Next Research Study In Pipeline

The Use of lncRNA to Preserve and Increase the Production of Dystrophin With ∆17-44 Exons Deletion

The research aims to study the use of lncRNA to preserve and increase the production of dystrophin and to create a mice model of the genetic deletion/duplication to test the research's findings in an in-vivo setting.

Dr. Aartsma-Rus (Leiden University Medical Center in the Netherlands See their website here) is currently confirming the use of lncRNA in another study published in the Q4 of 2024, to preserve dystrophin protein for Luka's deletion. This study will benefit exon-skipping studies, rare brain disease research as well as cancer research. 

Dr. Aartsma-Rus of Leiden University Medical Center in the Netherlands is considered to be one of the best translational geneticist researcher in Muscular Dystrophy worldwide.

Cost of study

Leiden University Medical Center in the Netherlands has funded preclinical discovery costs of the study.

Start Date	March , 01st 2025 Started
Targeted Completion Date	March, 01st 2026
Number of Patients Benefitting	All In-Frame Deletions
∆17-44  Pre-clinical Costs	$86,000 Funded by LUMC