Human genetic diseases transplantation autologous. Medical search. Frequent questions

For example, because hematopoietic stem cells can reconstitute the entire blood system, bone marrow transplantation has long been used in the clinic to treat various diseases. (articlecity.com)
We are developing a multi-systemic strategy consisting of transplantation of autologous hematopoietic stem cells genetically modified ex vivo using lentivirus vectors. (ucsd.edu)
gene transfer, hematopoietic stem cells, lentiviral vectors, mesenchymal stem/progenitor cells, bone marrow transplantation, hematopoiesis Introduction HIV-based lentiviral vectors (LV) were proven to be capable of transducing a broad spectrum of nondividing cells in multiple mammalian species cIAP1 Ligand-Linker Conjugates 14 [1]. (eyesoftheelephants.com)

2 These cells either originate from the patient (autologous) or a donor (allogeneic). (pharmaceuticalcommerce.com)
Recent clinical trials evaluating allogeneic retinal grafts derived from human embryonic stem cells (hESCs) show the procedure to be safe and potentially effective 1 . (nature.com)
Solid tumors, autoimmune diseases, graft versus host disease (GvHD), genetic diseases and neurodegenerative diseases are investigated using many types of autologous and allogeneic cells with rapidly evolving cell engineering and editing approaches. (bdbiosciences.com)
Haematopoietic stem cell transplants have been performed in more than 1 500 000 patients (both autologous and allogeneic) to date.1 Although haematological cancers remain the main indication, haematopoietic stem cell transplants are increasingly considered in the treatment of non-malignant disorders and genetic diseases such as haemoglobinopathies (sickle cell anaemia, thalassaemia) that can benefit greatly from this type of transplant. (who.int)
Prior to the invention of CAR, cell therapies were largely limited to allogeneic hematological transfusion and transplantation. (scienceboard.net)
As a result, developmental trends have shifted toward an allogeneic approach, seeking to replace autologous cell sources with consistent and scalable alternatives, such as iPSCs. (scienceboard.net)
Because allogeneic transplants are associated with high risks of mortality and morbidity, we developed an autologous transplantation strategy of HSPCs genetically modified ex vivo to express a functional CTNS gene using a SIN-lentivirus vector. (ucsd.edu)
In contrast, injections of the cells into immunologically unmatched recipients (called an allogeneic transplantation) caused the body to mount a stronger immune response. (medindia.net)

PTLDs develop in approximately 2-3% of patients who undergo immunosuppressive therapy after solid organ transplantation. (medscape.com)
In May 2010, the Sixty-third World Health Assembly adopted resolution WHA63.22,1 in which it endorsed the updated WHO Guiding Principles on Human Cell, Tissue and Organ Transplantation and provided strategic directions to support progress in human organ, tissue and cell donation with the aim of maximizing the benefits of transplantation, meeting the needs of recipients, protecting donors and ensuring the dignity of all involved. (who.int)
Organ transplantation is often the best, if not the only, treatment for acute and chronic organ failure. (who.int)
Another project focuses on the generation of T regs and their potential use as modulators of immune tolerance in organ transplantation. (bu.edu)
The ideal treatment option for terminally ill patients is organ transplantation. (frontiersin.org)
Organ transplantation is the ultimate treatment option for patients suffering from refractory diseases. (frontiersin.org)
Depending on the patient's medical condition, a refractory disease patient also requires an on-time selective option, such as less invasive cellular therapy options or curative organ transplantation that can function immediately after transplantation. (frontiersin.org)
In addition, as the genetic identity of the donor egg from which the ESCs are derived most likely will differ from that of potential recipients, patients who receive ESC-derived cells or tissues may face the same complications that result from organ transplantation (for example, immunorejection, graft-versus-host disease, and need for immunosuppression). (biomedcentral.com)

The research, presented at the American Society of Hematology's 2011 annual meeting on Dec. 12, points to an error in a single digit of DNA -- one of three billion letters in the human genetic code -- as the leading culprit in Waldenström's, and a prime target for new therapies against the disease. (sciencedaily.com)
This important proof-of-concept finding supports the development of personalized iPSC-based transplantation therapies for retinal disease. (nature.com)
He was also Unit Director at France's National Institute of Health and Medical Research (Inserm) - France's NIH equivalent -, Founding Director of France's Institute of Emerging Diseases and Innovative Therapies (iMETI), and CEA's High Councilor for Medical Innovation. (harvard.edu)
There are many studies involving autologous therapies and some allogenic therapies, based on the recovery of mobilized bone marrow cells, including mesenchymal stem cells (MSCs) and adipose derived stem cells that also include the stromal or adherent cell type that has an MSC phenotype. (biomedcentral.com)
The present report aims to provide a comprehensive analysis of the current situation and facilitate a forward-looking discussion on actions for improving access to transplantation therapies. (who.int)
The clinical success of anti-CD19 CAR T-cell therapies has not only provided efficacious treatment alternatives for cancer patients but also aided the developmental and regulatory precedent for the translation of cell-based therapies for other disease areas. (scienceboard.net)
First-generation ex vivo cell therapies, such as autologous anti-CD19 CAR T cells, demonstrated the clinical value and curative potential of enhancing the therapeutic qualities of immune and other patient-derived cell types. (scienceboard.net)
In contrast to ESC, iPS cells are genetically identical to the individual from whom they are derived, raising the prospect of utilizing iPS cells for autologous cell based therapies without risk of rejection. (bu.edu)
In this regard, whole organ generation via the BC approach holds great promise with a ready resource (livestock) for cellular therapies and as a radical treatment option for most terminal diseases. (frontiersin.org)
The potential use of embryonic stem cells (ESCs) for cell replacement therapies is limited by ethical concerns and the technical hurdles associated with their isolation from human embryos. (biomedcentral.com)
Ideally, iPSC-based therapies in the future will rely on the isolation of skin fibroblasts or keratinocytes, their reprogramming into iPSCs, and the correction of the genetic defect followed by differentiation into the desired cell type and transplantation. (biomedcentral.com)
Future progress in this area holds promise for advancing islet- and beta cell-directed therapies that could be implemented in the early stages of the disease and could be combined with immunotherapies. (springer.com)

Follow-up integration of daSNV gene editing with clinical cohort analyses suggested that magnesium transport dysfunction may increase neuropsychiatric disease risk and indicated that common genetic pathomechanisms may mediate specific symptoms that are shared across multiple neuropsychiatric diseases. (stanford.edu)
Building off experience in hematopoietic stem cell transplant and exploiting advances in genetic engineering, genome editing, immunology, immunotherapy, stem cell biology, understanding disease mechanisms and cell therapy manufacturing, considerable progress has been made with hundreds of clinical trials underway. (bdbiosciences.com)
We have successfully established BRONJ minipig and mouse models treated with oncologic doses of zolendronate (Zometa)/Dexamethasone (Dex) developed BRONJ-like pathological lesions with similar clinical, radiographic, and histological features as described in the human disease. (ca.gov)
Cell-based therapy using systemic infusion of GMSC in experimental colitis significantly ameliorated both clinical and histopathological severity of the colonic inflammation, restored the injured gastrointestinal mucosal tissues, reversed diarrhea and weight loss, and suppressed the overall disease activity in mice. (ca.gov)
We examined the clinical outcome of three autologous PDLP-treated patients in an effort to provide primary knowledge on the effectiveness of this treatment approach and preliminary clinical evidence for randomized controlled trial in the future. (ca.gov)
This study demonstrated clinical and experimental evidences supporting a potential efficacy and safety of utilizing autologous PDL cells in the treatment of human periodontitis. (ca.gov)
The public clinical trials database http://clinicaltrials.gov shows 123 clinical trials using MSCs for a very wide range of therapeutic applications (Figure 1 ), the majority of which are in Phase I (safety studies), Phase II (proof of concept for efficacy in human patients), or a mixture of PhaseI/II studies (Figure 2 ). (biomedcentral.com)
Her research will build on the clinical observation that people with glaucoma frequently battle other chronic health problems, such as high blood pressure, diabetes, and heart disease. (nih.gov)
The clinical data after the transplantation will allow us to see if the iPS cell bank is truly useful or not. (neurosciencenews.com)
We will present a summary of the effect of hearing loss on auditory development, existing preclinical and clinical data on progenitor cell therapy, and its potential role in the (re)habilitation of non-genetic SNHL. (intechopen.com)
However, there are still questions waiting for answers and results of large-scale randomized trials are needed for the appearance of myocardial cellular therapy as a clinical therapeutic option for patients with ischemic heart disease. (escardio.org)
Various cell types have been tested experimentally for cardiac repair so far, but only those of autologous origin have yet undergone clinical testing due to immune competency. (escardio.org)
In experimental and clinical studies unselected mononuclear bone marrow cells as well as specific subpopulations have been used for transplantation. (escardio.org)
These findings give a rationale to start autologous transplantation at least of neural cells in clinical situations," says senior author Dr. Jun Takahashi, of the Kyoto University's Center for iPS Cell Research and Application. (medindia.net)
Next, in order to study the potential of autologous cell replacement therapy we transplanted progenitors derived from a PD patient into a pre-clinical rat model. (lu.se)
In this thesis, human glia-to-neuron direct conversion and engineered viral vectors are explored using pre-clinical in vitro and ex vivo models. (lu.se)
The WHO classification is based on a combination of clinical, morphologic, immunophenotypic, and genetic features. (msdmanuals.com)
En route to the patient we can also remove disease-associated genes that might compromise the transplanted cells and eventually the clinical benefit of the graft over time," described Fredrik. (lu.se)
Any detectable titer of neutralizing antibody against poliovirus is considered protective against clinical paralytic diseases. (who.int)

This includes bone and cartilage repair, cell types into which MSCs readily differentiate, and immune conditions such as graft versus host disease and autoimmune conditions that utilize the MSC's immune suppressive properties. (biomedcentral.com)
Optimizing these first-in-class approaches focuses on improving their safety and efficacy profile via a plethora of modifications, including targeting tumor-associated antigens, incorporating control switches, and employing gene editing to prevent graft-versus-host disease and/or other toxicity risk components. (scienceboard.net)

If successful, this would provide a means of performing autologous bone marrow transplantation to correct the bleeding in hemophiliacs. (ny.gov)
In collaboration with investigators in Taiwan, we implanted one type of autologous OMSCs (periodontal ligament progenitors, PDLPs) to treat an orofacial infectious bone defect disease periodontitis. (ca.gov)
Human orofacial bone-derived mesenchymal stem cells (OMSCs) showed distinct differentiation traits from mesenchymal stem cells (MSCs) derived from long bones, mouse OMSCs have not been isolated due to technical difficulties, which in turn precludes using mouse models to study orofacial diseases. (ca.gov)
The discrepancy in bone development between orofacial bones and long axial/appendicular bones give rises to specific diseases in the orofacial bone region, such as periodontitis, cherubism, and hyperparathyroid jaw tumor syndrome, which only affect the jaw bones. (ca.gov)
We are also investigating the mechanisms of tissue repair by bone marrow-derived stem cells after hematopoietic stem cell transplantation in the context of a non-hematopoietic disease. (ucsd.edu)
While cross-correction, either upon secretion- recapture after bone marrow transplantation was shown in several lysosomal storage disorders caused by defective soluble hydrolases, our study is the first demonstration of cross-correction in the context of a lysosomal transmembrane protein and of TNTs as key cellular device in the transfer. (ucsd.edu)
Transplanting their own (autologous) bone marrow-derived stem cells into 48 patients with end-stage liver disease resulted in therapeutic benefit to a high number of the patients, report researchers publishing in the current issue of Cell Transplantation (19:11). (medicaldaily.com)
Recently, we exhibited detectable levels of transgene (up to 3.9%) in PBL of mice 4 months after secondary bone marrow transplantation (BMT) of HSC transduced by delivery of LV in newborn pups [11]. (eyesoftheelephants.com)
Bone marrow stem cells from adults have been viewed as the ideal target for gene- and cell-based therapy of genetic diseases, selected malignant diseases, and AIDS. (eyesoftheelephants.com)

The first part of the thesis (Paper I, II, III) shows the development and improvement of a hESC-based system of for virus-mediated direct reprogramming of human glial progenitor cells into both induced dopaminergic neurons (iDANs) and GABAergic interneurons. (lu.se)

The discovery of induced pluripotent stem cells (iPSCs) and the ability to edit the genome bring the possibility to provide definitive genetic therapy through corrected autologous tissues. (nih.gov)
Induced pluripotent stem cells (iPSCs) generated from patient fibroblasts could potentially be used as a source of autologous cells for transplantation in retinal disease. (nature.com)
Patient-derived iPSCs, however, would still harbor disease-causing mutations. (nature.com)
Using a patient's own cells for transplantation would avoid these pitfalls and is possible with induced pluripotent stem cells (iPSCs). (nature.com)
Although the original disease-causing mutation would still be present in patient iPSCs, precise mutation correction is possible through gene editing techniques adapted from the bacterial clustered regularly interspersed short palindromic repeats (CRISPR)/Cas9 system 3 . (nature.com)
We tested whether CRISPR could precisely edit the pathogenic mutation and produce gene-corrected iPSCs for eventual use in autologous transplantation. (nature.com)
Derivation of autologous induced pluripotent stem cells (iPSCs) through direct reprogramming of easily accessible somatic cells holds the potential to transform the field of regenerative medicine. (biomedcentral.com)
On the basis of the unlimited capacity to be propagated in vitro , iPSCs are good targets for genetic manipulation by gene therapy or gene correction by homologous recombination. (biomedcentral.com)
The team grew retinal pigment cells from induced pluripotent stem cells (iPSCs) and transplanted them into the damaged retina of a human participant. (neurosciencenews.com)
In order to avoid tissue rejection, they used autologous iPSCs-iPSCs that were created from the recipient's own skin cells. (neurosciencenews.com)
While this method is sound, producing autologous iPSCs is costly. (neurosciencenews.com)
In a separate study published in the same issue of Stem Cell Reports , the researchers saw similar results when they repeated this last experiment with human T cells and HLA-matched or unmatched retinal pigment cells grown from IPSCs. (neurosciencenews.com)
Promotes neurogenesis in human induced pluripotent stem cells (iPSCs) when used in combination with SB 431542 (Cat. (tocris.com)
Induced pluripotent stem cells (iPSCs) extracted from the patient itself may be extremely helpful in treating many disease. (medindia.net)
In the paper, publishing September 26 in the ISSCR's journal Stem Cell Reports , published by Cell Press, iPSCs from nonhuman primates successfully developed into the neurons depleted by Parkinson's disease while eliciting only a minimal immune response. (medindia.net)
iPSCs directed to differentiate into specific cell types offer the possibility of a renewable source of replacement cells and tissues to treat ailments, including Parkinson's disease, spinal cord injury, heart disease, diabetes, and arthritis. (medindia.net)
To test this in an animal that is more closely related to humans, investigators in Japan directed iPSCs taken from a monkey to develop into certain neurons that are depleted in Parkinson's disease patients. (medindia.net)

1 By contrast, cellular therapy uses healthy transplanted human cells to grow, replace, or repair damaged tissue that causes disease. (pharmaceuticalcommerce.com)
Accumulating evidence demonstrates that many of the 17 human UGTs characterized to date exhibit tissue-specific patterns of expression. (aspetjournals.org)
We isolated a new population of stem cells from human orofacial tissue gingiva, a tissue source easily accessible from the oral cavity, namely GMSC, which exhibited clonogenicity, self-renewal, and multipotent differentiation capacities. (ca.gov)
The benefits of human tissue transplantation can be seen in both children and adults, including in survival rates following severe burn trauma, recovery of movement, closure of chronic wounds, rehabilitation of heart function and restoration of sight. (who.int)
Corneal disease (scarring or perforation) can be successfully addressed through transplantation in 80% of affected individuals.3 Tissue transplantation allows many recipients to return to economically productive lives and promotes their independence. (who.int)
Similarly, the transplantation of other tissue-specific stem cells, such as stem cells isolated from epithelial and neural tissues, can treat mouse disease models and human patients in which epithelial and neural cells are damaged. (articlecity.com)
Chronic active Epstein-Barr virus disease is usually defined as a chronic illness lasting at least 6 months, an increased EBV level in either the tissue or the blood, and lack of evidence of a known underlying immunodeficiency ( 15 ). (frontiersin.org)
There are many genetic variations of MHCs, and after transplantation, if the MHCs of the transplanted cells are not recognized by the T cells of the host immune system, there is an immune response and the tissue is rejected. (neurosciencenews.com)
Since the introduction of the mouse small intestinal organoid model in 2009, 1 there has been an avalanche of developments in this field, including development of culture conditions for human organoids derived from primary colonic tissue, 2 as well as from human pluripotent stem cells (hPSCs). (stemcell.com)
Intradermal injection of lentiviral vectors corrects regenerated human dystrophic epidermolysis bullosa skin tissue in vivo. (medscape.com)
Transplantations of fetal tissue in the 1980s and 1990s provided proof-of-concept for the potential of cell replacement therapy for PD and some patients benefitted greatly from their transplants. (lu.se)
However, post-mortem analysis of transplanted tissue revealed accumulation of pathological Lewy bodies in a small subset of transplanted cells over time, revealing a host-to-graft disease propagation. (lu.se)

A portion of my lab is also involved in the invention of new technologies to facilitate highly multiplexed staining of protein (immunostaining) and RNA (in situ hybridization) of human tissues. (stanford.edu)
The transplantation of human tissues, organs or cells is an established form of treatment that has been acknowledged as the best and very often only life-saving therapy for several serious and life-threatening congenital, inherited and acquired diseases and injuries. (who.int)
While the development of xenografts (products of animal origin) and bioengineered alternatives is advancing, to date they cannot fully mimic or replace all human-sourced tissues on an equivalent basis. (who.int)
Thus, the availability of and access to human tissues for transplantation remains essential. (who.int)
In June 2018, the Secretariat established the WHO Task Force on Donation and Transplantation of Human Organs and Tissues as an advisory group composed of experts from all WHO regions. (who.int)
Other authors, particularly when defining severe CAEBV disease, require both an elevated level of EBV in the blood as well as infiltration of tissues by EBV-positive lymphocytes ( 16 ). (frontiersin.org)
Notes lead author Sunao Sugita, "In order to make iPSC transplantation a practical reality, the current goal is to create banks of iPSC-derived tissues that can be transplanted into anyone as they are needed. (neurosciencenews.com)

Given strong human genetic evidence, there is little doubt, however, that microglia play an important role in preventing degeneration of neurons. (stanford.edu)
Modeling gene × environment interactions in PTSD using human neurons reveals diagnosis-specific glucocorticoid-induced gene expression. (reprocell.com)
Age-related pathological impairments in directly reprogrammed dopaminergic neurons derived from patients with idiopathic Parkinson's disease. (reprocell.com)
When they were injected into the same monkey's brain (called an autologous transplantation), the neurons elicited only a minimal immune response. (medindia.net)
The team's work also suggests that transplantation of such neurons into immunologically matched recipients may be possible with minimal use of immunosuppressive drugs. (medindia.net)
Second, we used directly converted neurons from sporadic patient fibroblasts to study of age-related disease relevant pathology. (lu.se)
Direct neuronal reprogramming of a somatic cell into therapeutic neurons, without a transient pluripotent state, provides new promise for the large number of individuals afflicted by neurodegenerative diseases or brain injury. (lu.se)
However, current protocols for deriving neurons from human pluripotent stem cells give rise to heterogeneous cell populations both in regard to the temporal aspects and the cellular composition. (lu.se)

By accessing primary human target cells we hope to establish a new paradigm in the use of iPSC-derived cells for infectious diseases. (bu.edu)
Studies in rodents have suggested that iPSC-derived cells used for transplantation may be rejected by the body's immune system. (medindia.net)

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. (medscape.com)

All but one patient had previously received an autologous stem cell transplant. (eurekalert.org)

More research teams are accelerating the use of other types of adult stem cells, in particular neural stem cells for diseases where beneficial outcome could result from either in-lineage cell replacement or extracellular factors. (biomedcentral.com)
Variation of DNA methylation on the IRX1/2 genes is responsible for the neural differentiation propensity in human induced pluripotent stem cells. (reprocell.com)
This strategy provides a useful tool for visualization and identification of neural progeny derived from human pluripotent stem cells. (lu.se)
We provide detailed protocols for lentiviral transduction, neural differentiation, and subsequent analysis of human embryonic stem cells. (lu.se)

We generated patient-derived COL7A1-corrected epithelial keratinocyte sheets for autologous grafting. (nih.gov)
Now that we have established the lack of immune response in monkeys and in human cells in vitro," explains Sugita, "using the iPS cell bank appears to be a viable solution, at least in the case of retinal pigment epithelial cell transplantation. (neurosciencenews.com)

According to FDA, gene therapy is used to modify a person's genes with the intent to treat or cure a disease. (pharmaceuticalcommerce.com)
Gene therapy was first introduced in the late 1970s, and the first trial in humans began in 1990 when colleagues at the National Institutes of Health (NIH) performed the first-approved gene therapy procedure on a 4-year-old with severe combined immunodeficiency (SCID). (pharmaceuticalcommerce.com)
Interindividual UGT variability likely plays an important role in drug efficacy and xenobiotic toxicity, as well as in hormonal regulation and certain diseases, which in some cases may be amenable to therapeutic manipulations including gene therapy. (aspetjournals.org)
A potential alternative to transplantation is gene therapy. (nih.gov)
Autologous Ex Vivo Lentiviral Gene Therapy for Adenosine Deaminase Deficiency. (nih.gov)
Groundbreaking gene therapy for genetic beta thalassemia is now accessible as a treatment to a patient post-FDA approval. (medindia.net)
Gene therapy for a lethal genetic blistering disease: a status report. (medscape.com)

Genetic polymorphisms may predispose patients to the development of PTLD. (medscape.com)
The discovery of a genetic signature for Waldenström's will enable doctors to definitively determine which patients have the disease and not a similar condition such as other forms of lymphoma or multiple myeloma, Treon says. (sciencedaily.com)
Drugs that block the abnormal protein or other proteins in the NF-kB pathway could, theoretically, short-circuit the disease process in many patients. (sciencedaily.com)
He participates in research involving gene correction of CFTR in lung stem cells from patients with Cystic fibrosis followed by autologous stem cell transplantation to provide lifelong restoration of physiological activity. (stanford.edu)
Patients also need to have periodic infusions of antibodies derived from human blood plasma. (nih.gov)
Autologous hematopoietic cell transplantation (HCT) is under investigation as a promising therapy for treatment-refractory MS. Here we identify a reactive myeloid state in chronic experimental autoimmune encephalitis (EAE) mice and MS patients that is surprisingly associated with neuroprotection and immune suppression. (stanford.edu)
Patients with CAEBV in the United States most often present with disease involving B or T cells, while in Asia, the disease usually involves T or NK cells. (frontiersin.org)
Rare patients who become infected with EBV, or reactivate EBV, develop disease that does not resolve. (frontiersin.org)
In addition, reduced numbers of EBV-specific T cells have been described in patients with CAEBV disease ( 10 ). (frontiersin.org)
Unlike healthy persons with infectious mononucleosis, patients with CAEBV disease often have low numbers of EBV-specific CD8 cells ( 10 ). (frontiersin.org)
FRDA is an autosomal recessive mitochondrial disease characterized by neurodegeneration, cardiomyopathy and muscle weakness and patients will be in wheelchair within 10-15 years of onset. (ucsd.edu)
Does Transplantation Induce Aging In Patients? (medicaldaily.com)
As such, stem cell therapy cannot be regarded as a valid therapeutic option for patients with cardiovascular disease in the present era of evidenced-based medicine, yet it holds great hope for the future. (escardio.org)
Several preliminary reports have demonstrated that local stem cell transplantation in patients with acute myocardial infarction is safe and may lead to improved myocardial function and perfusion. (escardio.org)
Scientists aspire to utilize stem cell transplantation for pediatric patients grappling with blood-related conditions like aplastic anemia, and thalassemia. (medindia.net)
Sixteen responding patients were evaluable for assessment of minimal residual disease (MRD) and all tested MRD negative at one or more time points. (eurekalert.org)

Researchers at NIH's National Institute of Allergy and Infectious Diseases (NIAID) and National Human Genome Research Institute (NHGRI) participated in the research, and NIAID and NIH's National Heart, Lung, and Blood Institute (NHLBI) provided financial support. (nih.gov)
With my supervisors Calliope Dendrou at the Wellcome Centre for Human Genetics and James Davies at the Weatherall Institute for Molecular Medicine, I am investigating precision genome editing with tandem autologous transplantation as a therapy for multiple severe immune-mediated diseases. (ox.ac.uk)

Several projects in the lab utilizes the liver differentiated progeny of iPSC to study genetic and infectious diseases affecting the liver. (bu.edu)

Dr. Desai conducts basic and translational research on lung stem cells that repair and regenerate the lung after injury, and their role in diseases like IPF, Chronic Obstructive Pulmonary Disease (COPD), and lung adenocarcinoma. (stanford.edu)
Noncommunicable diseases such as diabetes and arterial hypertension, combined with various social determinants (e.g. alcoholism, obesity), may lead to chronic kidney disease and liver cirrhosis, conditions that are among the top 10 causes of death worldwide. (who.int)
For chronic or acute liver failure, as well as some cardiorespiratory conditions, the only alternative to transplantation is death. (who.int)
Chronic active Epstein-Barr virus (CAEBV) disease is a rare disorder in which persons are unable to control infection with the virus. (frontiersin.org)
This entity is referred to as chronic active EBV (CAEBV) disease. (frontiersin.org)
Osteoarthritis (OA) is one of the common chronic diseases in the world with its main symptoms including joint pain and joint stiffness. (ncl.edu.tw)
Conventional and genetic risk factors for chronic Hepatitis B virus infection in a community-based study of 0.5 million Chinese adults. (ox.ac.uk)
However, from the beginning of puberty after thymus degeneration, human cytomegalovirus (HCMV) persists due to the chronic activation of cytomegalovirus in humans causing repeated activation of T cells, which is considered the driving factor of human immune aging [ 9 ]. (hindawi.com)
Chronic Pancreatitis Associated With a Variant of CFTR Gene Treated With Total Pancreatectomy and Autologous Islet Cell Transplantation. (cdc.gov)
A few exceptions emerged when successful functional correction was exhibited in children with severe combined immunodeficiency (ADA-SCID and X-SCID) or chronic granulomatous disease following infusion of retrovirally transduced autologous CD34+ cells [12C14]. (eyesoftheelephants.com)

Comparative analysis of human induced pluripotent stem cell-derived mesenchymal stem cells and umbilical cord mesenchymal stem cells. (reprocell.com)

We also showed for the first time that TNTs could cross the thick, dense and stiff renal tubular basement membrane in vivo and transfer cystinosin-bearing lysosomes to the proximal tubular cells, providing a mechanism underlying the long-term kidney preservation after HSPC transplantation in the Ctns -/- mice. (ucsd.edu)
Immunohistopathological studies of pancreases from individuals with type 1 diabetes are generally compatible with these observations made in mice (see references below) but have exposed additional phenomena peculiar to the natural history of the disease in humans. (springer.com)

Induced pluripotent stem cells, which harness the power of genetic reprogramming - basically, the altering of a cell's DNA - to change the course of cellular development. (articlecity.com)
Single transcription factor efficiently leads human induced pluripotent stem cells to functional microglia. (reprocell.com)
Using Microfluidics to Generate Human Naïve and Primed Pluripotent Stem Cells. (reprocell.com)
In: Rugg-Gunn P. (eds) Human Naïve Pluripotent Stem Cells. (reprocell.com)
Given this, human pluripotent stem cells, such as human embryonic stem cells (hESCs) and induced pluripo- tent stem cells (hiPSCs), have become a unique cell source for study- ing early human brain development. (lu.se)

Parkinson's disease (PD) affects approximately 1% of people over the age of 60. (lu.se)
For the last four years, Fredrik has explored the use of patient-specific cells in cell replacement therapy for Parkinson's disease to identify strategies to safeguard these transplanted cells from disease-related degeneration. (lu.se)
My Ph.D. studies revolved around cell replacement therapy for Parkinson's disease. (lu.se)
My thesis cover illustrates the workflow of generating patient-specific cells for cell replacement therapy for Parkinson's Disease. (lu.se)
Cell Replacement Therapy for Parkinson's Disease - Evaluating the potential of autologous grafting. (lu.se)

Outcomes of corneal transplantation in Europe: report by the European Cornea and Cell Transplantation Registry. (who.int)

Therefore, it is not surprising to find that human OMSCs are distinct from BMMSCs in terms of differentiation traits and immunoregulation. (ca.gov)
Stem cells are defined by their capacity for self-renewal and multilineage differentiation, making them uniquely situated to treat a broad spectrum of human diseases. (articlecity.com)
We have established in the lab robust differentiation protocols for the induction of intestinal specification of these disease-specific iPSC. (bu.edu)
DMH-1 induces intestinal differentiation in human intestinal organoids (hIOs) derived from human pluripotent stems cells (hPSCs). (tocris.com)
Many existing protocols for neuronal differentiation of human pluripotent cells result in heterogeneous cell populations and unsynchronized differentiation, necessitating the development of methods for labeling specific cell populations. (lu.se)

Over the last few decades, the treatment of hematological malignancies has gained tremendous headway, but these diseases still have high morbidity and mortality [ 1 - 3 ]. (hindawi.com)
Traditionally, the treatment of hematological malignancies is administrated by chemotherapy, radiotherapy, and stem cell transplantation. (hindawi.com)
In recent years, with the increased understanding of the molecular genetic basis of these malignancies, immune-targeted therapy has become a new possibility for the treatment of hematological malignancies. (hindawi.com)

High-dose chemotherapy with autologous stem cell transplantation is infrequently also used. (sciencedaily.com)

Genetic engineering technologies including CRISPR/Cas9 and Base Editing for therapeutic applications. (ox.ac.uk)
Nevertheless, striking new data from cer agents from Volume 100A of these studies show increased risks the IARC Monographs are used to Therapeutic applications and of almost all site-specific cancers make qualitative comparisons be- trends in use that emerge during the fol ow-up tween cancers induced in humans period. (who.int)
indicate tative comparisons of carcinogens been narrowed and their therapeutic that no agents classified as carci- with respect to potency in humans use has declined, with the possible nogenic to humans (Group 1) are and in experimental animals are exception of cyclophosphamide. (who.int)

Multiple sclerosis (MS) is an autoimmune disease associated with inflammatory demyelination in the central nervous system (CNS). (stanford.edu)
GMSC can function as an immunomodulatory and anti-inflammatory component of the immune system in vivo and is a promising cell source for cell-based treatment in experimental inflammatory diseases. (ca.gov)
Our long standing interest in the immune system now focuses in the utilization of iPSC-derived immune cells, including myeloid dendritic cells (mDCs), T cells and others as the basis to study several inflammatory diseases such as lupus erythematosus and the signaling cascade responsible for immune dysregulation. (bu.edu)
Dendritic cells play a central role in the modulation of the immune response and several inflammatory diseases have been shown to have altered mDC function. (bu.edu)
Treat inflammatory bowel disease (swelling in your digestive tract). (mskcc.org)

Scientists have identified a gene mutation that underlies the vast majority of cases of Waldenstrom's macroglobulinemia, a rare form of lymphoma that has eluded all previous efforts to find a genetic cause. (sciencedaily.com)
Lung stem cells can also be exploited to treat monogenic diseases, by using CRISPR to correct the genetic mutation then transplanting them back into the patient. (stanford.edu)

The engineered TCR-NK technology in Syena is based on the scientific discoveries of Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation & Cellular Therapy at MD Anderson. (businesswire.com)

In addition, various LPDs (eg, Castleman disease, Kikuchi-Fujimoto disease, and Rosai-Dorfman disease) can occur in the head and neck in individuals who have not undergone transplantation. (medscape.com)

To address this knowledge gap, we developed an in vivo hematopoietic stem cell (HSC)-based large-scale CRISPR knockout screening platform to enable the genetic interrogation of hematopoiesis and broad aspects of immune cell function in vivo. (biorxiv.org)

However, hESC-based treatments involve the controversial use of human embryos and pose a risk of immune-mediated rejection. (nature.com)
Mechanisms behind the regulation of proteins involved in immune mediated diseases. (ox.ac.uk)

[ 5 , 6 ] This trend is postulated to be secondary to increased growth in transplantation, older age of donors and recipients, increased diagnosis and awareness of PTLD, and the use of novel, aggressive immunosuppressive regimens. (medscape.com)

For monogenic diseases, there are only a certain number of mutations, and then we'll plateau until we reach a stage where we can go after more common diseases," Hordeaux said. (ajmc.com)

hESCs can be generated by SCNT using dermal fibroblasts nuclei from both a middle-aged 35-year-old male and an elderly, 75-year-old male, suggesting that age-associated changes are not necessarily an impediment to SCNT-based nuclear reprogramming of human cells. (wikipedia.org)
Through established protocols 2 , fibroblasts from a skin biopsy can be returned to a pluripotent state and serve as a renewable, autologous source of replacement cells that avoids the ethical complications of hESCs. (nature.com)
Efficient reprogramming of human fibroblasts using RNA reprogramming with DAPT and iDOT1L under normoxia conditions. (reprocell.com)

Centers for Disease Control and Prevention. (cdc.gov)

We have developed a unique infrastructure for studying the genetics of platelet diseases using RNA profiling techniques. (ny.gov)
During my Masters of Biological Sciences (Advanced) at the Australian National University, I conducted my research project in " Examining the genetics of malaria parasite Plasmodium falciparum invasion of human erythrocytes using CRISPR-Cas9 gene engineering " at the John Curtin School of Medical Research. (ox.ac.uk)

PRACTICAL APPLICATIONS: Moringa oleifera has been a vocal appetite in mitigating cardiovascular disease induced by diabetes, but the formulation of a medicinal diet as an ameliorative route of attention to the pathology is fairly addressed, not talking of its combination with the synthetic antidiabetic drug, such as ACA. (bvsalud.org)
The potential uses for them are vast: from basic research leading to a better understanding of the source of birth defects and cancer to potential treatments for common conditions like arthritis, diabetes, and heart disease to treatments for rarer but severe conditions like spinal cord and brain injuries. (articlecity.com)
In particular, the dialogue between T cells and beta cells is far more complex in humans and recent evidence supports potential type 1 diabetes 'endotypes' based on age at onset [ 8 , 9 ]. (springer.com)

Because a large proportion of cases are fatal and the virus could acquire the ability to spread more efficiently (as was the case with severe acute respiratory syndrome coronavirus), WHO has recommended thorough contact investigations for confirmed human cases to identify, quantify, and prevent person-to-person transmission ( 16 ). (cdc.gov)
Children born with a rare genetic disorder called severe combined immunodeficiency (SCID) cannot produce the immune cells that fight infections. (nih.gov)

Expression-gene mapping, network analyses and chromatin looping nominated candidate disease-relevant target genes modulated by these daSNVs. (stanford.edu)
The genetic study showed promising results in "silencing" the genes that translate into tau protein, the primary component behind dementia. (medicaldaily.com)

The disease is progressive with markedly elevated levels of EBV DNA in the blood and infiltration of organs by EBV-positive lymphocytes. (frontiersin.org)

We are encouraged by the expansion and persistence of the CAR T-cells, as well as the deep and durable responses with a manageable safety profile we've seen for bb2121 to date," said senior author and principal investigator James N. Kochenderfer, M.D., Experimental Transplantation and Immunology Branch, National Cancer Institute Center for Cancer Research. (eurekalert.org)

PTLDs are an uncommon but serious complication of immunosuppressive therapy after both solid organ and hematopoietic stem cell transplantation. (medscape.com)
Hematopoietic blood stem cell transplantation-a cell therapy-was first explored in humans in the 1950s and is currently a well-established treatment for blood diseases. (pharmaceuticalcommerce.com)
Stem cell-derived cell transplantation in the eye is one therapy being explored for inherited retinal degenerations such as retinitis pigmentosa (RP). (nature.com)
A cell therapy approach to restore microglial Trem2 function in a mouse model of Alzheimer's disease. (stanford.edu)
To date, over 10 advanced cell therapy products have been approved -- the majority of which are T-cell-based and strictly autologous. (scienceboard.net)
In this review, we summarized the progression of the neuroimmune response in PD based on recent studies and focused on the use of mesenchymal stem cell (MSC) therapy and challenges as a strategy of disease-modifying therapy with multiple targets. (biomedcentral.com)
The overall aim of this thesis has been to assess the potential of autologous grafting in cell replacement therapy for PD. (lu.se)

For example, loss of function of the microglial gene Trem2 renders microglia dysfunctional and causes an early-onset neurodegenerative syndrome, and Trem2 variants are among the strongest genetic risk factors for AD. (stanford.edu)
Here, we show that systemic hematopoietic cell transplantation followed by enhancement of microglia replacement restores microglial function in a Trem2 mutant mouse model of AD. (stanford.edu)

Some of these techniques include novel tools for genetic manipulation, 4,5 approaches for in vitro disease modelling 6-9 and innovative co-culture system with autologous cell types 10,11 or bacteria, 12-14 as well as viral infection models. (stemcell.com)

However, patient-derived cells may be more prone to develop disease-associated pathology after grafting. (lu.se)
However, since the patient-derived cells have a disease background it could be so that they develop disease-associated phenotypes and degenerate over time. (lu.se)
My projects investigated whether patient-derived cells are more prone to develop disease phenotypes and what can be done to protect these cells after transplantation," explained Fredrik. (lu.se)

This article focuses primarily on PTLD, which occurs in the context of solid organ and hematopoietic stem cell transplantation. (medscape.com)

Anatomy19

Organisms8

Diseases50

Chemicals and Drugs30

Analytical, Diagnostic and Therapeutic Techniques and Equipment92

Phenomena and Processes33

Disciplines and Occupations5

Humanities2

Information Science7

Named Groups2

Health Care24

Hematopoietic stem3

Allogeneic8

Organ transplantation8

Therapies12

Clinical19

Graft versus2

Bone9

Progenitor cells1

IPSCs17

Tissue12

Tissues7

Neurons8

IPSC-derived cells2

Genetically1

Stem Cell Trans1

Neural4

Epithelial2

Gene therapy7

Patients16

Genome2

Infectious Diseases1

Chronic10

Mesenchymal stem1

Mice2

Pluripotent Stem5

Parkinson's5

Outcomes1

Differentiation5

Hematological3

Chemotherapy1

Therapeutic3

Inflammatory5

Mutation2

Cellular therapy1

Undergone1

Hematopoiesis1

Immune-mediated2

Donors1

Monogenic diseases1

Fibroblasts3

Centers1

Genetics2

Diabetes3

Severe2

Genes2

Organs1

Experimental1

Therapy7

Microglia2

Vitro1

Patient-derived3

Context1