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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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Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
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Related Experiment Video

Updated: May 3, 2026

Constitutive and Inducible Systems for Genetic In Vivo Modification of Mouse Hepatocytes Using Hydrodynamic Tail Vein Injection
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Methods for Targeted Gene Transfer to Liver Using DNA-Protein Complexes.

M A Findeis1, C H Wu, G Y Wu

  • 1Pharmaceutical Peptides, Inc., Cambridge, MA.

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Gene therapy research explores various delivery methods for treating diseases. Targeting the asialoglycoprotein receptor (ASGr) on liver cells offers a highly selective gene delivery strategy.

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Area of Science:

  • Biomedical research
  • Molecular biology
  • Gene therapy

Background:

  • Therapeutic gene transfer holds significant potential for treating human diseases.
  • Diverse research efforts are underway to develop effective gene therapy strategies.
  • Current methods include viral vectors, direct injection, and liposomes.

Purpose of the Study:

  • To explore and develop advanced gene therapy strategies.
  • To investigate targeted DNA delivery methods for enhanced therapeutic efficacy.
  • To focus on receptor-mediated gene transfer for selective delivery.

Main Methods:

  • Utilizing receptor-mediated endocytosis for targeted gene delivery.
  • Employing protein ligands to target specific cell-surface receptors.
  • Investigating the hepatic asialoglycoprotein receptor (ASGr) for liver-specific targeting.

Main Results:

  • Successful application of targeted DNA delivery using protein ligands to the ASGr.
  • Demonstrated high representation of ASGr on hepatocytes, enabling selective targeting.
  • Transferrin receptor also identified as a target for gene delivery.

Conclusions:

  • Targeting the ASGr provides a highly selective method for delivering genes to the liver.
  • Receptor-mediated gene transfer is a promising strategy for developing targeted therapies.
  • Further research into receptor-mediated delivery can advance gene therapy applications.