The National Centre for Research and Development has decided to award funding for scientific projects in the 15th edition of the LIDER Programme. A total of over PLN 71 million will be distributed to 41 young scientists selected from 392 applicants in this competition. We are pleased to announce that Dr. Anna Kotowska-Zimmer from the Department of Genomic Engineering is among the scientists who have received funding for their research. Dr. Kotowska-Zimmer will investigate the use of artificial miRNAs for allele-selective therapy of cerebellar spinocerebellar ataxias caused by the expansion of CAG repeats.

Spinal cerebellar ataxias are a group of rare genetic neurodegenerative diseases. In the case of ataxias belonging to the polyglutamine diseases, the mutation responsible for their development involves the expansion of CAG repeats in specific genes. This leads to the formation of elongated glutamine chains in proteins, which acquire toxic properties. Despite many years of research, these diseases remain incurable and ultimately lead to the death of patients.

The goal of the project is to develop a versatile molecule that will allele-selectively downregulate mutant proteins responsible for the development of spinocerebellar ataxias. Positive results from this project could pave the way for further research aimed at implementing an effective and safe therapy for patients suffering from spinocerebellar ataxias caused by CAG repeat expansion.

Congratulations and best wishes for continued success!