“An outstanding, creative, dedicated scientist, and a people oriented, reliable, inspiring colleague and leader.”
About
Goal: Entrepreneurial development of innovative medicine to benefit patients and…
Activity
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Dear friends and colleagues, after 19 years with Wyeth and Pfizer collectively, I've decided to leave Pfizer to pursue other opportunities. I'd like…
Dear friends and colleagues, after 19 years with Wyeth and Pfizer collectively, I've decided to leave Pfizer to pursue other opportunities. I'd like…
Liked by Gene Liau
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Esco Lifesciences is excited to announce the acquisition of our long standing distribution partner in South Korea. We will now have a direct sales…
Esco Lifesciences is excited to announce the acquisition of our long standing distribution partner in South Korea. We will now have a direct sales…
Liked by Gene Liau
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Incredibly thrilled to be awarded BBSRC ALERT grant to acquire #cryoEM in Newcastle. This is all down to superb teamwork Jon Marles-Wright, Pohl E.…
Incredibly thrilled to be awarded BBSRC ALERT grant to acquire #cryoEM in Newcastle. This is all down to superb teamwork Jon Marles-Wright, Pohl E.…
Liked by Gene Liau
Experience
Education
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Vanderbilt University
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Activities and Societies: Fencing Club
PhD candidate in the Biochemistry Department. Research in chromatin structure, vitamin A-mediated gene regulation.
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Activities and Societies: Alpha Phi Omega, Fencing
Volunteer Experience
Projects
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Potential Therapeutic Treatment of Friedrich’s Ataxia Using Highly Specific Engineered Meganucleases
- Present
Precision BioSciences, Durham, NC, USA
Friedreich's ataxia (FRDA) is a recessive genetic disorder that results in progressive neuromuscular deterioration. The most common causative genetic alteration of this disorder is an expansion of a trinucleotide (GAA/TTC) repeat within the first intron of the Frataxin (FXN) gene. This triplet expansion causes transcriptional defects, resulting in low FXN mRNA and protein levels. In patients, the correlation between number of repeats and severity of the…Precision BioSciences, Durham, NC, USA
Friedreich's ataxia (FRDA) is a recessive genetic disorder that results in progressive neuromuscular deterioration. The most common causative genetic alteration of this disorder is an expansion of a trinucleotide (GAA/TTC) repeat within the first intron of the Frataxin (FXN) gene. This triplet expansion causes transcriptional defects, resulting in low FXN mRNA and protein levels. In patients, the correlation between number of repeats and severity of the disease, age of onset and cardiomyopathy indicates that the repeat expansion is the primary cause of FRDA, thus making it a potential therapeutic target. We are investigating the potential use of our engineered meganuclease technology to precisely remove these trinucleotide repeats as a therapeutic treatment for FRDA. We have designed a pair of meganucleases that introduce double-strand breaks on either side of the FXN trinucleotide repeat region, generating compatible 3’ overhangs that can be repaired by direct ligation, resulting in precise excision of the intervening region. Introduction of this pair of meganucleases into FRDA patient fibroblasts in cell culture results in the successful deletion of the causative repeat region in greater than 15% of the transfected cells. This removal of the FRDA repeat region has been confirmed using both digital PCR-based analytics and deep sequencing. Additional analysis of Frataxin mRNA production, Frataxin protein levels, and cellular metabolism will further demonstrate the utility of the repeat excision approach for FRDA gene therapy.
Organizations
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American Society of Gene and Cell Therapy
Member: Translational Science Board
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We are hiring a Director for the Carolina Institute for Neurodevelopmental Disorders. This is a highly prestigious position and a great opportunity…
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This week I had the pleasure of welcoming Brad Loncar to Stoke’s lab! Thank you @BiotechTV for the conversation about our approach to upregulating…
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As we advance our clinical-stage lead program and broader pipeline, we're thrilled to announce key enhancements to our leadership team: 👨🔬 Robert…
As we advance our clinical-stage lead program and broader pipeline, we're thrilled to announce key enhancements to our leadership team: 👨🔬 Robert…
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I am proud to be serving as a US national judge for the EY Entrepreneur of the Year - US Overall competition. I am honored to participate in this…
I am proud to be serving as a US national judge for the EY Entrepreneur of the Year - US Overall competition. I am honored to participate in this…
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