Leading the Fight to treatand cure Tay-Sachs, Canavan and related diseases
Stop-Codon Read Through Technology is an emerging field that could potentially treat genetic diseases caused by stop codons. Stop codons are genetic phrases that indicate the end of the
gene, like the period at the end of a sentence. Some genetic diseases are caused by mutations which place the period before the end of the gene resulting in enzymes that do not work properly. Stop Codon Read Through Technology uses small molecules that cross the blood brain barrier into the
central nervous system and induce the cell to ignore the stop codon resulting in a properly functioning
There are currently clinical trials in a variety of diseases based on this approach, including cystic fibrosis and Duchenne Muscular Dystrophy.
This approach is limited to affected people with this particular type of
mutation. Scientists are still learning how to effectively use this technology to both read through disease causing stop codons and ignore stop codons in the proper place.
National Tay-Sachs & Allied Diseases Association2001 Beacon StreetSuite 204Boston, MA 02135
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At NTSAD, we are making a difference. Every day. Helping families and individuals find their way and providing hope by supporting research on many levels. All of this takes compassion and determination.
There is no time to waste, and families need our help. Those who are fighting today and those who will seek our help tomorrow.
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