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Related Concept Videos

Gene Therapy00:59

Gene Therapy

26.9K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
26.9K
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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Related Experiment Video

Updated: Nov 26, 2025

Delivery of Modified mRNA in a Myocardial Infarction Mouse Model
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Delivery of Modified mRNA in a Myocardial Infarction Mouse Model

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Current Progress in Messenger RNA-Based Gene Therapy.

Sibei Lei, Xueyan Zhang, Jingmei Li

    Journal of Biomedical Nanotechnology
    |December 14, 2020
    PubMed
    Summary
    This summary is machine-generated.

    Synthetically created messenger RNA (mRNA) shows great promise for gene therapy due to its rapid expression and adaptability. Recent advancements in delivery vectors and materials science are enhancing mRNA therapeutics for various diseases.

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    In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells
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    In Vitro Synthesis of Modified mRNA for Induction of Protein Expression in Human Cells

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    Area of Science:

    • Biotechnology
    • Molecular Biology
    • Gene Therapy

    Background:

    • In vitro synthesized messenger RNA (mRNA) offers advantages for gene therapy, including rapid expression, simple composition, low immunogenicity, and modification flexibility.
    • Progress in pathogenesis and materials science has significantly advanced mRNA-based therapeutic studies.
    • Numerous mRNA-derived therapeutics, such as vaccines, antibodies, cytokines, and growth factors, are being developed for diverse diseases.

    Purpose of the Study:

    • To review recent advancements in messenger RNA (mRNA)-based gene therapy.
    • To discuss novel delivery vectors and advanced strategies for mRNA therapeutics.

    Main Methods:

    • Review of current literature on mRNA-based gene therapy.
    • Analysis of novel delivery vector development.
    • Examination of advanced strategies including library screening, environmental interaction, and bio-inspired materials.

    Main Results:

    • Development of novel delivery vectors with improved capacity, safety, and properties for mRNA.
    • Application of advanced strategies leading to valuable research outcomes in mRNA therapeutics.
    • Emergence of diverse mRNA-derived therapeutics targeting various diseases.

    Conclusions:

    • Messenger RNA (mRNA) synthesized in vitro is a promising platform for gene therapy.
    • Continued research in materials science and delivery systems is crucial for optimizing mRNA therapeutic efficacy and safety.