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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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Microorganisms in Medicine and Therapeutics01:29

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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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Generating Recombinant Avian Herpesvirus Vectors with CRISPR/Cas9 Gene Editing
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Engineering HSV-1 vectors for gene therapy.

William F Goins1, Shaohua Huang, Justus B Cohen

  • 1Department of Microbiology and Molecular Genetics and Biochemistry, University of Pittsburgh School of Medicine, 424 Bridgeside Point-II, 450 Technology Dr., Pittsburgh, PA, 15219, USA, goins@pitt.edu.

Methods in Molecular Biology (Clifton, N.J.)
|March 28, 2014
PubMed
Summary
This summary is machine-generated.

Gene therapy using herpes simplex virus (HSV) vectors shows promise for treating brain cancer and neurodegenerative diseases. Researchers are developing improved, replication-defective HSV vectors for safer and more effective gene transfer.

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Area of Science:

  • Gene Therapy
  • Virology
  • Oncolytic Viruses
  • Neuroscience

Background:

  • Gene therapy has advanced to standard patient care with FDA-approved treatments like Glybera.
  • Herpes simplex virus (HSV) vectors are utilized in clinical trials for glioblastoma multiforme and malignant melanoma.
  • Replication-defective HSV vectors are being explored for neurological disorders, including pain and neurodegenerative conditions.

Purpose of the Study:

  • To describe the construction of recombinant genomic HSV vectors.
  • To outline purification methods for these vectors.
  • To detail small-scale production for cell culture and preclinical studies.

Main Methods:

  • Engineering of recombinant, replication-defective HSV-1 vectors.
  • Purification techniques for genomic HSV vectors.
  • Small-scale production protocols for experimental use.

Main Results:

  • Development of replication-defective, non-toxic HSV vectors.
  • Demonstrated potential for long-term transgene expression in neurons.
  • Established methods for vector construction and purification.

Conclusions:

  • Recombinant HSV vectors offer a viable platform for gene therapy applications.
  • Engineered HSV vectors show potential for treating various diseases, including cancers and neurological conditions.
  • The described methods facilitate the use of HSV vectors in research and preclinical development.