National Institute of Neurological Disorders and Stroke 5F31NS141619-02

Targeting transcription and translation of the antisense CCCCGG repeat expansion in C9ORF72 ALS/FTD

This NIH-funded project targets the antisense CCCCGG repeat expansion in the C9ORF72 gene linked to ALS/FTD. It aims to identify and validate the transcription start site of the antisense transcript and use CRISPR and antisense oligonucleotides to block and degrade the antisense RNA, potentially offering a therapeutic approach for these neurodegenerative diseases.

Description

The project aims to study transcription of antisense repeat RNA and evaluate therapeutic strategies targeting antisense transcription and translation. It involves locating the transcription start site (TSS) of the antisense transcript, validating it via CRISPR/Cas9 mutagenesis, and using antisense oligonucleotides (ASOs) to degrade antisense repeat RNA. The project focuses on neurodegenerative diseases such as ALS/FTD caused by repeat expansions in the C9ORF72 gene.

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