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Pioneering Gene Therapies for Nucleotide Repeat Disorders

 

Our Idea: ASRE

Artificial Site-Specific RNA Endonucleases (ASREs) are rationally designed proteins, that when expressed in cells, can target and destroy a disease-causing RNA.

ASREs are highly sequence specific and can be targeted against nucleotide repeats known to cause many diseases including: Huntington's disease, myotonic dystrophy and certain forms of ALS.

 
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“Enzerna's ASRE technology specifically targets disease-causing nucleotide repeats at the RNA level, a specialty that sets it apart from CRISPR/Cas and other DNA modifying technologies ”

— zefEng wang, Phd - Scientific Founder

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