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 will perform this research through Leiden University Medical Center in the Netherlands (See their website here)on the use of lncRNA to preserve dystrophin protein for Luka's deletion. The lncRNA study takes one year to complete, and 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 genetic's research scientist in Muscular Dystrophy worldwide.

Cost of study:

Funds raised for the studies will be provided to Leiden University Medical Center in The Netherlands in the form of donation as follow:

Targeted Start Date	March , 01st 2025
Estimated Completion Date	March, 01st 2026
Number of Patients Benefitting	All In-Frame Deletions
∆6-25  Mice Model Cost (Campaign's Goal)	$86,000