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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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Related Experiment Video

Updated: Dec 25, 2025

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

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Nanoparticle depots for controlled and sustained gene delivery.

Zhongyu Li1, William Ho1, Xin Bai2

  • 1Department of Chemical and Materials Engineering, New Jersey Institute of Technology, Newark, NJ 07102, USA.

Journal of Controlled Release : Official Journal of the Controlled Release Society
|March 21, 2020
PubMed
Summary

This study introduces a novel nanoparticle (NP) system for sustained gene therapy delivery. These biodegradable NPs enhance gene expression by prolonging the release of therapeutic nucleic acids, overcoming key delivery challenges.

Keywords:
Gene therapyNanoparticlesSustained release

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Area of Science:

  • Biotechnology
  • Nanomedicine
  • Gene Therapy

Background:

  • Gene therapy offers promising treatments for genetic diseases and cancer but faces delivery challenges like rapid clearance and degradation.
  • Sustained drug release is crucial for maintaining therapeutic levels and improving gene therapy efficacy.
  • Existing methods struggle with controlled and prolonged delivery of nucleic acid therapeutics.

Purpose of the Study:

  • To develop a controlled-release nanoparticle (NP) system for enhanced gene therapy.
  • To create a polymeric NP system using biodegradable polymers for sustained nucleic acid delivery.
  • To improve gene therapy efficacy through prolonged release of gene payloads for long-term expression or silencing.

Main Methods:

  • Synthesized a polymeric NP system via self-assembly using biodegradable and biocompatible polymers.
  • Encapsulated cationic polymer/nucleic acid complexes within the NPs to act as a drug depot.
  • Characterized physicochemical properties (morphology, size, zeta potential, encapsulation efficiency) and biological properties (release profile, cytotoxicity, transfection efficacy in Hek 293 cells) using pGFP as a reporter gene.

Main Results:

  • The NP system demonstrated excellent cellular biocompatibility and gene delivery efficacy.
  • Sustained release of the pGFP payload was observed over an 8-day period.
  • NP-mediated sustained release of pGFP resulted in enhanced GFP expression over time.

Conclusions:

  • The developed polymeric NP system provides safe and sustained release of gene payloads.
  • This NP-mediated gene delivery system shows potential for various nucleic acid-based therapeutics.
  • The technology has applications in both fundamental biological research and clinical translation of gene therapy.