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Related Concept Videos

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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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Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry
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Biodegradable Polymers for Gene Delivery.

T J Thomas1,2, Heidar-Ali Tajmir-Riahi3, C K S Pillai4

  • 1Department of Medicine, Rutgers Robert Wood Johnson Medical School, KTL N102, 675 Hoes Lane, Piscataway, NJ 08854, USA. thomastj@rwjms.rutgers.edu.

Molecules (Basel, Switzerland)
|October 20, 2019
PubMed
Summary
This summary is machine-generated.

Gene therapy faces challenges due to cell barriers. Biodegradable polymers offer a safer alternative to viral vectors for delivering DNA, improving gene therapy for genetic diseases.

Keywords:
DNA condensationDNA nanoparticlesbiodegradable polymerschitosandextrangene deliverygene delivery mechanismshyaluronic acidpoly-L-lysinepolyethyleneiminepullulan

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Area of Science:

  • Biotechnology
  • Polymer Science
  • Molecular Biology

Background:

  • Cell membrane barriers impede DNA transport, necessitating effective gene delivery vehicles for gene therapy.
  • Viral vectors, while effective, present significant limitations including immunogenicity, toxicity, and oncogenesis.
  • Non-viral polymeric carriers like polyethyleneimine condense DNA but raise toxicity concerns due to poor biodegradability and tissue accumulation.

Purpose of the Study:

  • To review the mechanistic aspects of gene delivery.
  • To summarize the development of biodegradable polymers for enhanced gene delivery applications.
  • To address the limitations of current gene delivery systems.

Main Methods:

  • Literature review of gene delivery mechanisms.
  • Analysis of biodegradable polymer development strategies.
  • Evaluation of polymer modifications and natural polymer utilization for gene delivery.

Main Results:

  • Understanding gene delivery mechanisms is crucial for vehicle design.
  • Biodegradable polymers are being developed through polymer modification and natural sources.
  • These advancements aim to mitigate the toxicity associated with non-biodegradable gene delivery systems.

Conclusions:

  • Biodegradable polymers represent a promising avenue for safer and more effective gene therapy.
  • Continued research into biodegradable materials is essential for overcoming current gene delivery challenges.
  • Developing efficient and non-toxic gene delivery vehicles is key to realizing the full potential of gene therapy.