What if milk could be used to deliver genetic therapies to the brain of SYNGAP1 patients? The first step is to see if natural nanoparticles (“exosomes”) in milk can improve SynGAP expression!
The SynGAP Research Fund (SRF) announces a new research grant award to the Zempleni Lab in the College of Education and Human Sciences at the University of Nebraska-Lincoln. The lab has received $100,000 to assess whether milk exosomes can be used as an effective delivery mechanism for future therapies for SYNGAP1.
SYNGAP1-related intellectual disability (ICD-10: F78.A1) is a rare genetic disorder caused by a variation on the SYNGAP1 gene, with nearly 1,000 diagnosed patients accounted for globally as of December 2021. It leads to several neurological issues in patients, including intellectual disability, epilepsy, autism, sleep challenges, gastro-intestinal and feeding problems, hypotonia (low muscle tone), apraxia (delayed/no speech), impulsivity and aggression.
Prof. Zempleni and his team are pioneering research on how to use natural nanoparticles (“exosomes”) to regulate gene expression. This cutting edge approach–already an area of investment by large pharmaceutical companies–has the potential to overcome the challenge of delivering therapeutics into brain cells. The ability to reliably deliver oral formulations that can cross the blood-brain barrier is exciting for a broad spectrum of disorders, including SYNGAP1.
Prof. Zempleni says, “Milk exosomes afford an opportunity to deliver therapeutics to diseased tissues. The delivery of SYNGAP1 mRNA to the brains of SYNGAP1 patients is a feasible application. My laboratory of 12 milk exosomes experts is uniquely qualified to deliver on the technology’s promise.”
The project will assess whether milk exosomes can effectively deliver gene coding information to the brain in SynGAP deficient mice, thereby increasing protein expression to normal levels. Previously published research by SRF grantee Dr. Gavin Rumbaugh has demonstrated that restoring SynGAP levels in mice, even in adulthood, improves measures of brain function and behavior.
Dr. Hans Schlecht, Research Director of SRF says, “Dr. Zempleni is a world class researcher who is pioneering a technique to deliver tailored payloads in little packages called exosomes. It is beyond fantastic to consider not only delivering SYNGAP1 mRNA to neurons to make up the balance of SYNGAP1 that our kids’ cells are not making but also, down-the-road, a payload that could edit their genes back to normal.”
Michael Graglia, Managing Director of SRF says, “SRF is entrusted with funds from families to support science with an eye to improving the future of our loved ones. Selecting those projects is hard, even with our remarkable Scientific Advisory Board. Prof. Zempleni’s project, however, was an easy choice. We are excited to support this work and grateful to have such a remarkable researcher working on SYNGAP1.”
The grant was made possible by fundraising efforts supported by families affected by SYNGAP1, including matching donations from SRF board members, a pop-up cafe in Estonia, a gala in New Jersey, a dice stacking challenge in the UK, and the #HopeForHadley effort.
To learn more about this project join the SRF Webinar with Prof. Zempleni on March 3, 2022 at 9 AM Pacific, register online at Syngap.Fund/Zempleni.
Source – SynGAP Research Fund