovirus-based gene therapy development program for rare diseases. If you are interested in our services, please contact us for more information.
Reference
- Maier, P.; et al. Retroviral vectors for gene therapy. Future microbiology, 2010, 5(10): 1507-1523.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.