State of NH Clinical Genetics

 

Genetic Therapy

 

 


What is Gene Therapy?

Gene therapy is a type of treatment which changes a person's genes to avoid illness. It is a means to cure disease by making corrections or replacing abnormal genes with healthy, normal genes or genetic components. This treatment is only beginning to surface in the scientific community and is surrounded by optimism and controversy. Scientific trials are being conducted on the use of gene therapy to treat such diseases as Cystic Fibrosis and Muscular Dystropy.

 



Types of Gene Therapy

How is Gene Therapy Done?

Gene Therapy Links:


 


 

Types of Gene Therapy

Somatic Gene Therapy:

This therapy changes genes within a diseased person. The changes in these genes are not passed on to the children the treated person creates. The majority of research in gene therapy involves manipulation of somatic genes.

 

Germline Gene Therapy:

This therapy is similar to somatic gene therapy, but the changes are made to the germline cells. Modified germline cells, sperm and eggs, pass the changes onto the next generation. These changes alter the expression of genes in future generations. The controversial nature of the possibility of creating a "Super Race" has caused most governments to ban research in germline gene therapy.

 


How is Gene Therapy Done?

To get the gene therapy agent to work directly on a gene, a vector is used. A vector surrounds the gene therapy agent and delivers it directly to the cell. Vectors can be made using viruses, or constructed in a lab using DNA, proteins, or lipids. The vector allows the gene therapy agent to enter the cell and make modifications.

 


Gene Therapy Links:

 

Institute for Human Gene Therapy

Comprehensive overview of gene therapy including research studies, responses to media, and general information.

 

Introduction to Gene Therapy

Education site including links to other research institutes, background information, and future prospects in gene therapy.

 

National Reference Center for Bioethics Literature

The Joseph and Rose Kennedy Institute of Ethics

Human Gene Therapy

Overview of gene therapy containing history, techniques, ethical issues, and policy statements.

 

UCLA Program on Medicine, Technology, and Society

Human Germline Engineering

Presentation of issues surrounding the controversial topic of human germline gene therapy.

 

 
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