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Related Concept Videos

MicroRNAs01:22

MicroRNAs

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MicroRNA (miRNA) are short, regulatory RNA transcribed from introns (non-coding regions of a gene) or intergenic regions (stretches of DNA present between genes). Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself, forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After the pre-miRNA...
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MicroRNA (miRNA) are short, regulatory RNA transcribed from introns—non-coding regions of a gene—or intergenic regions—stretches of DNA present between genes. Several processing steps are required to form biologically active, mature miRNA. The initial transcript, called primary miRNA (pri-mRNA), base-pairs with itself forming a stem-loop structure. Within the nucleus, an endonuclease enzyme, called Drosha, shortens the stem-loop structure into hairpin-shaped pre-miRNA. After...
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Gene Therapy00:59

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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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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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Overview of Exosomes01:36

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Exosomes are stable, lipid bilayer-enclosed vesicles capable of crossing biological barriers. They can carry a wide range of molecules required for intercellular communication. Once exosomes are released from the cell where they originated, they enter a recipient cell through various pathways such as fusion, receptor-mediated endocytosis, macropinocytosis, and phagocytosis.
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Systemic Delivery of MicroRNA Using Recombinant Adeno-associated Virus Serotype 9 to Treat Neuromuscular Diseases in Rodents
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Exosomes-Based Gene Therapy for MicroRNA Delivery.

Prabhu Mathiyalagan1, Susmita Sahoo2

  • 1Cardiovascular Research Center, Icahn School of Medicine, Mount Sinai, One Gustave L. Levy Place, Box 1030, New York, NY, 10029-6574, USA.

Methods in Molecular Biology (Clifton, N.J.)
|December 3, 2016
PubMed
Summary

Stem cell-derived exosomes can deliver microRNAs (miRNAs) to repair heart tissue. This novel gene therapy approach uses exosomes as natural nanovesicles for efficient cardiac repair and gene regulation.

Keywords:
CD34+ stem cellsExosomesGene therapyMicroRNAMicrovesicles

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Area of Science:

  • Cardiovascular Research
  • Regenerative Medicine
  • Nanotechnology

Background:

  • Cardiovascular disease remains a significant global health burden, necessitating advanced treatment strategies.
  • Current treatments for cardiovascular manifestations include drugs and surgeries, but gene-based therapies show great promise.

Purpose of the Study:

  • To investigate the potential of exosomes derived from human CD34+ stem cells for cardiac repair.
  • To develop a protocol for generating exosomes loaded with exogenous genetic material (miRNA precursors) for targeted gene delivery.

Main Methods:

  • Isolation and characterization of exosomes from human CD34+ stem cells.
  • Loading exosomes with Cy3 dye-labeled pre-microRNA (miRNA) precursors.
  • Demonstration of exosome uptake by recipient cardiac cells and delivery of miRNA precursors.

Main Results:

  • Human CD34+ stem cell exosomes efficiently enter recipient cells.
  • Delivered pre-miRNA precursors successfully regulate gene expression within target cells.
  • Exosomes serve as effective natural nanocarriers for RNA delivery, protecting cargo from degradation.

Conclusions:

  • Stem cell-derived exosomes offer a promising cell-free platform for cardiac gene therapy.
  • This method provides a novel tool for delivering exogenous genetic information to the heart for therapeutic purposes.
  • Further development of exosome-based therapies could lead to more effective cardiovascular disease treatments.