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2015 ; 5
(ä): 30
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Mesoangioblast delivery of miniagrin ameliorates murine model of
merosin-deficient congenital muscular dystrophy type 1A
#MMPMID26347253
Domi T
; Porrello E
; Velardo D
; Capotondo A
; Biffi A
; Tonlorenzi R
; Amadio S
; Ambrosi A
; Miyagoe-Suzuki Y
; Takeda S
; Ruegg MA
; Previtali SC
Skelet Muscle
2015[]; 5
(ä): 30
PMID26347253
show ga
BACKGROUND: Merosin-deficient congenital muscular dystrophy type-1A (MDC1A) is
characterized by progressive muscular dystrophy and dysmyelinating neuropathy
caused by mutations of the ?2 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. METHODS:
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.
RESULTS: 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 the peripheral nerves, thus did not affect the associated
peripheral neuropathy. CONCLUSIONS: Our study demonstrates the potential efficacy
of combining cell with gene therapy to treat MDC1A.
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