{"id":19417,"date":"2022-04-04T10:38:53","date_gmt":"2022-04-04T10:38:53","guid":{"rendered":"https:\/\/fundacionnoelia.org\/proyectos-de-investigacion\/un-nuevo-sistema-de-administracion-de-acidos-nucleicos-basado-en-nanoparticulas-para-el-tratamiento-de-la-distrofia-muscular-por-deficit-de-col6\/"},"modified":"2022-04-04T11:45:49","modified_gmt":"2022-04-04T11:45:49","slug":"a-new-nanoparticle-based-delivery-system-for-nucleic-acids-to-treat-collagen-vi-cmd","status":"publish","type":"page","link":"https:\/\/fundacionnoelia.org\/en\/research-projects\/a-new-nanoparticle-based-delivery-system-for-nucleic-acids-to-treat-collagen-vi-cmd\/","title":{"rendered":"A new nanoparticle-based delivery system for nucleic acids to treat Collagen VI-CMD"},"content":{"rendered":"[vc_row][vc_column width=”1\/6″][\/vc_column][vc_column width=”4\/6″][vc_empty_space]
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A NEW NANOPARTICLE-BASED DELIVERY SYSTEM FOR NUCLEIC ACIDS TO TREAT COLLAGEN VI-CMD<\/h2>\n[\/vc_column_text][vc_empty_space][vc_column_text]DYSTRO-SMARTY: a non-liposomal nanovesicle platform for delivery of<\/p>\n

nucleic acids to treat Collagen VI-related Congenital Muscular Dystrophy<\/p>\n

PROJECT DESCRIPTION<\/strong><\/p>\n

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.<\/p>\n

Project time frame<\/strong>
\n1 year, 2022<\/p>\n

Funding<\/strong><\/p>\n

97,463.00 $, Project co-financed 50% by Fundaci\u00f3n Noelia and Orphan Disease Center \u201cThe 2021 Million Dollar Bike Ride\u201d<\/p>\n

Contributions made to date by Fundacion Noelia:<\/strong>
\nFebruary, 2022: 43.000,00 $<\/strong>\u00a0<\/strong>(Proof of transfer)<\/a><\/p>\n

Location:<\/strong>
\nSant Joan de D\u00e9u University Hospital, Barcelona, Spain<\/p>\n

Researchers:<\/strong><\/p>\n