“Engineering Poison Exons for Allele-Specific Silencing of the Huntington’s Disease Gene HTT” ASHG Platform Presentation

Camelle Catamura

ASHG Abstract Presented by Camelle Catamura, University of California, Berkeley.

The following is a significant abstract released on Friday, November 3, during the Annual American Association of Human Genetics meeting (ASHG 2023), being held in Washington, DC, November 1-5.

In the ASHG abstract, Catamura and her colleagues wrote the following:

"Repeat expansion diseases that follow a dominant negative inheritance pattern, such as Huntington’s disease, present a great challenge for gene therapies. The most appealing therapeutic strategy requires allele-specific silencing of the disease allele while leaving the wild-type allele intact. Because some amount of repeat sequence is present in both alleles, selective CRISPR targeting of the mutant allele is profoundly challenging."

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