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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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...
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RNA Editing02:23

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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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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CRISPR01:59

CRISPR

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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What is Genetic Engineering?00:49

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Overview
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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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Updated: Jul 20, 2025

Adeno-Associated Virus-Mediated Delivery of CRISPR for Cardiac Gene Editing in Mice
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Gene editing therapeutics based on mRNA delivery.

Juliana Popovitz1, Rohit Sharma2, Reyhane Hoshyar1

  • 1GenEdit, 681 Gateway Blvd., South San Francisco, CA 94080, USA.

Advanced Drug Delivery Reviews
|July 29, 2023
PubMed
Summary

Gene editing therapeutics using mRNA offer new ways to treat genetic disorders. This review covers mRNA delivery, gene editing technologies, and future advancements in CRISPR and base editing systems.

Keywords:
Base editingCRISPRDeliveryGene editingGene therapyNanoparticlemRNA

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Area of Science:

  • Molecular Biology
  • Biotechnology
  • Genetic Engineering

Background:

  • Gene editing technologies like CRISPR-Cas, base editors, and other systems show significant therapeutic promise.
  • These technologies offer potential treatments for previously untreatable genetic disorders.

Purpose of the Study:

  • To review the therapeutic applications of gene editing technologies utilizing messenger RNA (mRNA) delivery.
  • To discuss advancements in gene editing technology engineering and improvement.

Main Methods:

  • Focus on mRNA delivery for gene editing applications.
  • Examination of both ex vivo and in vivo gene editing strategies.
  • Review of next-generation CRISPR and base editing systems.

Main Results:

  • mRNA delivery is a viable method for gene editing therapeutics.
  • Engineering and improvements are enhancing gene editing technology efficacy.
  • Ex vivo and in vivo approaches demonstrate diverse therapeutic potentials.

Conclusions:

  • mRNA-based gene editing holds substantial promise for treating genetic diseases.
  • Ongoing advancements in gene editing technologies, including CRISPR and base editors, are expanding therapeutic possibilities.