Related Experiment Video

Updated: Jan 14, 2026

In Vivo Gene Delivery into Mouse Mammary Epithelial Cells Through Mammary Intraductal Injection
05:36

In Vivo Gene Delivery into Mouse Mammary Epithelial Cells Through Mammary Intraductal Injection

Published on: February 10, 2023

2.1K

In vivo gene therapy: A strategy for mutations, degenerations, and tumors

Tao Wang1, Mingyang Yu2,3, Ping Liu1

  • 1Department of Hematology, Institute of Hematology, Changhai Hospital, Naval Medical University, Shanghai 200433, China.

Genes & Diseases
|October 27, 2025
PubMed

Insights

In vivo gene therapy shows promise for treating genetic disorders and cancers by utilizing DNA nucleases and delivery vectors. Further development of these components is crucial for future clinical applications.

Area of Science:

  • Biotechnology
  • Genetics
  • Medical Science

Background:

  • Gene mutations, organ degeneration, and cancer pose significant health risks.
  • Gene therapy offers potential cures for genetic disorders, degenerative diseases, and cancers.
  • Ex vivo gene therapy is established for certain conditions, but in vivo gene therapy is still under development.

Purpose of the Study:

  • To review the advancements in DNA nucleases and delivery vectors for in vivo gene therapy.
  • To highlight recent progress in the components essential for in vivo gene therapy.
  • To discuss the potential of in vivo gene therapy for future clinical use.

Main Methods:

  • Review of deoxyribonucleic acid (DNA) nucleases, including zinc finger nucleases, transcription activator-like effector nucleases, and CRISPR-Cas systems.
  • Examination of gene editing tools such as base editors and prime editors.
  • Analysis of various delivery vectors, encompassing viral and non-viral vehicles.

Main Results:

  • Significant progress has been made in the development of DNA nucleases and delivery vectors.
  • The review synthesizes current knowledge on key technologies for in vivo gene therapy.
  • The potential for in vivo gene therapy to become clinically available is discussed.

Conclusions:

  • Advancements in DNA nucleases and delivery vectors are paving the way for in vivo gene therapy.
  • Further development is needed to translate in vivo gene therapy into widespread clinical practice.
  • In vivo gene therapy holds great promise for treating a range of human diseases.
Keywords:
CancersDNA nucleasesDegeneration-related diseasesDelivery vectorsGenetic mutation disordersIn vivo gene therapy

More Related Videos

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium10:18

In Vivo Gene Transfer to the Rabbit Common Carotid Artery Endothelium

Published on: May 6, 2018

9.7K
CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy08:22

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

Published on: March 12, 2018

15.5K

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...
27.3K
In-vitro Mutagenesis01:16

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.
16.0K
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.
928