Bridges to Industry Program


Wednesday, October 2, 2019, 5:30pm to 7:30pm


HMS, Armenise 108 (210 Longwood Ave, Boston)
Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
Jinkuk Kim, Yu-Han Huang, Timothy Yu, and Peter Park
More than 6,000 individuals in the US alone suffer from frontotemporal degeneration (FTD) due to the insufficient expression of a gene called GRN, which encodes the progranulin protein. For these patients, progranulin augmentation therapy can address the root cause of the disease. Such therapy also has the potential to benefit a much larger population of patients with Alzheimer's disease or Parkinson's disease through enhancement of lysosomal function and modulation of immune activation.
While no such therapy is available currently, the recent remarkable success of Spinraza/nusinersen for a fatal neurological condition called spinal muscular atrophy has clearly demonstrated that antisense sense oligonucleotide (ASO)-based therapy can be highly effective for boosting the expression of relevant genes in CNS diseases.

Based on our computational analysis on the mechanism of gene expression, we have designed ASO drugs that can upregulate GRN expression by improving the efficiency of the gene expression process. We have shown that the drugs substantially elevate the progranulin protein and mitigate FTD-associated phenotypes in multiple human cell lines in a dose-dependent manner.

In a related project, we have collaborated with Dr. Tim Yu at Boston Children’s Hospital in developing a patient-customized ASO drug for a young girl with Batten disease, a fatal neurodegenerative disease. An n-of-1 clinical trial with the drug has shown a consistent and substantial reduction in seizure. Our expertise in gene regulation and ASO therapeutics is instrumental in successfully carrying out this research.
Parking and reimbursement for child care are available upon request. Please contact Andrea Chen ( if you have any question or if you have dietary restrictions or allergies.
To get to the Armenise building please use the entrance on the left side of the quad (when you face it). If the door is locked please call (617)500-2457 and someone will come to open it for you.