Trisomy 21 Mid-Trimester Amniotic Fluid Induced Pluripotent Stem Cells Maintain Genetic Signatures During Reprogramming: Implications for Disease Modeling and Cryobanking

Pipino, Caterina; Mukherjee, Sayandip; David, Anna L.; Blundell, Michael P.; Shaw, Steven W.; Sung, Peggy; Shangaris, Panicos; Waters, Jonathan J.; Ellershaw, Drew; Cavazzana, Marina; Mostoslavsky, Gustavo; Pandolfi, Assunta; Pierro, Agostino; Guillot, Pascale V.; Thrasher, Adrian J.; Coppi, Paolo De

doi:10.1089/cell.2013.0091

Cited by 15 publications

(12 citation statements)

References 44 publications

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“…Once cultured in adherence however, they do not express markers of hematopoietic lineage such as CD45, CD34, and CD133 and express CD29, CD44, CD73, CD90, and CD105 ( De Coppi, 2013 ). Interestingly, their plasticity, which is superior to adult stem cells, allow reprogramming into AFS derived induced pluripotent stem cells (iPS) with the change of the culture conditions they are exposed to ( Lu et al, 2012 ; Moschidou et al, 2012 , 2013 ; Pipino et al, 2014 ). This is particularly relevant as AFS cells can be utilized for cell banking of patient-specific pluripotent cells for potential applications in allogeneic cellular replacement therapies, pharmaceutical screening, and disease modeling ( Moschidou et al, 2012 , 2013 ).…”

Section: Amniotic Fluid As a Fetal Cell Source For In Utero Therapymentioning

confidence: 99%

In utero therapy for congenital disorders using amniotic fluid stem cells

et al. 2014

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Congenital diseases are responsible for over a third of all pediatric hospital admissions. Advances in prenatal screening and molecular diagnosis have allowed the detection of many life-threatening genetic diseases early in gestation. In utero transplantation (IUT) with stem cells could cure affected fetuses but so far in humans, successful IUT using allogeneic hematopoietic stem cells (HSCs), has been limited to fetuses with severe immunologic defects and more recently IUT with allogeneic mesenchymal stem cell transplantation, has improved phenotype in osteogenesis imperfecta. The options of preemptive treatment of congenital diseases in utero by stem cell or gene therapy changes the perspective of congenital diseases since it may avoid the need for postnatal treatment and reduce future costs. Amniotic fluid stem (AFS) cells have been isolated and characterized in human, mice, rodents, rabbit, and sheep and are a potential source of cells for therapeutic applications in disorders for treatment prenatally or postnatally. Gene transfer to the cells with long-term transgenic protein expression is feasible. Recently, pre-clinical autologous transplantation of transduced cells has been achieved in fetal sheep using minimally invasive ultrasound guided injection techniques. Clinically relevant levels of transgenic protein were expressed in the blood of transplanted lambs for at least 6 months. The cells have also demonstrated the potential of repair in a range of pre-clinical disease models such as neurological disorders, tracheal repair, bladder injury, and diaphragmatic hernia repair in neonates or adults. These results have been encouraging, and bring personalized tissue engineering for prenatal treatment of genetic disorders closer to the clinic.

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Section: Amniotic Fluid As a Fetal Cell Source For In Utero Therapymentioning

confidence: 99%

In utero therapy for congenital disorders using amniotic fluid stem cells

et al. 2014

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“…Once cultured in adherence however, they do not express markers of hematopoietic lineage such as CD45, CD34, and CD133 and express CD29, CD44, CD73, CD90, and CD105 (De Coppi, 2013). Interestingly, their plasticity, which is superior to adult stem cells, allow reprogramming into AFS derived induced pluripotent stem cells (iPS) with the change of the culture conditions they are exposed to (Lu et al, 2012;Moschidou et al, 2012Moschidou et al, , 2013Pipino et al, 2014). This is particularly relevant as AFS cells can be utilized for cell banking of patient-specific pluripotent cells for potential applications in allogeneic cellular replacement therapies, pharmaceutical screening, and disease modeling (Moschidou et al, 2012(Moschidou et al, , 2013.…”

Section: Growth and Characterization Of Amniotic Fluid-derived Stem Cmentioning

confidence: 99%

In Utero Therapy for Congenital Disorders Using Amniotic Fluid Stem Cells

Subramaniam¹,

Antoniadou²,

Coppi³

et al. 2018

Perinatal Stem Cells

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Congenital diseases are responsible for over a third of all pediatric hospital admissions. Advances in prenatal screening and molecular diagnosis have allowed the detection of many life-threatening genetic diseases early in gestation. In utero transplantation (IUT) with stem cells could cure affected fetuses but so far in humans, successful IUT using allogeneic hematopoietic stem cells (HSCs), has been limited to fetuses with severe immunologic defects and more recently IUT with allogeneic mesenchymal stem cell transplantation, has improved phenotype in osteogenesis imperfecta. The options of preemptive treatment of congenital diseases in utero by stem cell or gene therapy changes the perspective of congenital diseases since it may avoid the need for postnatal treatment and reduce future costs. Amniotic fluid stem (AFS) cells have been isolated and characterized in human, mice, rodents, rabbit, and sheep and are a potential source of cells for therapeutic applications in disorders for treatment prenatally or postnatally. Gene transfer to the cells with long-term transgenic protein expression is feasible. Recently, preclinical autologous transplantation of transduced cells has been achieved in fetal sheep using minimally invasive ultrasound guided injection techniques. Clinically relevant levels of transgenic protein were expressed in the blood of transplanted lambs for at least 6 months. The cells have also demonstrated the potential of repair in a range of preclinical disease models such as neurological disorders, tracheal repair, bladder injury, and diaphragmatic hernia repair in neonates or adults. These results have been encouraging, and bring personalized tissue engineering for prenatal treatment of genetic disorders closer to the clinic.

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“…This model revealed miR-155 and miR-802-two transcripts provided by chromosome 21-as key factors contributing to deficiency in neuronal differentiation. [ 50 51 ]…”

Section: Amniotic Fluid Stem Cells: a Resource For The Study And Treamentioning

confidence: 99%

Amniotic fluid stem cell models: A tool for filling the gaps in knowledge for human genetic diseases

2017

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Induced pluripotent stem (iPS) cells have attracted attention in recent years as a model of human genetic diseases. Starting from the diseased somatic cells isolated from an affected patient, iPS cells can be created and subsequently differentiated into various cell types that can be used to gain a better understanding of the disease at a cellular and molecular level. There are limitations of iPS cell generation, however, due to low efficiency, high costs, and lengthy protocols. The use of amniotic fluid stem cells (AFS) presents a worthy alternative as a stem cell source for modeling of human genetic diseases. Prenatal identification of chromosomal or Mendelian diseases may require the collection of amniotic fluid which is not only useful for the sake of diagnosis but also from this, AFS cells can be isolated and cultured. Since AFS cells show some characteristics of pluripotency, having the capacity to differentiate into various cell types derived from all three germ layers in vitro, they are a well-suited model for investigations regarding alterations in the molecular biology of a cell due to a specific genetic disease. This readily accessible source of stem cells can replace the necessity for generating iPS cells. Here, we expand on the applicability and importance of AFS cells as a model for discovery in the field of human genetic disease research. This paper is a review article. Referred literature in this paper has been listed in the references section. The data sets supporting the conclusions of this article are available online by searching various databases, including PubMed. Some original points in this article come from the laboratory practice in our research center and the authors’ experiences.

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Trisomy 21 Mid-Trimester Amniotic Fluid Induced Pluripotent Stem Cells Maintain Genetic Signatures During Reprogramming: Implications for Disease Modeling and Cryobanking

Cited by 15 publications

References 44 publications

In utero therapy for congenital disorders using amniotic fluid stem cells

In utero therapy for congenital disorders using amniotic fluid stem cells

In Utero Therapy for Congenital Disorders Using Amniotic Fluid Stem Cells

Amniotic fluid stem cell models: A tool for filling the gaps in knowledge for human genetic diseases

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