Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts

Maurilio Sampaolesi; Yvan Torrente; Anna Innocenzi; Rossana Tonlorenzi; Giuseppe D'Antona; M Antonietta Pellegrino; Rita Barresi; Nereo Bresolin; M Gabriella Cusella De Angelis; Kevin P Campbell; Roberto Bottinelli; Giulio Cossu

doi:10.1126/science.1082254

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Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts

Journal article

Peer reviewed

Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts

Maurilio Sampaolesi, Yvan Torrente, Anna Innocenzi, Rossana Tonlorenzi, Giuseppe D'Antona, M Antonietta Pellegrino, Rita Barresi, Nereo Bresolin, M Gabriella Cusella De Angelis, Kevin P Campbell, …

Science (American Association for the Advancement of Science), Vol.301(5632), pp.487-492

07/25/2003

DOI: 10.1126/science.1082254

PMID: 12855815

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Abstract

Preclinical or clinical trials for muscular dystrophies have met with modest success, mainly because of inefficient delivery of viral vectors or donor cells to dystrophic muscles. We report here that intra-arterial delivery of wild-type mesoangioblasts, a class of vessel-associated stem cells, corrects morphologically and functionally the dystrophic phenotype of virtually all downstream muscles in adult immunocompetent alpha-sarcoglycan (alpha-SG) null mice, a model organism for limb-girdle muscular dystrophy. When mesoangioblasts isolated from juvenile dystrophic mice and transduced with a lentiviral vector expressing alpha-SG were injected into the femoral artery of dystrophic mice, they reconstituted skeletal muscle in a manner similar to that seen in wild-type cells. The success of this protocol was mainly due to widespread distribution of donor stem cells through the capillary network, a distinct advantage of this strategy over previous approaches.

Membrane Glycoproteins - metabolism

Cytoskeletal Proteins - genetics

Male

Muscle, Skeletal - metabolism

Mesoderm - cytology

Muscle, Skeletal - cytology

Muscular Dystrophy, Animal - pathology

Endothelium, Vascular - physiology

Blood Vessels - embryology

Stem Cell Transplantation

Transfection

Muscular Dystrophy, Animal - metabolism

Cytoskeletal Proteins - metabolism

Female

Femoral Artery

Lentivirus - genetics

Muscular Dystrophy, Animal - therapy

Cell Differentiation

Dystrophin - metabolism

Muscle Fibers, Skeletal - physiology

Cell Line

Mice, Transgenic

Muscle, Skeletal - physiology

Blood Vessels - cytology

Membrane Glycoproteins - genetics

Mice, Knockout

Locomotion

Regeneration

Animals

Muscle Contraction

Sarcoglycans

Muscle Fibers, Skeletal - cytology

Stem Cells - physiology

Mice

Muscle, Skeletal - pathology

Genetic Vectors

Cell Movement

Details

Title: Subtitle: Cell therapy of alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts
Creators: Maurilio Sampaolesi - Stem Cell Research Institute, H. S. Raffaele, Via Olgettina 58, 20132 Milan, Italy
Yvan Torrente
Anna Innocenzi
Rossana Tonlorenzi
Giuseppe D'Antona
M Antonietta Pellegrino
Rita Barresi
Nereo Bresolin
M Gabriella Cusella De Angelis
Kevin P Campbell
Roberto Bottinelli
Giulio Cossu
Resource Type: Journal article
Publication Details: Science (American Association for the Advancement of Science), Vol.301(5632), pp.487-492
DOI: 10.1126/science.1082254
PMID: 12855815
NLM abbreviation: Science
ISSN: 0036-8075
eISSN: 1095-9203
Publisher: United States
Grant note: 463/BI / Telethon 1322 / Telethon
Language: English
Date published: 07/25/2003
Academic Unit: Neurology; Molecular Physiology and Biophysics; Iowa Neuroscience Institute
Record Identifier: 9984020989202771

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