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Gene Therapy

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...

Conservative Site-specific Recombination and Phase Variation

Conservative Site-specific Recombination and Phase Variation

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...

In-vitro Mutagenesis

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

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Related Experiment Video

Updated: Jun 12, 2026

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun

Published on: October 24, 2014

Tissue-specific gene delivery approaches.

Sarah S Nasr^1,2, Yahya Cheema¹, Alexa Stern¹

¹Fischell Department of Bioengineering University of Maryland College Park Maryland USA.

Bioengineering & Translational Medicine

|June 11, 2026

Summary

This summary is machine-generated.

Developing efficient gene delivery systems is crucial for genetic therapies. This review explores strategies for engineering adeno-associated viruses, lipid nanoparticles, and polymeric nanoparticles to target specific organs and improve therapeutic outcomes.

Keywords:

adeno‐associated virus gene delivery nanoparticles organ targeting protein corona

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Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

Related Experiment Videos

Last Updated: Jun 12, 2026

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun

Published on: October 24, 2014

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Preparation of rAAV9 to Overexpress or Knockdown Genes in Mouse Hearts

Published on: December 17, 2016

Area of Science:

Biomedical Engineering
Nanotechnology
Gene Therapy

Background:

Efficient delivery of genetic material to target organs is essential for successful gene therapy.
Gene delivery systems face biological barriers, leading to off-target accumulation and potential side effects.

Purpose of the Study:

To review strategies for engineering gene delivery systems for tissue-specific targeting.
To discuss advancements in adeno-associated viruses, lipid nanoparticles, and polymeric nanoparticles for gene delivery.

Main Methods:

Focus on engineering strategies for adeno-associated viruses (AAVs), lipid nanoparticles (LNPs), and polymeric nanoparticles (PNPs).
Discuss rational design and high-throughput screening approaches for discovering novel gene vectors.
Examine nano-bio interactions to understand vector fate and optimize organ targeting.

Main Results:

Engineered AAV variants, LNPs, and PNPs show promise for enhanced tissue-specific gene delivery.
Structure-function relationships are key to determining the biodistribution and efficacy of gene delivery systems.
High-throughput screening accelerates the discovery of efficient and safe gene vectors.

Conclusions:

Strategic engineering of gene delivery vectors is vital for overcoming biological barriers and achieving organ-specific gene therapy.
Advancements in nanoparticle and viral vector design offer improved targeting capabilities.
Continued research into nano-bio interactions and screening methods will drive the development of next-generation genetic therapies.