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TALEN-based Therapy Development Platform

Transcription activator-like effector nucleases (TALEN) have become an invaluable gene editing tool for researchers for potential gene therapy due to their unique advantage of precise targeting. Our company is committed to providing our customers with an all-in-one solution for TALEN-based therapy development, including experimental design, vector construction, and functional validation. Our professional and efficient scientific services can accelerate your rare disease gene therapy development projects.

Introduction to TALEN

Transcription activator-like effector nucleases (TALEN) are enzymes that target specific DNA sequences and recognize specific DNA base pairs with the help of TAL effectors, proteins secreted by Xanthomonas. A typical TALEN consists of an N-terminal domain containing the nuclear localization signal (NLS), a central domain containing the canonical tandem TALE repeat sequence that recognizes specific DNA sequences, and a C-terminal domain with FokI endonuclease function.

TALEN-based Therapy Development Platform-1

Fig.1 Illustration of applications of TALE and TALEN genome editing technologies. (Becker, S., and Boch, J., 2021)

Several research groups have reported the application of TALEN technology in gene therapy, such as for the design of stably modified human erythroid cell lines and human induced pluripotent stem cells (hiPSCs). In addition, there are some other widespread applications:

  • For the correction of genetic errors that cause disease.
  • For cell-level genome modification in yeast, animal and plant cells, etc.
  • For drug discovery and target validation.
  • Potential as a tool to control the immune system against cancer.

TALEN for Rare Diseases

TAL effectors can be designed and modified to recognize and bind almost any desired target DNA sequence. Combining TAL effectors with DNA cleavage domains yields restriction endonucleases capable of binding to DNA and cleaving DNA strands at specific sites. This technique has been widely used for gene knockout, knock-in, and transcriptional activation, and plays an important role in gene therapy for rare diseases. It has been used in the treatment of sickle cell anemia, neurological familial genetic diseases and other rare diseases.

Our Services

Our company's TALEN-based therapy development platform provides you with TALEN gene editing technology and TALEN development for rare disease research process. We can use TALEN to quickly construct a variety of animal models that simulate pathogenic mutations of human genetic diseases, and use TALEN gene editing technology to safely and stably express functional genes to treat genetic diseases, helping you complete a one-stop solution for gene rare diseases therapy. Our services include, but are not limited to:

TALEN Design

Find possible TALEN binding sites in specific DNA sequences to correctly design TALEN through various tools such as TALE-NT and TALENoffer, and provide our customers with unwanted off-target prediction services.

TALE and TALEN Assembly

Help customers construct TALEs and TALEN with tailored DNA-binding specificities through the assembly method of Golden Gate Cloning.

TALEN Optimization

  • Develop obligate heterodimerized TALEN variants to increase target specificity, improve nuclease activity, and reduce toxicity.
  • The foki-based TALEN structure was optimized through "Sharkey" mutations to improve the activity of heterodimeric TALENs.
  • Develop other variants, such as fusing TALE DNA-binding domains to the restriction endonuclease PvuII to obtain TALE-PvuII nucleases.

TALEN Expression Detection

In order to ensure the effectiveness of TALEN and reduce the workload of subsequent screening of mutants, our company provides TALEN expression detection services. The higher the activity of TALEN, the less workload of subsequent screening of mutants.

TALEN Delivery

TALEN can be used as DNA, RNA, or protein. Our company completes the delivery of TALEN through different methods, including:

  • physical methods (such as microinjection or electroporation)
  • virus-based delivery methods (such as adenoviruses, lentiviruses, and baculoviruses)
  • chemical methods (such as methods that utilize transfection reagents, PEG, or liposomes)

Project Workflow

TALEN-based Therapy Development Platform-2

With a best-in-class technology platform and a dedicated scientific team, our company provides TALEN-based therapy development services to customers around the world. TALEN effector proteins designed by our researchers have helped our customers advance numerous biomedical applications, including gene therapy for rare diseases. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.

Reference

  • Becker, S., and Boch, J., "TALE and TALEN genome editing technologies." Gene and Genome Editing 2 (2021): 100007.

All of our services and products are intended for preclinical research use only and cannot be used to diagnose, treat or manage patients.

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