The goal of
gene therapy is to restore the missing
enzyme by introducing the correct genetic code so proper enzyme production can occur. The correct genetic code may be delivered in various ways but the most common approach uses a viral vector. The non-disease-causing virus is genetically altered to contain the missing genetic code and is used as a vehicle to introduce the correct genetic information and thereby begin proper enzyme production.
Gene therapy clinical trials for Tay-Sachs and Sandhoff could begin as early as March 2013. Learn more about the Tay-Sachs Gene Therapy Consortium and their breakthrough efforts to cure Tay-Sachs and Sandhoff at www.tsgtconsortium.com.
Since the early 1980s over 20 children have received experimental Canavan gene therapy. Many parents feel that gene therapy helped their child. The results have been submitted for publication.
A different type of gene therapy for Canavan has recently shown very exciting results in Canavan mice. NTSAD is delighted to partner with the Canavan Research Foundation to fund this project. Visit our Research Initiative page to learn more about this project and other promising efforts funded by NTSAD.
In 2008 the world’s first gene therapy for inherited blindness showed sight improvement. The findings are a landmark for gene therapy technology.
Gene therapy has a lot of promise to cure but the Food and Drug Administration (FDA) has not yet approved any gene therapy for market. Current gene therapy is still considered experimental. Some of the challenges to develop a safe and effective gene therapy include:
• difficulty of creating effective vectors for gene delivery to the brain;
• need to introduce the gene into a large number of cells to be effective;
• challenge of inserting the genes into the appropriate brain cells; and
• potential for an oncogenic (cancer) event to occur as a result of the insertion of the gene into the cell’s chromosomes.
Learn more:
http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml
http://learn.genetics.utah.edu/content/tech/genetherapy/
