RESUMO
Peripheral blood mononuclear cells (PBMCs) were collected from 55-year old male patient with a confirmed diagnosis of hemorrhagic Moyamoya disease (MMD). PBMCs were reprogrammed using Sendai virus particles delivering the four Yamanaka factors. A footprint-free hiPSC line was characterized by the expression of pluripotency markers and a normal karyotype. These cells were able to give rise to Embryoid Bodies and to a progeny of differentiated cells belonging to the 3 germ layers. This hiPSC line represents a suitable tool for modelling in vitro MMD disease to investigate the cellular mechanisms underlying the occurrence of this pathology.
Assuntos
Células-Tronco Pluripotentes Induzidas/citologia , Doença de Moyamoya/patologia , Diferenciação Celular , Linhagem Celular , Reprogramação Celular , Corpos Embrioides/citologia , Corpos Embrioides/metabolismo , Vetores Genéticos/genética , Vetores Genéticos/metabolismo , Humanos , Células-Tronco Pluripotentes Induzidas/metabolismo , Cariótipo , Leucócitos Mononucleares/citologia , Masculino , Microscopia de Fluorescência , Pessoa de Meia-Idade , Doença de Moyamoya/metabolismo , Vírus Sendai/genética , Fatores de Transcrição/genética , Fatores de Transcrição/metabolismoRESUMO
Peripheral blood mononuclear cells (PBMCs) were collected from an 8-year old female patient affected by ischemic Moyamoya disease (MMD). Patient's PBMCs were reprogrammed using Sendai virus particles delivering the four Yamanaka factors. The footprint free hiPSC line expressed the major pluripotency markers and exhibited a normal karyotype. Cells were competent to give rise to progeny of differentiated cells belonging to the 3 germ layers. This hiPSC line represents a good tool to in vitro model MMD in order to shed light on the cellular and molecular mechanisms responsible for the occurrence of this syndrome.
Assuntos
Células-Tronco Pluripotentes Induzidas/citologia , Doença de Moyamoya/patologia , Diferenciação Celular , Linhagem Celular , Reprogramação Celular , Criança , Corpos Embrioides/citologia , Corpos Embrioides/metabolismo , Feminino , Vetores Genéticos/genética , Vetores Genéticos/metabolismo , Humanos , Células-Tronco Pluripotentes Induzidas/metabolismo , Cariótipo , Leucócitos Mononucleares/citologia , Microscopia de Fluorescência , Doença de Moyamoya/metabolismo , Vírus Sendai/genética , Fatores de Transcrição/genética , Fatores de Transcrição/metabolismoRESUMO
Peripheral blood mononuclear cells (PBMCs) were collected from a patient with treatment-refractory Schizophrenia who presented an exceptional clinical response to Clozapine. iPSC lines were established with a non-integrating reprogramming system based on Sendai virus. A footprint-free hiPSC line was characterized to confirm the expression of the main endogenous pluripotency markers and have a regular karyotype. Pluripotency was confirmed by differentiation into cells belonging to the three germ layers. This hiPSC line represents a valuable tool to study the molecular, biochemical and electrophysiological properties of mature neuronal populations belonging to Clozapine responder patients with a severe form of Schizophrenia.
Assuntos
Antipsicóticos/uso terapêutico , Clozapina/uso terapêutico , Células-Tronco Pluripotentes Induzidas/citologia , Esquizofrenia/tratamento farmacológico , Adulto , Diferenciação Celular , Linhagem Celular , Reprogramação Celular , Corpos Embrioides/citologia , Corpos Embrioides/metabolismo , Vetores Genéticos/genética , Vetores Genéticos/metabolismo , Humanos , Células-Tronco Pluripotentes Induzidas/metabolismo , Cariótipo , Leucócitos Mononucleares/citologia , Masculino , Microscopia de Fluorescência , Esquizofrenia/metabolismo , Esquizofrenia/patologia , Vírus Sendai/genética , Fatores de Transcrição/genética , Fatores de Transcrição/metabolismoRESUMO
Peripheral Blood Mononuclear Cells (PBMCs) were collected from a patient with clozapine-resistant (also known as "super-refractory") Schizophrenia. iPSCs were established with a non-integrating Sendai virus-based reprogramming system. A footprint-free hiPSC line was characterized to express the main endogenous pluripotency markers and to retain a normal karyotype. Cells showed pluripotency competency by giving rise to progeny of differentiated cells belonging to the three germ layers. This hiPSC line represents a valuable tool to obtain mature, pathology-relevant neuronal populations in vitro that are suitable to investigate the molecular background of the schizophrenic disorder and the resultant patients' response to treatments.
