Transplantation of Induced Pluripotent Stem Cell-derived Mesoangioblast-like Myogenic Progenitors in Mouse Models of Muscle Regeneration

Mattia F. M. Gerli; Sara M. Maffioletti; Queensta Millet; Francesco Saverio Tedesco

doi:10.3791/50532

Transplante de Progenitores Miogênicos derivados de células-tronco pluripotentes induzidos em mod...

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Bioengineering

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JoVE Journal Bioengineering

Transplantation of Induced Pluripotent Stem Cell-derived Mesoangioblast-like Myogenic Progenitors in Mouse Models of Muscle Regeneration

Transplante de Progenitores Miogênicos derivados de células-tronco pluripotentes induzidos em modelos de regeneração muscular

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10:03 min

January 20, 2014

DOI: 10.3791/50532-v

Mattia F. M. Gerli*¹, Sara M. Maffioletti*¹, Queensta Millet¹, Francesco Saverio Tedesco^1,2

¹Department of Cell and Developmental Biology,University College London, ²Division of Regenerative Medicine, Stem Cells and Gene Therapy,San Raffaele Hospital

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Overview

This protocol outlines the evaluation of myogenic potential of induced pluripotent stem cell (iPSC)-derived muscle progenitors in mouse models of muscle regeneration. It includes transplantation techniques and functional assessments to measure engraftment and differentiation.

Key Study Components

Area of Science

Stem Cell Biology
Muscle Regeneration
Cell Therapy

Background

Induced pluripotent stem cells (iPSCs) can differentiate into various cell types.
Myogenic progenitors derived from iPSCs are promising for treating muscular dystrophies.
Understanding engraftment and functional contribution is crucial for therapeutic applications.
This study focuses on both acute and chronic muscle regeneration models.

Purpose of Study

To assess the myogenic potential of iPSC-derived muscle progenitors.
To evaluate the effectiveness of transplantation methods.
To measure the functional improvement in dystrophic mouse models.

Methods Used

In vitro assessment of myogenic potential of iPSC-derived cells.
Intramuscular injection of cells for transplantation.
Microsurgical injection via the femoral artery as an alternative method.
Immunofluorescence and histology assays to evaluate engraftment and functionality.

Main Results

Successful engraftment of iPSC-derived muscle progenitors in host muscles.
Functional amelioration of the dystrophic phenotype observed in treated animals.
Histological analysis confirmed differentiation and integration of donor cells.
In vitro assessments correlated with in vivo outcomes.

Conclusions

iPSC-derived myogenic progenitors show promise for muscle regeneration therapies.
Transplantation methods effectively enhance muscle repair in dystrophic models.
Further studies are needed to optimize protocols for clinical applications.

Frequently Asked Questions

What are induced pluripotent stem cells?

Induced pluripotent stem cells (iPSCs) are reprogrammed somatic cells that can differentiate into various cell types, including muscle progenitors.

How are the cells transplanted in this study?

Cells are transplanted via intramuscular injection or through the femoral artery using microsurgical techniques.

What methods are used to assess engraftment?

Immunofluorescence and histology assays are employed to evaluate the engraftment and functional contribution of the transplanted cells.

What is the significance of this research?

This research aims to develop effective cell therapy strategies for treating muscular dystrophies using iPSC-derived muscle progenitors.

What models are used in this study?

Mouse models of acute and chronic muscle regeneration are utilized to assess the effectiveness of the treatment.

What are the expected outcomes of the transplantation?

The expected outcomes include successful engraftment of the cells and functional improvement in the dystrophic phenotype of the treated animals.

Progenitores miogênicos derivados de células-tronco pluripotentes induzidas (iPSC) são candidatos promissores para estratégias de terapia celular para tratar distrofias musculares. Este protocolo descreve transplantes e medidas funcionais necessárias para avaliar o enenxerto e diferenciação dos mesoangioblastos derivados do iPSC (um tipo de progenitores musculares) em modelos de camundongos de regeneração muscular aguda e crônica.

O objetivo geral deste procedimento é avaliar o potencial miogênico de progenitores musculares derivados de células-tronco pluripotentes induzidas em modelos de camundongos de regeneração muscular aguda e crônica. Isso é feito avaliando primeiro o potencial miogênico das células in vitro. O segundo passo é transplantar as células por injeção intramuscular.

Alternativamente, as células são injetadas na artéria femoral por um procedimento microcirúrgico. A etapa final é avaliar a melhora funcional do fenótipo distrófico dos animais tratados. Em última análise, ensaios de imunofluorescência e histologia são usados para mostrar o enxerto e a contribuição funcional das células doadoras para os músculos hospedeiros.

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