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RNA Editing Therapeutics Development

Existing treatment strategies for rare genetic diseases often represent only supportive or palliative care, lacking effective treatment options. Our company focuses on emerging RNA editing-based tools including RNA editing with adenosine deaminases acting on RNA (ADARs) technology and CRISPR-Cas13 technology to help our clients develop RNA editing-based gene therapies. Our researchers' expertise and experience in optimizing the accuracy, editing efficiency, and delivery of RNA editing tools can help our clients make progress in gene therapy for rare diseases.

Background

The rapid development of RNA-based gene editing technologies and gene delivery systems engineering offers the possibility of successful drug development for rare diseases. The greatest advantage of RNA editing over DNA editing is that RNA has a very short life cycle and therefore RNA modifications are reversible. Notably, RNA editing has unparalleled advantages in specific areas, especially in the face of diseases associated with abnormal variable splicing of transcripts.

CRISPR-Cas systems on RNA are under development. For example, CRISPR/Cas13 is widely used in RNA knockdown, RNA single-base editing, RNA targeted modification, RNA live cell tracing, and nucleic acid detection. In addition, researchers are also exploring new RNA editing techniques, such as ADAR-based RNA editing to correct G-to-A mutations behind genetic disorders such as Rett syndrome and Hurler syndrome. In conclusion, RNA editing-based therapies are expected to be efficient and safe gene therapies for the treatment of rare genetic diseases.

Fig. 1 Factors affecting ADAR-mediated RNA editing.

Fig. 1 Factors affecting ADAR-mediated RNA editing. (Booth B J, et al., 2023)

Our Services

We provide customized CRISPR/Cas13-related services to support the development of gene therapies for rare diseases. We help drug development companies and researchers to flexibly apply CRISPR/Cas13 systems for RNA group editing and human rare disease gene therapy research. We assist our clients to develop RNA base-based editing systems such as programmable A-to-I substitution RNA editing (REPAIR) through a rational design approach and further improve the specificity and efficiency of the system. Our services include, but are not limited to:

  • CRISPR/Cas13 crRNA design and synthesis
    We design and synthesize crRNA sequences based on the gene sequences provided by our clients to improve target editing performance.
  • CRISPR/Cas13 vector construction services
    We provide the design and construction of various vectors, such as crRNA-Cas13, crRNA only, and Cas13-ADAR.
  • CRISPR/Cas13-based RNA editing services
    We provide CRISPR-Cas13-based RNA editing services for RNA knockdown and post-transcriptional level regulation of gene expression. In addition, we offer CRISPR-dCas9-based methods for regulating ncRNA function, inducing RNA decay, or inhibiting RNA translation.
  • RNA modification
    We help our clients add chemical modifications to specific RNAs through Cas13 editing for therapeutic purposes to promote the stability of targeted mRNAs.
  • ADAR-mediated RNA editing services
    • CIRTS: CRISPR-Cas-inspired RNA targeting system. We correct transcripts containing G to A mutations by linking the system to an effector protein such as human adenosine deaminase.
    • RESCUE: RNA editing for specific C to U exchanges. We induce RNA editing by fusing a dead Cas13 (non-catalytic Cas13) to the modified deaminase structural domain of ADAR2.
    • RESTORE: Recruitment of endogenous ADAR to specific transcripts for oligonucleotide-mediated RNA editing. We provide chemically optimized antisense oligonucleotides to recruit ADARs to edit endogenous transcripts in a programmable and simple manner.
    • LEAPER: Programmable RNA editing using endogenous ADARs. We design short-engineered ADAR recruiting RNAs (arRNAs) for our clients and employ arRNAs to recruit ADARs and convert specific A to I. And we deliver arRNA via plasmids, viral vectors, or synthetic oligonucleotides for precise and efficient RNA editing.

As a biotechnology company focused on rare diseases, our company is dedicated to turning cutting-edge RNA editing technologies into life-saving therapies. We provide scientific services and technical support to help drug development companies and researchers develop RNA-targeted therapies for rare diseases across a range of therapeutic areas. If you are interested in our RNA editing therapy development services, please contact us for more details.

Reference

  • Booth, B. J.; et al. RNA editing: expanding the potential of RNA therapeutics. Molecular Therapy, 2023.

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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