Technology A Micro-RNA Approach for the Treatment of Spinocerebellar Ataxia Type 6 (SCA6) Spinocerebellar ataxia type 6 (SCA6) is a rare, dominantly inherited disease that causes Purkinje cell degradation and results in: loss of coordination, tremors, and uncoordinated muscle movement. Current therapies only…
News Genes Reveal Details of ‘the Crime,’ Pathway to Develop a Novel Treatment for Neurodegenerative Disease University of Chicago researcher Christopher Gomez is developing a gene therapy to treat a rare, neurodegenerative disease – and conducting a clinical trial, backed by Pfizer and Biogen, to establish...
Technology Gene Splice-Switching Technology for Treating CACNA1A-Associated Neurological Diseases This technology regulates the expression of the CACNA1A gene, which encodes a neuronal calcium channel subunit, by redirecting alternative splicing to treat related neurological diseases. This approach can potentially correct…
Technology Engineered Mutator tRNAs For Correcting Missense Genetic Defects And Inducing Cancer Cell Death Engineered tRNAs with altered anticodons and chemical modifications deliver the correct amino acid at mutated codons to restore protein function in genetic diseases or induce lethal mistranslation in cancer cells,…
