CRISPR-ATXN3

CRISPR-ATXN3 is an experimental CRISPR-based gene editing therapy under development by Cure Rare Disease for the treatment of Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease. SCA3 is a progressive neurodegenerative disorder caused by a CAG repeat expansion in the ATXN3 gene, which leads to the production of a toxic, misfolded ataxin-3 protein. This therapeutic approach utilizes CRISPR technology to specifically target the ATXN3 gene, aiming to either knock down the expression of the mutant allele or correct the underlying genetic defect. By reducing the levels of toxic ataxin-3 protein, the treatment seeks to prevent neuronal loss and slow the progression of motor symptoms. The program is currently in the preclinical stage of development.

Other names

ATXN3 CRISPR gene editing therapyATXN-3 CRISPR gene editing therapyATXN 3 CRISPR gene editing therapySCA3 CRISPR therapySCA-3 CRISPR therapySCA 3 CRISPR therapy