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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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 inserted. The...

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Related Experiment Video

Updated: Jun 20, 2026

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry
08:51

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

Published on: March 1, 2013

Non-viral gene delivery using nanoparticles.

Andrew J Ditto1, Parth N Shah, Yang H Yun

  • 1The University of Akron, Sidney Olson Research Center, Department of Biomedical Engineering, Akron, OH 44325, USA.

Expert Opinion on Drug Delivery
|September 29, 2009
PubMed
Summary
This summary is machine-generated.

Developing safer gene therapy vectors is crucial. Biodegradable nanoparticles offer targeted delivery and controlled release, showing promise over toxic viral vectors for treating genetic diseases.

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Related Experiment Videos

Last Updated: Jun 20, 2026

Evaluation of Polymeric Gene Delivery Nanoparticles by Nanoparticle Tracking Analysis and High-throughput Flow Cytometry
08:51

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09:41

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08:53

Synthesis, Functionalization, and Characterization of Fusogenic Porous Silicon Nanoparticles for Oligonucleotide Delivery

Published on: April 16, 2019

Area of Science:

  • Biomedical Engineering
  • Nanotechnology
  • Gene Therapy

Background:

  • Gene therapy holds promise for genetic diseases, but human trials face challenges due to viral vector toxicity and immunogenicity.
  • Current research focuses on developing safer, non-viral gene delivery systems.
  • Degradable nanoparticles are emerging as a promising alternative due to their targeted delivery and controlled release capabilities.

Purpose of the Study:

  • To review current non-viral gene delivery devices.
  • To emphasize nanoparticle-based gene delivery systems.
  • To present the development and assessment of l-tyrosine-based polyphosphate nanoparticle systems.

Main Methods:

  • Review of existing literature on non-viral gene delivery systems.
  • Focus on nanoparticle-based delivery mechanisms.
  • Development and evaluation of novel l-tyrosine-based polyphosphate nanoparticles.

Main Results:

  • Degradable nanoparticles offer targeted intracellular delivery with controlled release, unlike other non-viral vectors.
  • These nanoparticles exhibit reduced toxicity and avoid accumulation compared to non-degradable counterparts.
  • l-tyrosine-based polyphosphate nanoparticles are highlighted as a specific development in this area.

Conclusions:

  • Degradable nanoparticles represent a significant advancement in non-viral gene delivery.
  • They offer improved safety and efficacy profiles over traditional viral vectors.
  • Further research into systems like l-tyrosine-based polyphosphate nanoparticles is warranted to overcome gene therapy challenges.