Lentiviral Vector Design for Gene Editing
Through decades of development and progress, gene-editing technology has made many breakthroughs in the field of disease treatment. As a lead company committed to human health improvement, Creative Biolabs has developed and licensed a set of advanced genetic therapy platform to facilitate clients' project progress by offering high-quality gene editing products and services.
Development of Gene Editing
Gene editing, a kind of genome engineering, is a technology to alter (including insertion, deletion or replacement) a specific DNA sequence in the genome of an organism or cell. As early as in the 1970s, scientists successfully introduced exogenous genes into the organisms in a random way. After decades of technique and expertise updating, researchers now can intentionally add, remove or change specific genes at target DNA sites in a rapid and precise manner. Gene editing technology is possibly applied in many fields, such as crops yield increase, pharmaceutical development, diagnosis, etc., particularly in gene therapy.
Currently, gene editing can be achieved by many recognized technologies and methods, including (i) commonly used engineered nucleases like clustered regularly interspaced short palindromic repeats (CRISPR) and transcription activator-like effector nucleases (TALENs); (ii) viral vector systems. Recently, viral vectors as delivery tools are found to exert increasing important actions in gene editing.
Figure 1. Genome-editing technologies (cartoons illustrating the mechanisms of targeted nucleases). (Gaj, 2016)
Lentivirus Vectors Design for Gene Editing
As known to all, engineered nucleases are the most extensively used approach to perform site-specific gene editing at target locations. Editing genes in vivo to achieve diseases therapy, an appropriate vector system is needed to deliver these nucleases, like CRISPR-Cas9 and TALEN. Compared with non-viral vector delivery, viral vectors exhibit several advantages, among which lentivirus and adenovirus vectors are commonly-used viral vehicles to deliver foreign genes into cells both in vivo and in vitro.
There are numbers of attractive advantages of lentivirus vectors for genes delivery, such as:
- The ability to transduce dividing cells and non-dividing cells, and even differentiated neurons;
- Prominent and efficient gene alteration;
- Lentivirus vector pseudotyping engineering with other viral proteins enables a desired cellular tropism.
Notably, the integrase enzyme in lentivirus makes lentivirus vectors randomly integrate into the host genome, leading to efficient, stable transgene expression. This non-specifically integration may cause unwanted results, such as insertional mutagenesis, gene silencing, off-target activity in CRISPR delivery. Non-integrating lentivirus vectors design is a favorable modification to alleviate these unwanted problems.
Figure 2. Viral vector methods for delivery of CRISPR. (Lino, 2018)
Services
Since the advent of CRISPR, gene editing has made dramatic progress in just a few years. At Creative Biolabs, gene therapy service is our focus as well as flourishing section. Based on the expertise and abundant experience accumulated in the past few years, we provide various lentiviral vector design services for different applications, which include but not limited to:
If you are interested, please contact us, we can customize our offering to meet your specific project needs.
References
- Gaj, T.; et al. (2016). Genome-Editing Technologies: Principles and Applications. Cold Spring Harbor Perspectives in Biology. 8(12): pii: a023754.
- Lino, C.A.; et al. (2018). Delivering CRISPR: a review of the challenges and approaches. Drug Delivery. 25(1): 1234-1257.
