Induced Pluripotent Stem Cell Reprogramming by Integration-Free Sendai Virus Vectors from Peripheral Blood of Patients with Craniometaphyseal Dysplasia

Chen, I‐Ping; Fukuda, Keiichi; Fusaki, Noemi; Iida, Akihiro; Hasegawa, Mamoru; Lichtler, Alexander C.; Reichenberger, Ernst

doi:10.1089/cell.2013.0037

Cited by 47 publications

(31 citation statements)

References 66 publications

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“…This demonstrates that the reprogramming procedure requires further optimization for the clinical applications of iPSCs and their derivatives. More recently, iPSCs have been derived with a lentivirus‐mediated approach and non‐integrated Sendai virus vectors with the hope to reduce these concerns . Additional approaches using RNAs, proteins, and chemical‐mediated reprogramming have been developed .…”

Section: Cell‐based Therapies For MImentioning

confidence: 99%

Stem Cell Therapy of Myocardial Infarction: A Promising Opportunity in Bioengineering

Jiang

Shamul

et al. 2020

Advanced Therapeutics

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Myocardial infarction (MI) is a life‐threatening disease resulting from the irreversible death of cardiomyocytes (CMs). Stem cell‐based therapies have been studied for MI treatment over the last two decades with promising outcomes. Here, the past work in this field is critically reviewed to elucidate the advantages and disadvantages of treating MI using pluripotent stem cells (PSCs) including both embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs), adult stem cells, and cardiac progenitor cells. Overall, PSCs (particularly iPSCs) have more advantages than the other cells for cardiac regeneration. However, the use of iPSCs is also facing critical challenges, which may be resolved by using biomaterials to engineer stem cells for reduced immunogenicity, improved immobilization/survival in the heart, and increased integration with the host cardiac tissue to eliminate arrhythmia. Biomaterials have also been applied in the derivation of CMs in vitro to increase the efficiency and maturation of cardiac differentiation. Collectively, a lot has been learned from the past failures of simply injecting intact stem cells or their derivatives in vivo for treating MI, and bioengineering stem cells with biomaterials may be a valuable strategy for advancing stem cell therapy towards its widespread application for MI treatment in the clinic.

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Section: Cell‐based Therapies For MImentioning

confidence: 99%

Stem Cell Therapy of Myocardial Infarction: A Promising Opportunity in Bioengineering

Jiang

Shamul

et al. 2020

Advanced Therapeutics

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“…Recent studies using RNA‐based technology , pluripotency‐associated protein transfection , non‐integrating methods of the pluripotent gene containing plasmid usage and a pluripotent gene containing Sendai viral vectors are hinting towards safe clinical usage of footprint‐free hiPSC‐derived cellular products, such as iMSCs, since these directed differentiated cells will not have any risk of undesired genomic modifications associated with reprogramming protocol.…”

Section: Establishment Of Reliable and Standardized Source Of Functiomentioning

confidence: 99%

“…Recent studies using RNA-based technology [84], pluripotencyassociated protein transfection [85], non-integrating methods of the pluripotent gene containing plasmid usage [86] and a pluripotent gene containing Sendai viral vectors [87,88] are hinting towards safe clinical usage of footprint-free hiPSC-derived cellular products, such as iMSCs, since these directed differentiated cells will not have any Umbilical cord MSCs [46] Placental MSCs [47] Amniotic membrane MSCs [46] Amniotic fluid-derived cells [48] Retinal pigment epithelial cells [53] Corneal epithelial cells [44]…”

Section: Establishment Of Reliable and Standardized Source Of Functiomentioning

confidence: 99%

“…Progressive familial cholestasis (PFD) [162] Crigler-Najjar syndrome (CN) [162] Hurler syndrome (HS) [163] Neuronal ceroid lipofuscinosis (NCL) [164] Wilson's disease (WD) [165] Mitochondrial diabetes (MT) [166] Fabry disease (FD) [87] Mucopolysaccharidosis type IIIB disease (MPS) [167] Cardiovascular LEOPARD syndrome (LS) [78] Long QT syndrome type 1 (LQTS1) [168] Long QT syndrome type 2 (LQTS2) [169] Long QT syndrome type 3 (LQTS3) [170] Supervascular aortic stenosis (SVAS) [171] Hypertrophic cardiomyopathy (HCM) [172] Diabetic cardiomyopathy (DCM) [173] Hypoplastic left heart syndrome (HLHS) [174] Moyamoya disease (MMD) [175] Catecholaminergic polymorphic ventricular tachycardia (CPVT) [176] Familial dilated cardiomyopathy (DCM) [177] Familial hypertrophic cardiomyopathy (HCM) [178] Primary immunodeficiency SCID/Leaky SCID [179] Omenn syndrome (OS) [179] Cartilage-hair hypoplasia (CHH) [179] Herpes simplex encephalitis (HSE) [179] Musculoskeletal disorder Craniometaphyseal dysplasia (CMD) [88] Duchenne muscular dystrophy (DMD) [125] Becker muscular dystrophy (BMD) [125] Osteogenesis imperfect (OI) [180] Thanatophoric dysplasia (THD) [181] Achondroplasia (ACH) [181] Hutchinson-Gilford progeria syndrome (HGPS) [182] Werner syndrome (WS) [183] ª technologies without viral transgene such as chemicals, plasmids and recombinant protein-based approaches might augment the clinical utilization of these safe iPSCs [85,211,212]. The low efficiency of iPSCs generation might be a serve-de...…”

Section: Disease Modelling Referencesmentioning

confidence: 99%

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hiPSC‐derived iMSCs: NextGen MSCs as an advanced therapeutically active cell resource for regenerative medicine

Sabapathy

Kumar

2016

J Cellular Molecular Medi

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Mesenchymal stem cells (MSCs) are being assessed for ameliorating the severity of graft‐versus‐host disease, autoimmune conditions, musculoskeletal injuries and cardiovascular diseases. While most of these clinical therapeutic applications require substantial cell quantities, the number of MSCs that can be obtained initially from a single donor remains limited. The utility of MSCs derived from human‐induced pluripotent stem cells (hiPSCs) has been shown in recent pre‐clinical studies. Since adult MSCs have limited capability regarding proliferation, the quantum of bioactive factor secretion and immunomodulation ability may be constrained. Hence, the alternate source of MSCs is being considered to replace the commonly used adult tissue‐derived MSCs. The MSCs have been obtained from various adult and foetal tissues. The hiPSC‐derived MSCs (iMSCs) are transpiring as an attractive source of MSCs because during reprogramming process, cells undergo rejuvination, exhibiting better cellular vitality such as survival, proliferation and differentiations potentials. The autologous iMSCs could be considered as an inexhaustible source of MSCs that could be used to meet the unmet clinical needs. Human‐induced PSC‐derived MSCs are reported to be superior when compared to the adult MSCs regarding cell proliferation, immunomodulation, cytokines profiles, microenvironment modulating exosomes and bioactive paracrine factors secretion. Strategies such as derivation and propagation of iMSCs in chemically defined culture conditions and use of footprint‐free safer reprogramming strategies have contributed towards the development of clinically relevant cell types. In this review, the role of iPSC‐derived mesenchymal stromal cells (iMSCs) as an alternate source of therapeutically active MSCs has been described. Additionally, we also describe the role of iMSCs in regenerative medical applications, the necessary strategies, and the regulatory policies that have to be enforced to render iMSC's effectiveness in translational medicine.

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“…Synthetic mRNA reprogramming shows a safe and high efficiency and has produced MGD patient iPS cells (shown in Table ), but with only a small fraction of the samples not from blood cells . Using SeV, transgene‐free normal human or MGD patient iPSCs can be more efficiently generated from different donor cell types in feeder‐free conditions than other reprogramming methods (shown in Table ). However, there is no clinical‐grade SeV for reprogramming, and current studies are dependent on only one commercial vendor .…”

Section: Stem Cells Originating From Mgd Patientsmentioning

confidence: 99%

Concise Review: Patient-Derived Stem Cell Research for Monogenic Disorders

Qin

Gao

2015

Stem Cells

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Monogenic disorders (MGDs) are caused by a single gene mutation and have a serious impact on human health. At present, there are no effective therapeutic methods for MGDs. Stem cell techniques provide insights into potential treatments for MGDs. With the development of patient-derived stem cells, we can begin to progressively understand the molecular mechanism of MGDs and identify new drugs for MGD treatment. Using powerful genome editing tools, such as zinc finger nucleases, transcriptional activator-like effector nucleases, and the clustered regulatory interspaced short palindromic repeat/Cas9 system, MGD-associated gene mutations can be corrected in MGD stem cells in vitro and then transplanted into MGD animal models to assess their safety and therapeutic effects. Despite the continued challenges surrounding potential pluripotent stem cell tumorigenicity and concerns regarding the genetic modification of stem cells, the extensive clinical application of MGD patient-specific stem cells will be pursued through further advances in basic research in the MGD field. In this review, we will summarize the latest progress in research into the use of patient-derived stem cells for the potential treatment of MGDs and provide predictions regarding the direction of future investigations. STEM CELLS 2016;34:44-54 SIGNIFICANCE STATEMENTMonogenic disorders (MGDs) are caused by a single gene mutation and have a serious impact on human health. At present, there are no effective therapeutic methods for MGDs. Stem cell techniques provide insights into potential treatments for MGDs. With the development of patient-derived stem cells, we can begin to progressively understand the molecular mechanism of MGDs and identify new drugs for MGD treatment. Using powerful genome editing tools, such as zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs) and the clustered regulatory interspaced short palindromic repeat (CRISPR)/Cas9 system, MGD-associated gene mutations can be corrected in MGD stem cells in vitro and then transplanted into MGD animal models to assess their safety and therapeutic effects. Despite the continued challenges surrounding potential pluripotent stem cell tumorigenicity and concerns regarding the genetic modification of stem cells, the extensive clinical application of MGD patient-specific stem cells will be pursued through further advances in basic research in the MGD field. In this review, we will summarize the latest progress in research into the use of patient-derived stem cells for the potential treatment of MGDs and provide predictions regarding the direction of future investigations.

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Induced Pluripotent Stem Cell Reprogramming by Integration-Free Sendai Virus Vectors from Peripheral Blood of Patients with Craniometaphyseal Dysplasia

Cited by 47 publications

References 66 publications

Stem Cell Therapy of Myocardial Infarction: A Promising Opportunity in Bioengineering

Stem Cell Therapy of Myocardial Infarction: A Promising Opportunity in Bioengineering

hiPSC‐derived iMSCs: NextGen MSCs as an advanced therapeutically active cell resource for regenerative medicine

Concise Review: Patient-Derived Stem Cell Research for Monogenic Disorders

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