CRD-ATXN7

CRD-ATXN7 is an experimental CRISPR-based gene editing therapy under development by Cure Rare Disease for the treatment of Spinocerebellar ataxia type 7 (SCA7). SCA7 is a rare, autosomal dominant neurodegenerative disorder caused by a polyglutamine (CAG) repeat expansion in the ATXN7 gene, which leads to the production of a toxic, misfolded ataxin-7 protein. This toxic protein accumulates in neurons, particularly in the cerebellum and retina, causing progressive motor impairment and vision loss. The CRD-ATXN7 program utilizes CRISPR/Cas9 technology to specifically target the ATXN7 gene, aiming to either excise the repeat expansion or silence the mutant allele to reduce the expression of the toxic protein. By lowering the levels of mutant ataxin-7, the therapy seeks to preserve neuronal function and halt the progression of the disease. The program is currently in the preclinical stage of development.

Other names

ATXN7 CRISPR therapyATXN-7 CRISPR therapyATXN 7 CRISPR therapyCure Rare Disease SCA7 gene therapy