TUBB4A-shRNA program

Synaptix Biotherapeutics, in collaboration with the Children's Hospital of Philadelphia (CHOP), is developing a gene-silencing program utilizing short hairpin RNA (shRNA) to treat TUBB4A-related leukodystrophy. This rare neurodegenerative condition, which includes Hypomyelination with Atrophy of the Basal ganglia and Cerebellum (H-ABC), is caused by mutations in the TUBB4A gene that lead to the production of toxic tubulin beta-4A protein. The shRNA modality is designed to specifically target and knockdown the expression of the TUBB4A gene, thereby reducing the levels of the pathogenic protein and mitigating its disruptive effects on oligodendrocyte function and myelin maintenance. Currently in the preclinical discovery stage, this program represents a second-generation approach following the company's lead antisense oligonucleotide candidate, SYB-001.

Other names

Synaptix TUBB4A-shRNA programDiscovery program (shRNAs)Synaptix Biotherapeutics-TUBB4A-shRNA-LeukodystrophyTUBB4A gene-silencing shRNATUBB-4A gene-silencing shRNATUBB 4A gene-silencing shRNA