Methods to treat Danon disease


Dr. Song at the University of Colorado has developed a new gene therapy to treat Danon disease. The new method includes genetic correction of LAMP-2 mutations and targeting defective LAMP-2B-mediated autophagy.  Results show that all cellular functions were restored in gene corrected hiPSC-CM from Danon patients. 


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For Information, Contact:
Doreen Molk
University of Colorado
Kunhua Song
Congwu Chi
Yingqiong Cao
Disease Areas:
Regenerative Medicine
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