Lentiviral Vector Development Platform
As a commonly used molecular biology tool in scientific research, Lentiviral vectors are essential for gene delivery, particularly in the fields of cell and gene therapy. Our company focuses on cutting-edge technologies in vector design and manufacturing of lentiviral vectors for gene therapy applications to provide our customers with the development of lentiviral vectors for rare disease gene therapy. Our dedicated research team is confident in providing satisfactory services to our customers in terms of improving vector safety, efficacy, and manufacturing robustness.
Introduction to Lentiviral Vectors
Lentiviral vectors (LV) are typically constructed based on modified non-pathogenic HIV (Human Immunodeficiency Virus) or other lentiviruses. These carriers are designed to be non-pathogenic but can effectively deliver specific genes or payloads into cells. Lentiviral vectors can deliver genes directly or coordinate with suitable viral packaging genes to form lentiviruses. Lentiviruses are a type of retrovirus and a typical characteristic of retroviruses is their RNA genome's ability to reverse transcribe into a cDNA copy, which can then stably integrate into the host cell's genome.
Fig.1 The mechanism of lentiviral transduction. (Yew, Chee-Hong Takahiro, et al., 2022)
Lentiviral vectors are capable of efficiently transducing into cells, integrating the carried target genes into host cells for stable gene expression, thus playing a crucial role in cell and gene therapy-related research and technology development. Multiple research results indicate that using lentiviral vector transduced cells or inducing gene expression has high safety and outstanding efficacy.
Lentiviral Vectors for Rare Diseases
In the field of rare diseases, the use of lentiviral vectors in gene therapy may apply to many rare lysosomal diseases, and several lentiviral gene therapies for lysosomal diseases are being investigated. Lentiviral gene therapy has been shown to involve the insertion, modification, or deletion of genes using the family of lentiviruses that cause AIDS and other diseases. Lentiviral gene therapy has already been approved for the therapeutics of metachromatic leukodystrophy (MLD) and beta-thalassemia and is being investigated for use in at least a dozen other rare diseases, such as Fabry disease, Gaucher type 1 disease, Hunter disease, and Sanfilippo syndrome disease.
Our Services
As an innovative company specializing in the development of therapies for rare diseases, we use a variety of strategies to improve the expression and transduction efficiency of lentivirus vectors and achieve tissue or cell type specificity to help customers design and develop efficient and safe lentivirus vectors, as well as vector evaluation services.
- Lentiviral Vector Construction
Helping customers develop lentiviral vectors that carry therapeutic genes but cannot replicate or transfer viral functions to target cells.
- Lentiviral vector modification
- Gene integration
- Plasmid amplification
- Lentiviral vector sequencing
- Lentiviral Vector Production
Producing lentiviral vectors by the split genome approach and generating replication-incompetent lentiviral vectors through a producer cell.
- Lentiviral vectors were prepared by transient transfection or using producer cell lines.
- High-titer lentiviral vectors are prepared through different cell culture systems (adherent or suspension) and at different scales.
- Lentiviral Vector Purification
Provides purification services for lentiviral vectors using separation technologies based on chromatography and membrane processes.
Project Workflow
With leading technology and many years of successful experience in the development of gene delivery tools, our company is well-positioned to provide customers with lentiviral vector development services for the gene therapy of rare diseases. We are constantly working to optimize the efficiency of lentiviral vectors for gene delivery to achieve wider application in future gene therapy. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
Reference
- Yew, Chee-Hong Takahiro, et al. "Integrase deficient lentiviral vector: prospects for safe clinical applications." PeerJ 10 (2022): e13704.
All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.