Mesoangioblast delivery of miniagrin ameliorates murine model of merosin-deficient congenital muscular dystrophy type 1A

Skeletal Muscle
Teuta DomiStefano Carlo Previtali

Abstract

Merosin-deficient congenital muscular dystrophy type-1A (MDC1A) is characterized by progressive muscular dystrophy and dysmyelinating neuropathy caused by mutations of the α2 chain of laminin-211, the predominant laminin isoform of muscles and nerves. MDC1A has no available treatment so far, although preclinical studies showed amelioration of the disease by the overexpression of miniagrin (MAG). MAG reconnects orphan laminin-211 receptors to other laminin isoforms available in the extracellular matrix of MDC1A mice. Mesoangioblasts (MABs) are vessel-associated progenitors that can form the skeletal muscle and have been shown to restore defective protein levels and motor skills in animal models of muscular dystrophies. As gene therapy in humans still presents challenging technical issues and limitations, we engineered MABs to overexpress MAG to treat MDC1A mouse models, thus combining cell to gene therapy. MABs synthesize and secrete only negligible amount of laminin-211 either in vitro or in vivo. MABs engineered to deliver MAG and injected in muscles of MDC1A mice showed amelioration of muscle histology, increased expression of laminin receptors in muscle, and attenuated deterioration of motor performances. MABs did not enter ...Continue Reading

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Citations

Nov 2, 2017·Bioscience Reports·Jaitip TipaneeMarinee K Chuah
Sep 19, 2017·Human Gene Therapy·Jaitip TipaneeMarinee K Chuah
May 28, 2020·Frontiers in Molecular Neuroscience·Kinga I Gawlik, Madeleine Durbeej
May 12, 2020·Frontiers in Molecular Neuroscience·Stefano Carlo Previtali, Alberto Andrea Zambon
Aug 20, 2021·Frontiers in Genetics·Olivier BoyerUNKNOWN Study Group

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Methods Mentioned

BETA
transgenic
protein assay
transfection
PCR

Software Mentioned

QWin
GraphPad Prism
ImageJ
Scion Image

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