#ScienceSaturday posts share relevant and exciting scientific news with the KAND community. This project is a collaboration between KIF1A.ORG’s Research Engagement Team Leader Alejandro Doval, President Kathryn Atchley, Science Communication Volunteer Aileen Lam and Chief Science Officer Dr. Dominique Lessard. Send news suggestions to our team at impact@kif1a.org.

Advance Science This Giving Season!

KIF1A.ORG is getting an early start to #GivingTuesday this year! We fund life-changing and life-saving research to find treatments and cures for our superheroes. With today’s scientific advancements and our relentless community, it’s not a matter of IF we will find a cure, it’s a matter of WHEN. Help us reach our #GivingTuesday fundraising goal by donating on Facebook or at kif1a.org/donate.


KIF1A-Related Research

Modulating intracellular pathways to improve non-viral delivery of RNA therapeutics

RNA therapeutics continue to be a hot topic of discussion for genetic rare diseases like KAND. With advancements in RNA therapeutic options such as antisense oligonucleotides (more commonly referred to as ASOs) and RNA-interference technology, there is great potential for these therapies to treat all types of diseases and disorders. Like any developed treatment, one must consider how to effectively deliver it to a patient. In other words, once a treatment is identified how will it be delivered exactly where it needs to be and when it needs to be in the body to do its job?

The review paper we are featuring today walks us through how the scientific community has attempted to modify certain functions in human cells to increase the efficacy of RNA therapeutic delivery. Specifically, this paper focuses on how we can work around cellular obstacles, like processes in our cells that are designed to block RNA delivery, by introducing small molecules or genetic modulators to change how cells respond to the introduction of an RNA therapeutic. Many different types of cellular functions are discussed in this paper, so we including some functions that we know KIF1A to be involved with, such as the transport of cellular cargo. Additionally, a relationship was identified between KIF1A (as well as other members of the kinesin superfamily of proteins) and productive delivery of ASOs. Want to learn more about RNA therapeutics? Check out the video below featuring Nobel Prize winner Dr. Jennifer Doudna!

Rare Roundup

Dancing molecules’ successfully repair severe spinal cord injuries

This week, we are featuring some impressive news out of Northwestern University regarding the treatment of spinal cord injury! Recently, a research team out of Northwestern was able to create a synthetic substance (lab made; not naturally occurring) that helps promote the repair of spinal cord tissue after injury in a mouse model of paralyzing human spinal cord injury. In other words, when this substance was administered to mice with spinal cord injuries, they began to “heal” in many ways and even regained movement capabilities, as seen in the video below! How is this possible? This new therapy works by communicating with injured/damaged cells and gives them signals and support to begin to heal. Naturally, one of our follow up questions at KIF1A.ORG is “could this be applied to a KAND mouse model and show improved motor capabilities?”. Answering this question would involve answering a host of other questions such as “how could this apply to a model system mimicking neurodegeneration considering that neurodegenerative tissue damage happens in a very different way that a spinal cord injury?” or “could this substance still help the spinal cord tissue if the way in which the tissue is damaged is different?”. While we don’t know the answers to these questions right now, this is a therapeutic development that we will certainly be keeping our eyes on!

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