Gene Editing for Gene Therapy
According to the design and implementation of site-specific nuclease technologies, scientists in Creative Biolabs are transforming basic science and personalized medicine through classical and contemporary approaches. We provide Transcription activator-like effector nucleases (TALENs), and CRISPR/Cas (CRISPR associated) systems services to our clients all over the world. Our services enable investigators to disrupt, add, and correct any gene in a diverse range of cell types and organisms. By using our transformative tools, the ability to manipulate and study model organisms has dramatically expanded. Also, we are trying our best to support the promise of correcting the genetic causes of many diseases.
Transcription activator-like effector nucleases (TALENs) technology
TALENs are fusions of the FokI cleavage domain and DNA-binding domains derived from TALE proteins. The central repeat domain of a TALEN consists of repeating units of 33-35 amino acids. TALENs induce targeted DSBs which activate DNA damage response pathways and enable custom alterations. TALENs represent a new class of artificial nucleases with high specificity and modularity. The simple DNA recognition code and the modular nature make TALEN an ideal platform for use in targeted gene correction. So far, TALENs have been proven as an efficient, specific, and relatively safe genome engineering tool to achieve specific gene targeting and manipulation in a variety of human cell types.
CRISPR/Cas (CRISPR associated) systems technology
CRISPR (clustered regulatory interspaced short palindromic repeats) systems are loci that contain multiple short direct repeats and provide acquired immunity to bacteria and archaea. Based on the crRNA and tracrRNA, CRISPR systems can introduce sequence-specific silencing of invading foreign DNA. The CRISPR/Cas systems have rapidly gone from being a niche technology to a mainstream method used by many biological researchers. Our service provides rapid, easy and efficient modification of endogenous genes in a wide variety of biomedically important cell types and in organisms that have traditionally been challenging to manipulate genetically. Also, a modified version of the CRISPR-Cas9 system has been developed, this system can recruit heterologous domains that can regulate endogenous gene expression or label specific genomic loci in living cells.
References
- Gaj, T., Gersbach, C. and Barbas, C. (2013). ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering. Trends in Biotechnology, 31(7), pp.397-405.
- Sander, J. and Joung, J. (2014). CRISPR-Cas systems for editing, regulating and targeting genomes. Nature Biotechnology, 32(4), pp.347-355.
- Niu, J., Zhang, B. and Chen, H. (2014). Applications of TALENs and CRISPR/Cas9 in Human Cells and Their Potentials for Gene Therapy. Molecular Biotechnology, 56(8), pp.681-688.
