THERAPEUTIC STRATEGIES

Our team is currently actively involved in developing innovative therapeutic solutions that aims to provide treatment options for genetic movement disorders in certain eligible cases. 

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Drug repurposing, which aims to identify new therapeutic uses for existing medications, is an emerging strategy for accelerating treatment development. Our lab collects biological samples such as blood or saliva from patients and performs RNA sequencing to characterize disease-associated gene expression patterns. These molecular signatures are then compared against publicly available datasets and prior studies to identify existing drugs that attempts to normalize the affected pathways. Because these compounds have already undergone extensive safety and clinical evaluation, this approach can significantly reduce the time and cost associated with developing entirely new therapies. We are currently investigating drug repurposing strategies for KMT2B mutations, which causes dystonic movement disorders.

Antisense oligonucleotides (ASOs) are tiny fragments of single stranded DNA or RNA that binds to a targeted mRNA molecule to regulate gene and protein expression. ASO therapy has been very effective in treating movement disorder cases with rare gain-of-function mutations in certain genes. Our current research is exploring the viability of utilizing ASO therapy in the treatment of movement disorder due to gene mutations in SPTAN1, HNRNPH2, ADCY5,  MAPK8IP3, and FBOXO31 among many others.