CD Formulation is an industry leader in gene therapy formulation development, we focus on updating gene editing technology and provide customized services for gene therapy formulation development, including from customized media services to disposable technology and gene editing services. We have the expertise and equipment in gene therapy formulation development to help you rapidly advance the development of gene therapy formulations.
Challenges in Gene Editing Technology
While gene editing technology plays an important role in the development of gene therapy agents, it may also face multiple challenges, such as the off-target effect that CRISPR-Cas9 may trigger when targeting specific genes, leading to potential side effects. As well as gene editing technology may lead to genomic instability when applied, increasing the risk of cellular cancer. In addition, although gene editing technology is developing rapidly, the cost of its research and development and in practical application is still higher than many other problems, so facing the challenges of traditional gene editing technology in the development of gene therapy formulations, it is necessary to continue to find and optimize the problems of gene editing technology in the application of gene editing technology, and better help researchers to apply this technology in the development of gene therapy formulations.
Innovative Gene Editing Technology
We continue to optimize and update our technology systems to address the challenges of gene editing technology in the development of gene therapy agents. For example, we are developing novel CRISPR systems, including the miniature CRISPR-Cas system, to enable efficient genome editing in bacterial and human cells. We are also working to improve the efficiency and specificity of gene editing tools and to reduce the occurrence of accidental editing to improve the safety and accuracy of gene editing. At the same time, we are committed to improving the safety of gene editing and reducing the genotoxic effects generated by gene editing. In addition, we are continuing to optimize the gene expression system after gene editing to ensure the long-term stable expression of modified genes, etc.
Fig.1 Problems with conventional gene editing techniques. (CD Formulation)
Our Novel Gene Editing Technology
Our gene editing technology provides new solutions for gene function and disease research, and has been widely used in cell modeling, drug target screening, and gene function research, and has shown promising applications in gene therapy.
ZFNs technology
Zinc finger proteins specifically recognize and bind to three nucleotides on DNA to regulate gene expression. Utilizing this property of zinc finger proteins, zinc finger proteins capable of recognizing target DNA are obtained by designing specific zinc finger motifs, and then by fusing specific zinc finger proteins with non-specific nucleic acid endonucleases, zinc finger nucleases capable of specific cleavage of the target site can be obtained. Zinc finger nucleic acid technology greatly improves the efficiency of genome target modification compared with traditional gene recombination methods and exhibits higher specificity and precision.
TALENs technology
Like zinc fingers, such TALEN modules are designed in pairs to bind opposing DNA target motifs with appropriate spacing between the two binding sites, but in contrast to zinc finger proteins, TALENs do not require redesign of the junctions between the repetitive sequences that make up the long TALEN arrays, which can be targeted to individual genomic loci. Following the pioneering work with zinc finger proteins, multiple effector domains have become available to support the fusion of TALE repeat sequences for different genome modification purposes, including nucleases, transcriptional activators, and site-specific recombinases.The unlimited targeting range of TALENs, coupled with the ease of designing new proteins, makes TALENs an attractive platform for targeted gene editing.
CRISPR/Cas gene editing technology
For years, research into the CRISPR system across different microbial species has uncovered a fascinating process. Short segments of foreign nucleic acids are integrated into specific CRISPR regions, where they are transcribed and processed into CRISPR RNA (crRNA). This crRNA, along with trans-activated crRNA (tracrRNA), forms a complex with CRISPR-associated (Cas) proteins, playing a crucial role in the system's defense mechanism. The CRISPR/Cas9 system can be engineered to perform effective genetic modification in mammalian cells. CRISPR/Cas nucleases do not require the design of new proteins for each DNA target site. New sites can be targeted with relative ease by simply changing the short region of gRNA that determines specificity.
State-of-the-Art Platforms for Gene Editing Technology
| Our Technology Platforms |
Specifics |
Zinc finger nuclease (ZFNs) technology
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ZFNs technology is able to accurately recognize specific DNA sequences and edit target genes to minimize the impact on non-target genes. They can also create double-strand breaks in the target DNA, prompting the cell to utilize its own repair mechanisms to achieve gene editing. In addition, ZFNs are relatively flexible and adaptable in their design, and are also capable of being passed on to progeny cells during cell division. Currently, ZFNs technology has been used in studies for the development of gene therapy formulations for various diseases. We have continuously optimized the design and developed more efficient and safer methods for designing ZFNs to reduce the occurrence of off-target effects. |
Transcriptional activator effector nucleases (TALENs) technology
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TALENs technology plays an important role in the development of gene therapy agents. In addition to reducing interference with non-target genes, the application of this technology can be customized for any DNA sequence, which allows our researchers to rapidly develop gene editing tools for different diseases according to specific needs. In addition, TALENs can be used in combination with other gene editing technologies to increase the efficiency and diversity of gene editing. |
CRISPR/Cas gene editing technology
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CRISPR-Cas technology is the mainstream technology for gene editing, especially CRISPR-Cas9, CRISPR-Cas12a and CRISPR/Cas13 gene editing technologies. In addition, single base editing (BE) and wire editing (PE) technologies developed based on the CRISPR/Cas system have been widely used in various biological systems. CRISPR-Cas technology is characterized by greater precision and efficiency, and plays an irreplaceable and unique role in the areas of gene screening, modeling, and mechanism research. |
Highlights of Our Gene Editing Technology
- Extensive experience. To date, we have provided gene editing technology to several companies in the gene therapy field to aid in the development of gene therapy agents.
- Strict QC. We have a number of customizable quality control systems, high sensitivity NGS testing, to ensure the correctness of the test sequence.
- Compliance. Our technical services comply with the requirements of the relevant departments to declare the production environment, documentation, reporting information.
- Professional team. We have a R&D team that specializes in difficult issues, an efficient and rigorous production team, and a quality control team that understands regulations.
Explore Our Gene Editing Technology Related Services
Viral Gene Delivery Systems Development
We have enhanced our viral vector development services, which are designed to enable the delivery of targeted genes through viral vectors, and this service provides an important tool and plays an important role in the development of gene therapy formulations.
Non-Viral Gene Delivery Systems Development
We offer non-viral gene delivery system development services aimed at delivering genetic material to host cells with the help of non-viral vectors, which have demonstrated enhanced biosafety in gene therapy agent development applications. Our service involves lipid nanoparticles, bacterial vectors, exosomes, and others.
Viral Vectors Packaging
We provide viral vector packaging services by inserting target genes into viral vectors and delivering them to host cells, and this service has a promising application in the development of gene therapy formulations.
CD Formulation has grown to become a global provider of specialized services in the development of gene therapy formulations. We can provide comprehensive support services, especially for gene editing technology, to help our customers develop gene therapy drug formulations. We provide high-quality services with fast turnaround time, and we look forward to working with you. If you are interested in us, please feel free to contact us.
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