Related Experiment Video
Updated: Aug 9, 2026
21:55
Engineering and Evolution of Synthetic Adeno-Associated Virus (AAV) Gene Therapy Vectors via DNA Family Shuffling
Published on: April 2, 2012
Mammalian artificial chromosomes as tools for gene therapy
1Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill 27599-7295, USA. vos@med.unc.edu
Current Opinion in Genetics & Development
|August 5, 1998
Summary
Mammalian artificial chromosomes (MACs) offer promising gene therapy and transgenesis applications. Researchers are exploring genetic elements to overcome size and delivery challenges for future widespread use in treating genetic diseases.
Area of Science:
- Genetics and Molecular Biology
- Gene Therapy
- Epigenetics
Background:
- Mammalian artificial chromosomes (MACs) are advanced tools for gene therapy and animal transgenesis.
- Existing MACs include linear genomic human artificial chromosomes (HACs) and circular mouse artificial episomal chromosomes (MAECs).
Purpose of the Study:
- To discuss the potential of novel genetic cis-elements and epigenetic phenomena for MACs.
- To highlight the future therapeutic applications of MACs once delivery and size constraints are resolved.
Main Methods:
- Development of first-generation linear HACs and circular chimeric MAECs.
- Shuttling of HACs into mouse embryonal stem cells to generate trans-chromosomic mice.
Main Results:
- Successful generation of human trans-chromosomic mice.
- Identification of potential genetic cis-elements and epigenetic phenomena influencing MACs.
Conclusions:
- MACs hold significant therapeutic potential, especially for complex genetic disorders.
- Overcoming size and delivery limitations is key to unlocking the full potential of MACs in medicine.
More Related Videos
Related Concept Videos
01:16In-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.
00:59Gene 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...