Antisense Oligonucleotide as Substrate Reduction Therapy for Mucopolysaccharidoses type III
Awardee: Manor Yehoshua
Institution: Sheba Medical Center
Grant Amount: $57,645.00
Funding Period: February 1, 2024 - January 31, 2025
Summary:
Our project aimed at finding a much-needed treatment for MPS type III, a condition that currently has limited treatment options. Leveraging the advanced capabilities of RNA-based therapy, specifically Antisense Oligonucleotides (ASOs), we are crafting a method to reduce the buildup of the harmful substance of heparan sulfate in the body and brain that is the hallmark of this illness. This technology has not only proven to be more effective but also more cost-efficient compared to other existing therapies. Moreover, it has demonstrated successful outcomes in both laboratory settings and preliminary human trials. By building on this innovative approach, we aspire to create a treatment that can significantly alleviate the CNS symptoms of all types of MPS III patients, potentially revolutionizing the management of this condition and offering hope for improved quality of life to those affected.