skip to Main Content

OPTIMIZATION OF VIRAL VECTORS FOR THE EFFECTIVE GENE DELIVERY TO MUSCLE TISSUE CELLS

Directed evolution of adeno-associated virus (AAV) capsids for effective gene delivery to the muscle fibro-adipogenic progenitors (FAPS)

PROJECT DESCRIPTION

In order to administer any gene therapy, it is necessary to make sure it effectively reaches the target cell. This is achieved by means of vectors that transport the therapy specifically to the cells we want to treat. Collagen VI-related muscular dystrophies (COL6-RD) are caused by mutations in one of the collagen VI genes, a protein produced by a specific type of cells present in the muscle (fibro-adipogenic progenitors). Today, although there are vectors used in the administration of gene therapies to certain cells, there is none that offers the efficiency or specificity necessary to reach this specific type of cell. The objective of this project is to design, based on currently available vectors, a new variant that allows any of the therapies being developed in other projects to be delivered efficiently to these cells, to restore the normal expression of collagen VI.

Project time frame

2021-2023

Funding

50.000,00 US Dollars, co-financed by Fundacion Noelia and Cure CMD.

Contributions made to date by Fundacion Noelia:
December 1, 2021: 12.500,00$  (Proof of transfer)

Contributions pending to be made by the Noelia Foundation:

December 1, 2022: 12.500,00$

Location:
NINDS National Institute of Neurological Disease and Stroke, Bethesda, Maryland EEUU

Researchers:

  • Carsten G. Bönnemann, MD (Principal Investigator)
  • Veronique Bolduc (research fellow)
  • Astrid Brull-Canagueral (postdoctoral fellow)
  • Fady Guirguis (graduate student)

 

Collaboration agreement.

Back To Top