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mRNA for Gene Therapy

Background mRNA-based Gene Therapy Services Highlights FAQs Published Data

Background

Creative Biolabs is a leading service provider that focuses on messenger ribonucleic acid (mRNA) therapeutics development. Currently, various platforms have been established for analyzing the function and mechanism of action of mRNA. By combining cutting edge technologies with proprietary innovations, scientists at Creative Biolabs provide a full range of services on mRNA-based gene therapy. With our extensive experience and advanced platform, we are always dedicated to assisting our clients with the most satisfactory data.

  • Introduction of Gene Therapy

Gene therapy has shown promising results in the treatment of human diseases, including tumors, autoimmune disorders, and infectious diseases. Normally, gene therapy is generated by transferring specific genetic information into target cells to alter their functions in disease therapy. In particular, gene therapy can be used for replacing or blocking mutated genes that cause diseases. Moreover, recent reports have demonstrated that gene therapy can also be used for inserting target genes against diseases. Meanwhile, many studies have indicated that vectors play a significant role in delivering genes. Different types of delivery vectors have been designed in the past few years of studies. Among them, several kinds of virus-based vectors, such as retroviral vectors, lentiviral vectors, adenoviral vectors, as well as adeno-associated viral vectors, are widely used in gene therapy development. Besides, many advanced technologies, such as genetic engineering, recombinant DNA technique, as well as mRNA technology, have been utilized in improving the performance of gene therapy in disease treatments.

In vitro mRNA production process.Fig.1 Generation process of the mRNA.1

Services for mRNA-based Gene Therapy

Since the great success of gene therapy in cancer treatment, many attempts have been made recently to improve their safety and efficacy both in preclinical trials and clinical trials. Also, recent researchers have illustrated that some viral vectors can cause many severe side effects in disease models. Nowadays, mRNA molecules have been considered as suitable alternative plasmids for gene therapy due to their rapid and high-level expression in cancers. Furthermore, many reporters have revealed that mRNA-based gene therapy can be achieved at different levels, such as cell level, exosome level, and molecular-level. Till now, several mRNA-based modalities have been designed and developed for treating glioblastoma multiforme (GBM) in preclinical studies and some of them are undergoing different phases of clinical trials.

Therefore, Creative Biolabs has established a panel of platforms to design and validate potential mRNA molecules for the treatment of various human diseases, including cancers. Equipped with several advanced technologies, we can provide a seamless service chain, ranging from in silico mRNA structure prediction, mRNA constructs design, animal modeling, to safety and toxicity evaluation. Meanwhile, we are capable of offering a wide array of optimization assays to improve the efficacy of mRNA-based gene therapy.

  • De novo synthesis of the functional proteins
  • The delivery of the specific mRNA to the target cells
  • In vitro and in vivo assessment
Schematic diagram of the mechanism of action of retroviral vectors in gene therapy.Fig.2 The retroviral vectors used in gene therapy.2

With over years of experience in mRNA therapeutics development, Creative Biolabs offers a large collection of mRNA-based assays targeting different types of disease targets. Our client-focused, integrated team will anticipate challenges, overcome difficulties, and successfully convert various potential targets into clinical trials. If you are interested in our services, please feel free to contact us for more information.

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Highlights

  • Comprehensive Service Chain: Creative Biolabs offers a complete range of services, from in silico mRNA structure prediction to animal modeling, safety, and toxicity evaluation, ensuring robust mRNA-based gene therapy development.
  • Wide Application Scope: The platform is designed to treat various diseases, including cancers, autoimmune disorders, and infectious diseases, with a focus on improving safety and efficacy in preclinical and clinical trials.
  • Client-Focused Approach: The experienced team at Creative Biolabs anticipates challenges and converts potential therapeutic targets into clinical trials, offering tailored solutions for successful gene therapy outcomes.
  • Optimization and Assessment: Creative Biolabs provides optimization assays to enhance mRNA efficacy, coupled with comprehensive in vitro and in vivo assessments to ensure the therapeutic potential of the developed mRNA constructs.

FAQs

Q: What is mRNA-based gene therapy?

A: mRNA-based gene therapy involves using messenger RNA (mRNA) to deliver specific genetic information into target cells. This therapy aims to treat or prevent diseases by either replacing faulty genes, blocking harmful genes, or introducing new genes that can fight disease. Unlike DNA-based gene therapy, mRNA does not integrate into the genome, offering a potentially safer approach.

Q: How does mRNA-based gene therapy differ from traditional gene therapy?

A: Traditional gene therapy often uses viral vectors to deliver DNA into the genome, which can lead to long-term gene expression but also risks genomic integration and related side effects. mRNA-based therapy, on the other hand, provides transient expression without integrating into the genome, reducing the risk of insertional mutagenesis and making it a safer option for some applications.

Q: What diseases can be targeted using mRNA-based gene therapy?

A: mRNA-based gene therapy is being explored for a wide range of diseases, including cancer, genetic disorders, autoimmune diseases, and infectious diseases. It is particularly promising for conditions that require rapid and high-level expression of therapeutic proteins, such as certain types of cancer and rare genetic diseases.

Q: What are the advantages of using mRNA in gene therapy?

A: mRNA offers several advantages, including rapid production, high levels of protein expression, and the ability to avoid the risks associated with DNA integration into the genome. Additionally, mRNA-based therapies can be tailored for specific patients, providing a more personalized approach to treatment.

Q: How does Creative Biolabs ensure the safety of mRNA-based gene therapies?

A: Safety is ensured through comprehensive preclinical evaluations, including in vitro and in vivo assessments, to test the efficacy, toxicity, and potential off-target effects of the mRNA therapies. Creative Biolabs also employs advanced delivery systems and optimized mRNA constructs to minimize immune responses and enhance therapeutic outcomes.

Q: What services does Creative Biolabs offer for optimizing mRNA-based gene therapies?

A: Creative Biolabs provides a range of optimization services, including mRNA sequence optimization, delivery system development, and in vitro/in vivo testing. These services are designed to enhance the stability, expression, and therapeutic efficacy of mRNA-based gene therapies, ensuring they meet the specific needs of each research project.

Published Data

In this study, survivinT34A mRNA was employed as a therapeutic agent for colon cancer gene therapy, delivered via a liposome-protamine lipoplex (CLPP). The survivinT34A mRNA encodes a mutant form of survivin, an anti-apoptotic protein, which induces apoptosis in cancer cells. The CLPP delivery system was designed to protect the mRNA and enhance its delivery into target cells. Experimental results showed that the CLPP/mSur-T34A formulation effectively inhibited tumor growth both in vitro and in vivo in various colon cancer models. The treatment resulted in significant tumor cell apoptosis, reduced tumor volume, and decreased metastatic spread. The mRNA-based therapy demonstrated superior efficacy compared to its DNA plasmid counterpart, highlighting its potential as a promising candidate for colon cancer therapy.

The synthetic mSur-T34A mRNA can effectively inhibit tumor cell growth in vitro.Fig.3 CLPP/mSur-T34A particles inhibited the growth of C26 colon cancer cells in vitro.3

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References

  1. Michel, T.; et al. Next-Generation Therapeutics: mRNA as a Novel Therapeutic Option for Single-Gene Disorders. Modern Tools for Genetic Engineering. 2016, 18: 3-20.
  2. Verma, I. M.; et al. Gene therapy: promises, problems, and prospects. InGenes and Resistance to Disease. 2000, 20: 147-157.
  3. Zhang, Xueyan, et al. "Local and systemic delivery of mRNA encoding survivin-T34A by lipoplex for efficient colon cancer gene therapy." International Journal of Nanomedicine (2019): 2733-2751.
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