KIF1A.ORG Community,

We’re excited to introduce one of our newest partners, the Bařinka Lab at Institute of Biotechnology of the Czech Academy of Sciences in the Czech Republic! Our partnership will enable the Bařinka Lab to collaborate with HNF Pharma Inc. and The Jackson Laboratory to study a potential drug therapy for KIF1A Associated Neurological Disorder. 

Cyril Bařinka earned his PhD at the Institute of Organic Chemistry & Biochemistry in Prague, and spent five years at the NIH National Cancer Institute before starting his own lab in 2010. Dr. Bařinka is known for his expertise in the biochemistry and structure-function of the histone deacetylase (HDAC) enzyme gene family. Our partnership will explore HDAC6 inhibition as a potential therapeutic for KAND. How will we know if HDAC6 inhibition has an effect on KAND? This project leverages Dr. Bařinka’s expertise and assay technology, which enables scientists to measure the activity and potential effectiveness of HDAC6 inhibition in a mouse model of KAND. 

Now, take a step back with me to 2017. KIF1A.ORG was just founded and after consulting with leading rare disease experts, one of our first calls to action became clear: #WeNeedAMouse. Here’s why:

From the PBS Ken Burns documentary The Gene: An Intimate History

With this community’s advocacy, our friends at The Jackson Laboratory, the leading experts in mouse modeling, joined our mission. We now have multiple mouse models in our community-driven Tools for Development that are accessible to any researcher or biotech working on treatments for KAND. 

This collaborative partnership is our strategy in action to urgently discover treatments for KAND. KIF1A.ORG creates robust development tools and gathers the best and brightest in the field to work relentlessly on bringing treatment to this generation of people affected by KAND. 

Thank you to every person in this community for making this rapid progress toward treatment possible. Learn more about this and other projects on our Funded Projects page, and donate today to accelerate our urgent mission. 

Relentlessly,

Kathryn Atchley
President, KIF1A.ORG

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