A NEW NANOPARTICLE-BASED DELIVERY SYSTEM FOR NUCLEIC ACIDS TO TREAT COLLAGEN VI-CMD
DYSTRO-SMARTY: a non-liposomal nanovesicle platform for delivery of
nucleic acids to treat Collagen VI-related Congenital Muscular Dystrophy
PROJECT DESCRIPTION
Although nucleic acid (RNA & DNA) -based therapies are being developed at a fast pace for rare diseases, delivering therapeutic amounts of these nucleic acids to the target tissue remains a major hurdle, particularly for muscle diseases. The project aims at testing a validated nanotechnology platform as a valid vector to deliver nucleic acids to target cells from COL6-related Congenital Muscular Dystrophy (COL6-CMD ) patients. The chosen therapeutic agent are antisense oligonucleotides that have been shown to correct a common mutation in collagen VI genes in vitro. This novel therapeutic approach will be evaluated in terms of its properties, distribution, specificity to the target cells and ability to effectively correct collagen VI mutations. This work will facilitate the future translation of this and other potential nucleic acids therapies into new treatments for col6-CMD patients.
Project time frame
1 year, 2022
Funding
97,463.00 $, Project co-financed 50% by Fundación Noelia and Orphan Disease Center “The 2021 Million Dollar Bike Ride”
Contributions made to date by Fundacion Noelia:
February, 2022: 43.000,00 $ (Proof of transfer)
Location:
Sant Joan de Déu University Hospital, Barcelona, Spain
Researchers:
- Cecilia Jimenez-Mallebrera (Principal Investigator)