Cell therapy for hereditary epidermolysis bullosa / 中华皮肤科杂志

Hereditary epidermolysis bullosa (EB) is a rare mutilating and lethal single-gene genodermatosis, and places a heavy burden on society and families. Cell therapy has become a very promising method for the treatment of EB due to its excellent and stable clinical efficacy. This review summarizes progress in laboratory research and clinical application of stem cell- and somatic cell-based therapies in EB in recent years.

Mechanisms of new targeted drugs in follicular lymphoma and clinical options / 白血病·淋巴瘤

Follicular lymphoma is the most common indolent lymphoma. At present, it is commonly treated with immunochemotherapy, but the prognosis of patients with progression and relapse is still poor. New targeted drugs include cell surface antibodies, immunomodulators, cell signaling pathway kinase inhibitors, and chimeric antigen receptor T cell therapy and dendritic cell vaccines have greatly extended the survival period of these patients and provided more options for clinical treatment.

Tratamento das cicatrizes atróficas com insulina subcutânea / Atrophic scars treatment with subcutaneous insulin

As cicatrizes atróficas de acne, sequelas persistentes, são indesejáveis e causam impacto negativo cosmético e psicossocial nos pacientes. A questão torna-se mais delicada quando tais cicatrizes localizam-se na região pré-esternal, pelo risco do surgimento de cicatrizes hipertróficas com a realização de procedimentos na região. O papel da insulina na promoção da síntese de proteínas e gorduras é, há muito tempo, reconhecido. A utilização de suas propriedades como fator de crescimento para tratar estas sequelas parece lógica e provou ser gratificante cosmeticamente, com melhora na qualidade de vida

Atrophic acne scars are persistent and undesirable sequelae that have a negative cosmetic and psychosocial impact on patients. This issue becomes more delicate when such scars are located in the presternal region due to the risk of hypertrophic scars appearing when performing these procedures in the area. The literature has long recognized the role of insulin in promoting protein and fat synthesis. Insulin properties as a growth factor to treat these sequelae seems logical and has proved cosmetically satisfying, with quality of life improvement

Progress and application of cellular immunotherapy / 中国医师杂志

Incidence of autoimmune diseases (AID) and tumors has increased dramatically,which seriously affects the survival and the quality of life in patients.Recently,cellular immunotherapy has become more and more attractive in the treatment of AID and tumors.Emerging studies have shown that cellular immunotherapy can alleviate or even reverse the course of AID and tumors.We reviewed to focus on the advances of cellular immunotherapy in AID and tumors.

Progress of therapeutic angiogenesis in lower limb ischemia / 国际外科学杂志

Lower limb ischemia is common and refractory diseases,in severe cases,it can impact on life quality even threat life.The traditional treatment contain medical and surgical,but cannot obtain satisfactory results.And therapeutic angiogenesis has achieve satisfactory results in animal experimnents and clinical study as its put forward.In recent years,Cell Therapy as one of the research hotspot,the micle aims to introduce the research of progress about therapeutic angiogenesis in detail.

Progresses of cell-based therapy and tissue engineering in the treatment of age-related macular degeneration / 中华眼底病杂志

Age-related macular degeneration (AMD) has become one of the leading causes of irreversible blindness worldwide.With the advancement of stem cell technology,tissue engineering and biomaterials,cell-based therapy has been inspiring for many degenerative diseases.For its unique advantages,AMD has become one of the most promising fields for cell-based therapy,which involve retinal pigment epithelium(RPE)cells,induced differentiation of neural retina cells and related cytokine regulations.RPE cells can be derived from human embryonic stem cells (hESC) or Induced pluripotent stem cells (iPS).Recently hESC-derived RPE cells have been applied to patients with dry AMD with initial success in clinical trials.In terms of tissue engineering,studies are focused on factors affecting the long-term survival of transplanted cells,including tissue scaffolds,soluble hybrid materials and scaffold anchoring.This article briefly reviews the RPE differentiation,neural retina differentiation and related cytokines of cell-based therapy and scaffolds,materials,and cell-scaffolds interactions of tissue engineering in AMD treatment.

Circulating fibrocytes and chronic wound healing / 中国组织工程研究

BACKGROUND:Circulating fibrocytes originated from the peripheral blood are a fibroblast-like cel subpopulation of leukocytes. Circulating fibrocytes play an essential role in wound repair by secreting extracel ular matrix proteins and cytokines, initiating antigen-specific immunity, accelerating wound contraction as wel as promoting angiogenesis. However, the potential role for improving chronic wound healing and its mechanism is stil unclear. OBJECTIVE:To summarize the biological features of circulating fibrocytes and its potential role and mechanism in accelerating chronic wound healing. METHODS:A computer-based search of CNKI database from 2000 to 2014, PubMed database from 1994 to 2014 and Foreign Medical Journal Ful-Text Service database from 2000 to 2014 was performed using“circulating fibrocytes, non-healing wounds, diabetic foot ulcer, wound healing, cel therapy”in Chinese and English as the keywords to retrieve articles related to circulating fibrocytes, refractory mechanism of chronic wound healing and cel therapy for chronic wounds. Total y 54 articles meeting the inclusive criteria were included in result analysis. RESULTS AND CONCLUSION:Cel therapy with circulating fibrocytes is a safe and effective treatment for improving wound healing and has been used to repair various chronic wounds in the recent decades. Circulating fibrocytes are a distinct subpopulation of fibroblast-like leukocytes in the peripheral blood. It is able to migrate into wound sites at the early stage of wound repair and plays an active role in the wound healing process by secreting extracel ular matrix proteins, cytokines, presenting antigens, contracting wound and promoting neovascularization. Recently, it has been proved in animal experiments that circulating fibrocytes can accelerate chronic wound healing, especial y diabetic chronic wound.

Bone marrow mesenchymal stem cells for osteoarthritis:its possibility and future / 中国组织工程研究

BACKGROUND:Nowadays treatment of osteoarthritis with drugs is not ideal. In recent years, more and more scientists try to use stem cells to treat osteoarthritis. OBJECTIVE:To explore the stem celltreatment for osteoarthritis in order to promote its clinical application and find out its difficulties. METHODS:A computer-based online search of PubMed database and CNKI database between March 1998 and October 2013 was performed to search related articles with the key words of“stem cell, osteoarthritis, bone metabolism, bone marrow mesenchymal stem cells”in English or in Chinese, respectively. The word“AND”was used for the connection between the word retrieval. Literatures related to stem cells treatment for osteoarthritis were selected;in the same field, the articles published lately in authoritative journals were preferred. RESULTS AND CONCLUSION:A total of 79 literatures were primarily selected, and 50 documents were involved for summary according to inclusion criteria. The stem celltreatment for osteoarthritis is realized mainly by promoting the repair of cartilage tissue. Commonly used methods are stem celltransplantation and induced differentiation of stem cells. Stem celltreatment has broad application prospects for the treatment of osteoarthritis.

Synovial mesenchymal stem cells-based therapy for cartilage repair An issue concerning clinical transformation / 中国组织工程研究

BACKGROUND:Cartilage injury is stil one of the clinical problems difficult to be treated completely so far. Recently, the discovery of synovial mesenchymal stem cells (SMSCs) has brought about the new hope to cartilage repair. OBJECTIVE:To explore the process concerning SMSCs-based therapy for cartilage repair in the past few years, such as the characteristics of SMSCs, culture conditions, preclinical and clinical studies, and then to summarize the literatures published in recent years. METHODS:A computed-based online search of PubMed and SpringerLink databases was performed using the key words of“synovial mesenchymal stem cells, cartilage repair”for literatures published from January 1993 to May 2013. RESULTS AND CONCLUSION:Final y, 37 articles were included. SMSCs have a greater proliferative capability, colony-forming potential and chondrogenic potential than other mesenchymal stem cells. The diseases such as osteoarthritis and rheumatoid arthritis can influence the characteristics of SMSCs. Numerous articles have aimed at the studies of cellculture in vitro and celltransplantation in vivo. However, the process of SMSCs therapy is mostly at its preliminary stage. Reports on its unique characteristics, optimal culture conditions and the high-quality clinical studies are stil largely lacking. In a word, though further studies are needed, SMSCs appear to be a promising cellsource for cartilage repair in the future.

Systematic review and meta-analysis of target terapies for the treatment of metastatic renal cancer

Objectives At present there are several drugs for the treatment of advanced renal cell carcinoma (ARCC). The main objective of this work was to perform a systematic review (SR) and meta-analysis (MA) of clinical randomized studies that compared target cell therapies (TCT). Materials and Methods SR identified clinical randomized trials that compared TCT versus interferon-alpha in the treatment of patients with ARCC. In order to analyze efficiency, it was evaluated free-survival progression (FSP), total survival (TS) and response rate (RR). Results In relation to first line treatment, seven studies of TCT were identified using sunitinib, sorafenib, bevacizumab and temsirolimus; and two studies with sorafenib and everolimus for second line treatment. Relative risk (RRi) of MA for FSP of first line therapies was: 0.83, CI = 0.78-0.87, I2 = 94% and p < 0.00001. Best results of RR of specific FSP among studies were: 0.38, sunitinib, CI = 0.25-0.58, bevacizumab, 0.62, CI = 0.47-0.83; and temsirolimus, 0.78, CI = 0.70-0.87. MA didn't show any benefit regarding TS of first line treatment of all analyzed drugs. As for RR significant results were: sunitinib, 3.83 CI = 2.86-5.12; bevacizumab, 2.52 CI = 1.78-3.57 and bevacizumab, 1.97 CI = 1.43-2.71. Conclusions: For first line treatment, sunitinib was the most effective TCT in relation to FPS; there was no alteration of TS and RR was small but significant for sunitinib and bevacizumab. Available studies could not conclude any results for second line treatments. .

Transplante de células mesenquimais de tecido adiposo na cardiopatia chagásica crônica experimental / Transplantation of adipose tissue mesenchymal stem cells in experimental chronic chagasic cardiopathy

FUNDAMENTO: A doença de Chagas, causada pelo protozoário Trypanosoma cruzi, é uma das mais importantes causas de insuficiência cardíaca na América Latina. A terapia celular vem sendo investigada como uma possível opção terapêutica para pacientes com doenças cardiovasculares. OBJETIVO: O objetivo deste estudo foi avaliar os efeitos da terapia com células-tronco mesenquimais em um modelo experimental de cardiomiopatia chagásica crônica. MÉTODOS: Camundongos C57BL/6 foram infectados com 1000 tripomastigotas da cepa Colombiana de T. cruzi e, após seis meses de infecção, foram tratados com células-tronco mesenquimais derivadas de tecido adiposo humano (CTTAs) ou com meio DMEM (controle). O grupo tratado recebeu duas injeções intraperitoneais de CTTAs (1x106 células / dose), com um mês de intervalo entre as duas doses. Antes e após 1 e 2 meses de tratamento, os animais chagásicos e controles normais foram submetidos à eletrocardiograma e teste ergoespirométrico. Todos os animais foram sacrificados sob anestesia após 2 meses de tratamento, para análise histopatológica do coração. RESULTADOS: Não foi observada melhora de arritmias e da função cardiovascular no grupo tratado com CTTAs, porém secções de corações de camundongos deste grupo apresentaram uma redução significativa do número de células inflamatórias (p < 0,0001) e da área de fibrose (p < 0,01) em comparação com animais chagásicos tratados com DMEM. CONCLUSÃO: Deste modo, conclui-se que a administração de CTTAs por via intraperitoneal é capaz de reduzir inflamação e fibrose no coração de camundongos cronicamente infectados por T. cruzi, porém não teve efeitos na função cardíaca dois meses após o transplante.

BACKGROUND: Chagas disease, caused by the protozoan Trypanosoma cruzi, is a major cause of heart failure in Latin America. Tissue therapy has been investigated as a possible therapeutic option for patients with cardiovascular disease. OBJECTIVE: This study evaluated the effects of therapy with mesenchymal stem cells in an experimental model of chronic Chagasic cardiomyopathy. METHODS: C57BL/6 mice were infected with 1000 trypomastigotes from the Colombian strain of T. cruzi and, after six months of infection, were treated with mesenchymal human stem cells from adipose tissue (STAT) or with Dulbecco/Vogt modified Eagle's minimal essential medium - DMEM (control). The treated group received two intraperitoneal injections of STAT (1x10(6) cells/dose), with a month interval between the two doses. Before and after the first and second months of treatment, the chagasic and normal control animals underwent cardiopulmonary exercise testing and electrocardiography. All animals were sacrificed under anesthesia after two months of treatment for histopathological analysis of the heart. RESULTS: No improvement was observed in arrhythmias and cardiovascular function in the group of animals treated with STAT; however, sections of mice hearts in this group revealed a significant reduction in the number of inflammatory cells (p<0.0001) and areas of fibrosis (p<0.01) in comparison with chagasic animals treated with DMEM. CONCLUSION: Thus, it is concluded that administration of intraperitoneal STAT can reduce inflammation and fibrosis in the heart of mice chronically infected with T. cruzi; however, there were no effects on the cardiac function two months after transplantation.

Counteracting the enzymatic activity of dipeptidylpeptidase 4 for potential therapeutic advantage, with an emphasis on cord blood transplantation

Dipeptidylpeptidase (DPP) 4, also known as CD26, is an enzyme present on the surface of a number of different cell types. It is also found within cells and as a soluble protein in body fluids. It can specifically truncate proteins at the penultimate N-terminus residue for some amino acids, such as alanine, proline, serine, and perhaps others. DPP4 has been implicated in regulating the in vitro and in vivo functional activities of a number of hematopoietically active molecules, and this information, along with that on inhibition of DPP4, has been studied in efforts to enhance hematopoietic cell transplantation (HCT), hematopoiesis after stress in mouse models, and in the clinical setting of single-unit cord blood (CB) HCT. This article reviews the current status of this compound's effects on regulatory proteins, the field of CB HCT, a potential role for modulating DPP4 activity in enhancing single-unit CB HCT in adults, and future aspects in context of other cellular therapies and the area of regenerative medicine.

Clinical applications of mesenchymal stem cells

Mesenchymal stem cells (MSCs) are self-renewing, multipotent progenitor cells with multilineage potential to differentiate into cell types of mesodermal origin, such as adipocytes, osteocytes, and chondrocytes. In addition, MSCs can migrate to sites of inflammation and exert potent immunosuppressive and anti-inflammatory effects through interactions between lymphocytes associated with both the innate and adaptive immune system. Along with these unique therapeutic properties, their ease of accessibility and expansion suggest that use of MSCs may be a useful therapeutic approach for various disorders. In the clinical setting, MSCs are being explored in trials of various conditions, including orthopedic injuries, graft versus host disease following bone marrow transplantation, cardiovascular diseases, autoimmune diseases, and liver diseases. Furthermore, genetic modification of MSCs to overexpress antitumor genes has provided prospects for clinical use as anticancer therapy. Here, we highlight the currently reported uses of MSCs in clinical trials and discuss their efficacy as well as their limitations.

Cell Therapy for Diabetic Neuropathy Using Adult Stem or Progenitor Cells

Diabetic neuropathy (DN) is the most common and disabling complication of diabetes that may lead to foot ulcers and limb amputations. Despite widespread awareness of DN, the only effective treatments are glucose control and pain management. A growing body of evidence suggests that DN is characterized by reduction of vascularity in peripheral nerves and deficiency in neurotrophic and angiogenic factors. Previous studies have tried to introduce neurotrophic or angiogenic factors in the form of protein or gene for therapy, but the effect was not significant. Recent studies have shown that bone marrow (BM)-derived stem or progenitor cells have favorable effects on the repair of cardiovascular diseases. Since these BM-derived stem or progenitor cells contain various angiogenic and neurotrophic factors, these cells have been attempted for treating experimental DN, and turned out to be effective for reversing various manifestations of experimental DN. These evidences suggest that cell therapy, affecting both vascular and neural components, can represent a novel therapeutic option for treatment of clinical DN.

Tratamiento de la enfermedad arterial periférica de las extremidades inferiores con células mononucleares de médula ósea autólogas: reporte de seguimiento a un año / Experience of autologous bone marrow mononuclear cells implantation as a treatment in patients with peripheral arterial disease: one year follow-up

Introducción: las células mononucleares de la médula ósea son efectivas para el tratamiento de pacientes con enfermedad arterial periférica (EAP). Los factores de crecimiento que ellas sintetizan se usan para inducir angiogénesis. Objetivo: establecer en Colombia la seguridad, factibilidad y resultado del tratamiento de la EAP utilizando células autólogas, para evitar su progreso a estadios más avanzados. Métodos: previo consentimiento, se hizo aspirado de médula ósea bajo anestesia local. Las células obtenidas se concentraron y se inyectaron intramuscularmente en los gastrocnemios de las extremidades isquémicas. El grupo control retrospectivo no recibió células. La evaluación clínica incluyó varios parámetros. Las células se analizaron por citometría de flujo. Resultados: edad media de los pacientes: 69 ± 11 años; viabilidad celular: 99,15% ± 0,76%; número de células inyectadas: 9,2 x 108 ± 6,2 x 108. Los análisis angiográficos postratamiento mostraron formación de vasos colaterales nuevos en las extremidades afectadas, con mínimo engrosamiento. Se observaron aumento en la distancia caminada libre de dolor y mejoría del dolor en reposo. La obtención y aplicación de las células no se asoció con ninguna complicación. Conclusiones: este estudio preliminar demostró que la terapia celular autóloga es segura, factible y cambia positivamente la historia natural de la EAP. Se sugiere el estudio de un número mayor de pacientes antes de establecer este tratamiento en Colombia.

Introduction: Autologous bone marrow mononuclear cells have been shown to be safe and effective for treatment of patients with peripheral arterial disease (PAD). Angiogenesis can also be induced by growth factors synthesized by them. Objective: To determine in Colombia the feasibility, safety and outcome of the afore-mentioned treatment. Methods: After informed consent, bone marrow was obtained by aspiration under local anesthesia; mononuclear cells were concentrated and their number and viability were established. They were suspended in saline solution and implanted by intramuscular injection into the gastrocnemius muscles of ischemic legs. Control patients were left untreated. Clinical evaluation included several parameters. Flow cytometry was used for cell analysis. Results: Mean age of patients: 69 ± 11 years; cell viability: 99.15 ± 0.76%; total number of injected cells: 9.2 x 108 ± 6.2 x 108. After treatment, angiographic studies showed the formation of new collateral vessels in all patients, with minimal thickness increase. There were no complications from bone marrow aspiration and intramuscular administration of cells. All treated patients experienced increase in the walking distance and improvement of rest pain. Conclusions: These preliminary results demonstrate that autologous cell therapy is safe, feasible and positively changes the natural history of patients with advanced peripheral arterial disease. In order to establish this treatment as a current practice in Colombia, we suggest the study of a larger number of patients.

Association of mesenchymal stem cells with platelet rich plasma on the repair of critical calvarial defects in mice / Associação de células-tronco mesenquimais com plasma rico em plaquetas na reparação de defeitos críticos em calvária de camundongos

PURPOSE: To evaluate the effects of mesenchymal stem cells (MSC) from eight mice C57BL/6 gfp+ bone marrows expanded in cultures associated with platelets rich plasma (PRP) deriving from another eight mice, in the repair of critical defects in calvarial bone produced in twenty-four adult isogenic mice C57BL/6. METHODS: The animals were submitted to a cranial defect of 6.0mm in diameter and divided into two equal experimental groups. Control group did not receive treatment and the treated group received a MSC pellet containing 1.0 x 10(7) cells/mL associated with 50.0µL of plasma gel containing 1.0 x 10(9) autologous platelets within the defect. RESULTS: In the treated group was observed process of angiogenesis and bone repair better than control group. CONCLUSION: Mesenchymal stem cells derived from bone marrow of C57BL/6 gfp+ mice associated with PRP gel applied in bone critical defects produced in calvarial contributes positively to the process of bone repair.

OBJETIVO: Avaliar os efeitos da associação das células-tronco mesenquimais (MSC) oriundas da medula óssea de oito camundongos jovens C57BL/6 gfp+ e expandidas em culturas, com Plasma Rico em Plaquetas (PRP) provenientes de outros oito camundongos, na reparação de defeitos críticos confeccionados em calvária de 24 camundongos adultos C57BL/6. MÉTODOS: Os animais foram submetidos a um defeito craniano de 6,0mm de diâmetro e separados em dois grupos experimentais iguais. O grupo controle não recebeu tratamento e no grupo tratado foi administrado, no interior do defeito, pellet de MSC contendo 1,0 x 10(7) células/mL associado com 50,0µL de plasma em gel autólogo contendo 1,0 x 10(9) plaquetas. RESULTADOS: No grupo tratado verificou-se processo de angiogênese e reparação óssea superior ao grupo controle. CONCLUSÃO: A associação das células-tronco mesenquimais (MSC) derivadas da medula óssea de camundongos C57BL/6 gfp+ com gel de PRP aplicadas em defeitos ósseos críticos confeccionadas em calvária de camundongos C57BL/6 jovens, contribuiu positivamente para o processo de reparação óssea.

Establishing a stem cell culture laboratory for clinical trials

Adult stem/progenitor cells are found in different human tissues. An in vitro cell culture is needed for their isolation or for their expansion when they are not available in a sufficient quantity to regenerate damaged organs and tissues. The level of complexity of these new technologies requires adequate facilities, qualified personnel with experience in cell culture techniques, assessment of quality and clear protocols for cell production. The rules for the implementation of cell therapy centers involve national and international standards of good manufacturing practices. However, such standards are not uniform, reflecting the diversity of technical and scientific development. Here standards from the United States, the European Union and Brazil are analyzed. Moreover, practical solutions encountered for the implementation of a cell therapy center appropriate for the preparation and supply of cultured cells for clinical studies are described. Development stages involved the planning and preparation of the project, the construction of the facility, standardization of laboratory procedures and development of systems to prevent cross contamination. Combining the theoretical knowledge of research centers involved in the study of cells with the practical experience of blood therapy services that manage structures for cell transplantation is presented as the best potential for synergy to meet the demands to implement cell therapy centers.

Functional improvement of porcine neonatal pancreatic cell clusters via conformal encapsulation using an air-driven encapsulator

Transplantation of islet cells into diabetic patients is a promising therapy, provided that the islet cells are able to evade host immune rejection. With improved islet viability, this strategy may effectively reverse diabetes. We applied 2% calcium alginate to generate small and large capsules to encapsulate porcine neonatal pancreatic cell clusters (NPCCs) using an air-driven encapsulator. After encapsulation, the viability was assessed at 1, 4, 7, 14 and 28 days and secretion of functional insulin in response to glucose stimulation were tested at days 14 and 28. Selective permeability of the small alginate capsules was confirmed using various sizes of isothiocyanate-labeled dextran (FITC-dextran). Encapsulation of NPCCs was performed without islet protrusion in the small and large capsules. The viability of NPCCs in all experimental groups was greater than 90% at day 1 and then gradually decreased after day 7. The NPCCs encapsulated in large capsules showed significantly lower viability (79.50 +/- 2.88%) than that of naive NPCCs and NPCCs in small capsule (86.83 +/- 2.32%, 87.67 +/- 2.07%, respectively) at day 7. The viability of naive NPCCs decreased rapidly at day 14 (75.67 +/- 1.75%), whereas the NPCCs encapsulated in small capsules maintained (82.0 +/- 2.19%). After 14 and 28 days NPCCs' function in small capsules (2.67 +/- 0.09 and 2.13 +/- 0.09) was conserved better compared to that of naive NPCCs (2.04 +/- 0.25 and 1.53 +/- 0.32, respectively) and NPCCs in large capsules (2.04 +/- 0.34 and 1.13 +/- 0.10, respectively), as assessed by a stimulation index. The small capsules also demonstrated selective permeability. With this encapsulation technique, small capsules improved the viability and insulin secretion of NPCCs without islet protrusion.

Disease-specific induced pluripotent stem cells: a platform for human disease modeling and drug discovery

The generation of disease-specific induced pluripotent stem cell (iPSC) lines from patients with incurable diseases is a promising approach for studying disease mechanisms and drug screening. Such innovation enables to obtain autologous cell sources in regenerative medicine. Herein, we report the generation and characterization of iPSCs from fibroblasts of patients with sporadic or familial diseases, including Parkinson's disease (PD), Alzheimer's disease (AD), juvenile-onset, type I diabetes mellitus (JDM), and Duchenne type muscular dystrophy (DMD), as well as from normal human fibroblasts (WT). As an example to modeling disease using disease-specific iPSCs, we also discuss the previously established childhood cerebral adrenoleukodystrophy (CCALD)- and adrenomyeloneuropathy (AMN)-iPSCs by our group. Through DNA fingerprinting analysis, the origins of generated disease-specific iPSC lines were identified. Each iPSC line exhibited an intense alkaline phosphatase activity, expression of pluripotent markers, and the potential to differentiate into all three embryonic germ layers: the ectoderm, endoderm, and mesoderm. Expression of endogenous pluripotent markers and downregulation of retrovirus-delivered transgenes [OCT4 (POU5F1), SOX2, KLF4, and c-MYC] were observed in the generated iPSCs. Collectively, our results demonstrated that disease-specific iPSC lines characteristically resembled hESC lines. Furthermore, we were able to differentiate PD-iPSCs, one of the disease-specific-iPSC lines we generated, into dopaminergic (DA) neurons, the cell type mostly affected by PD. These PD-specific DA neurons along with other examples of cell models derived from disease-specific iPSCs would provide a powerful platform for examining the pathophysiology of relevant diseases at the cellular and molecular levels and for developing new drugs and therapeutic regimens.

Clinical Applications of Neural Stem Cells for the Treatment of Peripheral Neuropathy / 한양의대학술지

Extraordinary advances in stem cell research have initiated an era of hope for strategies to treat intractable human diseases. Personalized regenerative treatment using stem cells is expected to accelerate continuous investment and research efforts throughout the world. Despite of their constraints, adult stem cells and embryonic stem cells have been used for cell transplantation for several intractable diseases. Besides adult and embryonic stem cells, the recent studies of induced pluripotent stem cells widened the possibility of patient-specific cell therapy, drug discovery, and disease modeling. This review focuses on the developments and potential applications of the stem cells for the treatment of peripheral neuropathy.