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Non-coding DNA Therapy Development

Various ongoing studies in gene therapy focus on providing the complete genetic loci of the genes of interest, which includes natural regulatory elements such as introns, 3' and 5' flanking sequences, promoter elements, and enhancer elements. Our company provides comprehensive services for leading and emerging biopharmaceutical companies and scientific research institutions to develop Non-coding DNA therapy for rare diseases.

What is Non-Coding DNA?

In DNA, only about 1% consists of protein-coding genes, while the remaining 99% is non-coding DNA. Non-coding DNA does not provide instructions for protein production and was once considered "junk DNA". However, researchers have gradually discovered that at least some non-coding DNA is an indispensable part of cellular functions, especially in controlling gene activity. Several non-coding DNA contains sequences that act as regulatory elements, determining when and where genes are turned on or off. DNAzyme obtained through SELEX in vitro selection, which can specifically recognize mRNA sequences and perform cleavage, is another type of non-coding DNA.

2-1-3-1-3 Non-coding DNA Therapy Development-1Fig.1 Schematic diagram of the proposed genome defense by non-coding DNA in younger (A) and older (B) adult somatic cells. (Qiu, Guo-Hua, et al., 2019)

Non-coding DNA comprises various elements that collectively regulate gene activation and suppression. In the past, the role of non-coding DNA was often overlooked. Currently, researchers have found that mutations in the non-coding regions of genes, including those associated with rare diseases and cancer, are prevalent. Targeting these mutations specifically for the treatment of rare diseases and inhibiting cancer growth is what's referred to as personalized/precision gene therapy.

Types of Non-Coding DNA

Non-Coding DNA Description
Satellite DNA Tandemly repeating sequences of DNA (e.g. STRs) structural components of heterochromatin and centromeres commonly used for DNA profiling
Telomeres Regions of repetitive DNA at the end of a chromosome protect against chromosomal deterioration during replication
Introns Non-coding sequences within genes are removed by RNA splicing before the formation of mRNA
Non-coding RNA genes Codes for RNA molecules that are not translated into protein, examples include genes for tRNA
Gene regulatory sequences Sequences that are involved in the process of transcription include promoters, enhancers, and silencers
DNAzyme DNAzymes are a special form of DNA molecules that possess catalytic activity, allowing them to catalyze specific chemical reactions, such as cutting or synthesizing RNA chains

Our Services

Based on advanced technology and a professional team, our company provides one-stop non-coding DNA therapy development services and can conduct in-depth research on various non-coding sequences and mutations. Treat various rare diseases by interfering with gene expression and regulating cell function through non-coding DNA therapy. Our customized solutions and comprehensive after-sales support can accelerate your research and development of rare disease therapies.

Non-coding DNA Related Rare Disease Diagnostics

  • Use next generation sequencing (NGS) ethods to sequence individual non-coding DNA to detect early-stage diseases and provide precise non-coding DNA therapy development.

Optional Non-coding DNA Therapeutics

  • Introns therapeutics development
  • Pseudogenes therapeutics development
  • Satellite DNA therapeutics development

Non-coding DNA Therapy Delivery

  • Electroporation
  • Ultrasound
  • Gene gun

Why Choose Us?

As an integrated CRO, our company has extensive expertise in non-coding DNA therapy development for rare disease research. Whatever your specific requirements, we will work closely with you to develop a customized solution. We can quickly respond to the changing needs of your rare disease research projects. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.

References

  • Qiu, Guo-Hua, et al. "The protective function of non-coding DNA in DNA damage accumulation with age and its roles in age-related diseases." Biogerontology 20.6 (2019): 741-761.
  • Simna, S. P., and Zongchao Han. "Prospects of non-coding elements in genomic DNA based gene therapy." Current gene therapy 22.2 (2022): 89.

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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