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Related Concept Videos

Microorganisms in Medicine and Therapeutics01:29

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.
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...
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...
What is Genetic Engineering?00:49

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CRISPR01:59

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...

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Preparation and In Vitro Characterization of Magnetized miR-modified Endothelial Cells
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Published on: May 2, 2017

Nanoengineering Systems for Gene Therapy: Mechanisms, Modalities, and Future Directions.

Raheem Mais1, Ayush Kumar1, Armand Ahmetaj1

  • 1Department of Bioengineering, New York Institute of Technology, New York, NY 11568, USA.

International Journal of Molecular Sciences
|July 15, 2026
PubMed
Summary

Nanotechnology enhances gene therapy and genome editing by improving delivery, stability, and safety. This approach, particularly with CRISPR-Cas systems, offers new treatments for genetic disorders and diseases like cancer.

Keywords:
CRISPR-Cas systemsgene therapygenetic disordersgenome engineeringnanomaterialsnanotechnologytargeted delivery

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Area of Science:

  • Biotechnology
  • Materials Science
  • Genetics

Background:

  • Gene therapy and genome editing face challenges like inefficient delivery and off-target effects.
  • Nanotechnology offers novel delivery strategies to improve precision, efficiency, and safety of genetic modifications.

Purpose of the Study:

  • To review the role of nanomaterials in overcoming barriers in gene therapy and genome editing.
  • To highlight the synergy between nanomaterials and genome engineering tools like CRISPR-Cas systems.
  • To discuss next-generation editing technologies and their delivery challenges.

Main Methods:

  • Review of current literature on nanotechnology applications in gene therapy and genome editing.
  • Emphasis on the integration of nanomaterials with CRISPR-Cas and other advanced editing systems.
  • Analysis of therapeutic potential and delivery challenges of various genome engineering modalities.

Main Results:

  • Nanomaterials improve the stability, intracellular delivery, and specificity of genome-editing systems.
  • Combined use of nanomaterials with CRISPR-Cas enhances editing efficacy and safety.
  • Nanocarriers offer controlled release, protection from degradation, and improved biocompatibility.

Conclusions:

  • Nanotechnology is crucial for advancing gene therapy and genome editing, enabling effective treatments for genetic and acquired diseases.
  • Continued convergence of nanotechnology and genome engineering paves the way for personalized medicine.
  • Translational challenges such as immunotoxicity and manufacturing require further attention.