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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 Zinc-finger nucleases (ZFNs), 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.

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Zinc-finger nucleases (ZFNs) services

The ZFNs are fusions of the nonspecific DNA cleavage domain from the FokI restriction endonuclease with zinc-finger proteins. ZFN dimers induce targeted DNA double-strand breaks (DSBs) that stimulate DNA damage response pathways. The designed zinc-finger domain can binding specificity genomic site by directs the ZFN. Several different outcomes that can result from the introduction of a site-specific DNA break. For example, if the break is resolved via non-homologous end joining (NHEJ), this can lead to the gene disruption, if the break is resolved via homology-directed repair (HDR), this can result in gene correction.

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Transcription activator-like effector nucleases (TALENs) services

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. Similar to the ZFNs, 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.

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CRISPR/Cas (CRISPR associated) systems services

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.

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References

  1. 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.
  2. Kianianmomeni, A. (2011). Genome editing using engineered zinc finger nucleases. Clinical Biochemistry, 44(13), p.S28.
  3. Sander, J. and Joung, J. (2014). CRISPR-Cas systems for editing, regulating and targeting genomes. Nature Biotechnology, 32(4), pp.347-355.
  4. 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.

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  • 45-1 Ramsey Road, Shirley, NY 11967, USA
  • Tel:1-631-381-2994
  • Fax: 1-631-207-8356
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