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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...

Microorganisms in Medicine and Therapeutics

Microorganisms in Medicine and Therapeutics

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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Related Experiment Video

Updated: Dec 15, 2025

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

Published on: January 15, 2015

Silica-Based Gene Delivery Systems: From Design to Therapeutic Applications.

Ana Maria Carvalho¹, Rosemeyre A Cordeiro¹, Henrique Faneca¹

¹Center for Neuroscience and Cell Biology, University of Coimbra, 3004-504 Coimbra, Portugal.

|July 15, 2020

Summary

Silica-based vectors offer a safer, cost-effective alternative for gene therapy delivery. These versatile nanomaterials show promise for treating genetic diseases and enabling targeted drug release.

Keywords:

gene therapy hybrid silica nanosystems silane chemistry silica-based vectors stimuli-responsive release targeted gene delivery

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Area of Science:

Biomedical Engineering
Nanotechnology
Gene Therapy

Background:

Gene therapy holds promise for treating genetic diseases, but effective gene delivery systems are crucial.
Viral vectors face limitations, driving research into safer, non-viral alternatives.
Silica-based vectors are emerging as promising candidates due to their unique properties.

Purpose of the Study:

To review recent advances in silica-based systems for gene therapy.
To highlight the properties, fabrication, and applications of these novel vectors.
To discuss their potential in various therapeutic areas.

Main Methods:

Review of current literature on silica-based gene delivery systems.
Analysis of fabrication techniques and surface modification strategies.
Evaluation of in vitro and in vivo studies demonstrating therapeutic efficacy.

Main Results:

Silica vectors offer enhanced safety, stability, and cost-effectiveness compared to viral vectors.
Their modifiable structure allows for high loading capacity, genetic material protection, and targeted delivery.
Stimuli-responsive release mechanisms and combined imaging functions are being developed.
Promising results in preclinical studies for cancer and brain therapies.

Conclusions:

Silica-based nanosystems represent a versatile and effective platform for advanced gene therapy.
Further development holds significant potential for treating a wide range of genetic disorders.
These materials offer a promising avenue for next-generation therapeutic strategies.