Influence of Immune Responses in Gene/Stem Cell Therapies for Muscular Dystrophies

Author:

Farini Andrea1ORCID,Sitzia Clementina1,Erratico Silvia2,Meregalli Mirella1ORCID,Torrente Yvan1

Affiliation:

1. Laboratorio Cellule Staminali, Dipartimento di Fisiopatologia Medico-Chirurgica e dei Trapianti, Università degli Studi di Milano, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Centro Dino Ferrari, Via F. Sforza 35, 20122 Milano, Italy

2. Ystem s.r.l., Via Podgora 7, 20122 Milano, Italy

Abstract

Muscular dystrophies (MDs) are a heterogeneous group of diseases, caused by mutations in different components of sarcolemma, extracellular matrix, or enzymes. Inflammation and innate or adaptive immune response activation are prominent features of MDs. Various therapies under development are directed toward rescuing the dystrophic muscle damage using gene transfer or cell therapy. Here we discussed current knowledge about involvement of immune system responses to experimental therapies in MDs.

Funder

Associazione La Nostra Famiglia Fondo DMD Gli Amici di Emanuele

Publisher

Hindawi Limited

Subject

General Immunology and Microbiology,General Biochemistry, Genetics and Molecular Biology,General Medicine

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