The LDN thanks the Batten Disease Support and Research Association (BDSRA) for pointing the way to the following information.
CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is one of the new technologies some researchers are using to explore possible genetic treatment of rare genetic disease. CRISPR is a tool for precision gene editing.
This episode of NOVA, which originally was broadcast by PBS in the United States on May 23, 2018, describes how some researchers are using CRISPR. You may wish to skip to minute 32 to see how gene therapy may help the adrenoleukodystrophy (“ALD”) community.
For an even shorter overview of how CRISPR works, check out this 1.5-minute NOVA Wonders video By Michael Rivera.
CRISPR is one of the topics that will be addressed by expert speakers at “The Next Revolution: Genome Engineering 2018” conference at the University of Minnesota in Minneapolis, July 19-21, 2018. For more details visit our Calendar page, where events are shown in chronological order.