Topics

PubMed Journals Articles About "Affordable Sickle Cell Disease Gene Therapies" - Page: 20 RSS

18:02 EST 28th January 2020 | BioPortfolio

Affordable Sickle Cell Disease Gene Therapies PubMed articles on BioPortfolio. Our PubMed references draw on over 21 million records from the medical literature. Here you can see the latest Affordable Sickle Cell Disease Gene Therapies articles that have been published worldwide.

More Information about "Affordable Sickle Cell Disease Gene Therapies" on BioPortfolio

We have published hundreds of Affordable Sickle Cell Disease Gene Therapies news stories on BioPortfolio along with dozens of Affordable Sickle Cell Disease Gene Therapies Clinical Trials and PubMed Articles about Affordable Sickle Cell Disease Gene Therapies for you to read. In addition to the medical data, news and clinical trials, BioPortfolio also has a large collection of Affordable Sickle Cell Disease Gene Therapies Companies in our database. You can also find out about relevant Affordable Sickle Cell Disease Gene Therapies Drugs and Medications on this site too.

Showing "Affordable sickle cell disease gene therapies" PubMed Articles 476–500 of 51,000+

Very early lineage-specific chimerism after reduced intensity stem cell transplantation is highly predictive of clinical outcome for patients with myeloid disease.

The importance of chimerism status in the very early period after hematopoietic stem cell transplantation is unclear. We determined PBMC and T-cell donor chimerism 50 days after transplantation and related this to disease relapse and overall survival.


Alzheimer's disease - history of failures - a short opinion on the contemporary status of research.

Over 110 years have passed since first description of Alzheimer's disease (AD). Despite intensive research, AD remains incurable. The vast majority of clinical trials on new therapies aimed at suppressing undesirable aggregation of β-amyloid have failed. This requires rethinking of the strategy to fight AD. A lot of evidence supports the maintenance of β-amyloid as the central object of AD pathology, however, its aggregation into larger supramolecular structures is probably an intermediate stage and not t...

PRPF4 is a novel therapeutic target for the treatment of breast cancer by influencing growth, migration, invasion, and apoptosis of breast cancer cells via p38 MAPK signaling pathway.

Pre-mRNA processing factor 4 (PRPF4), a core protein in U4/U6 snRNP, maintains snRNP structures by interacting with PRPF3 and cyclophilin H. Expression of the PRPF4 gene affects cell survival as well as apoptosis and is responsible for retinitis pigmentosa (RP). Proteomics analysis shows that PRPF4 may be a therapeutic target in human cancers. Nevertheless, the exact function and role of the PRPF4 gene are unclear. In this study, we assessed the expression of PRPF4 gene in human breast cancer cells. First, ...


Multigene vector delivery with herpes simplex virus 1 amplicons.

Gene expression studies and gene therapy require efficient gene delivery into cells. Different technologies by viral and non-viral mechanisms have been used for gene delivery into cells. Small gene vectors transfer across the cell membrane with a relatively high efficiency, but not large genes or entire loci spanning several kilobases, which do not remain intact following introduction. Previously, we developed an efficient delivery system based on herpes virus simplex type 1 (HSV-1) amplicons to transfer la...

Cerebrospinal fluid and serum glycosphingolipid biomarkers in canine globoid cell leukodystrophy (Krabbe Disease).

Globoid cell leukodystrophy (GLD, Krabbe disease, Krabbe's disease) is caused by genetic mutations in the gene encoding, galactosylceramidase (GALC). Deficiency of this enzyme results in central and peripheral nervous system pathology, and is characterized by loss of myelin and an infiltration of globoid cells. The canine model of GLD provides a translational model which faithfully recapitulates much of the human disease pathology. Targeted lipidomic analysis was conducted in serum and cerebrospinal fluid (...

Molecular Portraits of Early Rheumatoid Arthritis Identify Clinical and Treatment Response Phenotypes.

There is a current imperative to unravel the hierarchy of molecular pathways that drive the transition of early to established disease in rheumatoid arthritis (RA). Herein, we report a comprehensive RNA sequencing analysis of the molecular pathways that drive early RA progression in the disease tissue (synovium), comparing matched peripheral blood RNA-seq in a large cohort of early treatment-naive patients, namely, the Pathobiology of Early Arthritis Cohort (PEAC). We developed a data exploration website (h...

Three-dimensional chromosome architecture and drug addiction.

Aberrant gene expression underlies drug addiction. Therefore, studying the regulation of gene expression in drug addiction may provide mechanistic insights into this disease, for which there are still only limited treatments. Recently, the three-dimensional (3D) organization of linear DNA in the nucleus has been recognized as having a major influence on gene transcription. Here, we review its roles in both basic brain function and neuropsychiatric disorders, while also highlighting its emerging implications...

Coverage, Financial Burden, and the Patient Protection and Affordable Care Act for Patients With Cancer.

Evidence suggests coverage has improved significantly for patients with cancer, particularly in the lower-income population, after the implementation of the Affordable Care Act (ACA). Yet no study has examined changes in type of coverage or the resulting effect on spending and financial burden.

miR-99b-3p is induced by vitamin D3 and contributes to its antiproliferative effects in gastric cancer cells by targeting HoxD3.

Vitamin D3 is known to have anticancer actions by affecting tumorigenesis including the cell cycle and cell apoptosis in gastric cancer (GC) cells; the genes including microRNAs (miRNAs) regulated by vitamin D3 signaling remain discovered. miR-99b-3p, the tumor suppressor gene, is not only decreased in GC tissues, but is also induced by vitamin D3 through the vitamin D receptor (VDR) binding on the promoter domain of miR-99b. Further study indicates that miR-99b-3p inhibits cell viability and induces cell a...

Nationwide retrospective review of hematopoietic stem cell transplantation in children with refractory Langerhans cell histiocytosis.

The efficacy of and indications for hematopoietic stem cell transplantation (HSCT) in pediatric Langerhans cell histiocytosis (LCH) remain undetermined. This retrospective study analyzed 30 children with refractory LCH who underwent HSCT in Japan between 1996 and 2014. Eleven patients received a myeloablative conditioning (MAC) regimen, while 19 patients received a reduced-intensity conditioning (RIC) regimen. Among the 26 patients with complete data, 23 patients had risk organ (RO) involvement during clini...

Type-I interferons in atherosclerosis.

The contribution of dyslipidemia and inflammation in atherosclerosis is well established. Along with effective lipid-lowering treatments, the recent success of clinical trials with anti-inflammatory therapies and the accelerated atherosclerosis in many autoimmune diseases suggest that targeting inflammation may open new avenues for the prevention and the treatment for cardiovascular diseases (CVDs). In the past decades, studies have widened the role of type-I interferons (IFNs) in disease, from antivirus de...

Alzheimer's Disease: key developments support promising perspectives for therapy.

Alzheimer's is the neurodegenerative disease affecting the largest number of patients in the world. In spite of the intense research of the last decades, progress about its knowledge and therapy was limited. In particular, various cytotoxic processes remained debated, while the few drugs approved for therapy were of only marginal relevance. Recent studies have identified key aspects of the disease, such as the mechanisms governing the development of pathology. In order to operate the Aβ peptide, known as t...

Bone Marrow Necrosis in a Patient Following Blinatumomab Therapy.

Bone marrow necrosis (BMN) is an extremely rare condition characterized by necrosis of the myeloid tissue and medullary stroma leaving an amorphous eosinophilic background and ill-defined necrotic cells in the hematopoietic bone marrow. Several conditions are associated with BMN, including sickle cell disease, metastatic carcinoma, and hematological malignancies. It is also associated with the use of antineoplastic drugs, such as fludarabine, interferon alpha, and imatinib. Blinatumomab is a CD19/CD3 bispec...

Illuminating biological pathways for drug targeting in head and neck squamous cell carcinoma.

Head and neck squamous cell carcinoma (HNSCC) remains a morbid disease with poor prognosis and treatment that typically leaves patients with permanent damage to critical functions such as eating and talking. Currently only three targeted therapies are FDA approved for use in HNSCC, two of which are recently approved immunotherapies. In this work, we identify biological pathways involved with this disease that could potentially be targeted by current FDA approved cancer drugs and thereby expand the pool of p...

A Human REPIN1 Gene Variant: Genetic Risk Factor for the Development of Nonalcoholic Fatty Liver Disease.

We tested the hypothesis that a genetic deletion (Del) variant in the REPIN1 gene is associated with the severity of nonalcoholic fatty liver disease (NAFLD) in humans.

Monocyte glycolysis determines CD8+ T-cell functionality in human Chagas disease.

Chagas disease is a life-long pathology resulting from Trypanosoma cruzi infection. It represents one of the most frequent causes of heart failure and sudden death in Latin America. Herein we provide evidence that aerobic glycolytic pathway activation in monocytes drives nitric oxide (NO) production, triggering tyrosine nitration (TN) on CD8 T cells and dysfunction in patients with chronic Chagas disease. Monocytes from patients exhibited higher frequency of hypoxia inducible factor (HIF)-1α and increased ...

Follicular lymphoma: Update on management and emerging therapies at the dawn of the new decade.

Follicular lymphoma is the most common indolent non-Hodgkin lymphoma. Survival has improved over the last several decades, mainly due to the incorporation of the anti-CD20 antibody rituximab into pre-existing or rediscovered agents. The disease has a relapsing and remitting pattern, coupled with a risk of transformation into an aggressive lymphoma, and considered incurable for most patients. Next generation sequencing technologies have increased our understanding of the biology and genetic landscape of the ...

MicroRNA-144 Regulates Cardiomyocyte Proliferation and Apoptosis by Targeting TBX1 through the JAK2/STAT1 Pathway.

It is currently believed that the TBX1 gene is one of the core genes of congenital heart disease (CHD). However, there are few studies on the abnormal regulation of TBX1 gene expression. The purpose of this work was to investigate the role of miR-144 and TBX1 in cardiac development by studying the regulatory relationship and mechanism of miR-144 on TBX1/JAK2/STAT1 in cardiomyocytes. Cell proliferation was detected by MTT and clone formation assay and cell cycle and apoptosis by flow cytometry. The levels of...

Breast milk stem cells survive in the neonate's gut, enter into the neonate circulation and are adapted into the body.

The stem cell exchange during pregnancy is thought to remain chimeras for life. Few studies recently revealed that maternal transfer of viable stem cells to the offspring continues even after birth during breastfeeding. Some of these stem cells are likely to integrate into different organs (brain, blood, kidneys, and pancreas), including neurons and insulin-producing cells in the pancreas to become functional cells. This finding opens a new avenue for research on therapeutic uses of breast milk-derived stem...

The study of genes and signal transduction pathways involved in mustard lung injury: A gene therapy approach.

Sulfur mustard (SM) is a destructive and harmful chemical agent for the eyes, skin and lungs that causes short-term and long-term lesions and was widely used in Iraq war against Iran (1980-1988). SM causes DNA damages, oxidative stress, and Inflammation. Considering the similarities between SM and COPD (Chronic Obstructive Pulmonary Disease) pathogens and limited available treatments, a novel therapeutic approach is not developed. Gene therapy is a novel therapeutic approach that uses genetic engineering sc...

Development and verification of a nomogram for prediction of recurrence-free survival in clear cell renal cell carcinoma.

Nowadays, gene expression profiling has been widely used in screening out prognostic biomarkers in numerous kinds of carcinoma. Our studies attempt to construct a clinical nomogram which combines risk gene signature and clinical features for individual recurrent risk assessment and offer personalized managements for clear cell renal cell carcinoma. A total of 580 differentially expressed genes (DEGs) were identified via microarray. Functional analysis revealed that DEGs are of fundamental importance in ccRC...

Effects of Porcine STC-1 on Cell Metabolism and Mitochondrial Function.

Stanniocalcin (STC-1), a kind of glycoprotein hormone, was first found in fish and mainly regulates calcium/phosphorus metabolism in the body. To explore the biological function of the porcine STC-1 gene, the effects of changes in stanniocalcin expression on cellular metabolism and mitochondrial function were studied. A vector overexpressing the STC-1 gene and an siRNA silencer of the STC-1 gene were transfected into porcine kidney epithelial PK15 cells. After the STC-1 gene expression level was induced to ...

Single-Cell Signature Explorer for comprehensive visualization of single cell signatures across scRNA-seq datasets.

The momentum of scRNA-seq datasets prompts for simple and powerful tools exploring their meaningful signatures. Here we present Single-Cell_Signature_Explorer (https://sites.google.com/site/fredsoftwares/products/single-cell-signature-explorer), the first method for qualitative and high-throughput scoring of any gene set-based signature at the single cell level and its visualization using t-SNE or UMAP. By scanning datasets for single or combined signatures, it rapidly maps any multi-gene feature, exemplifi...

The contribution of multicellular model organisms to neuronal ceroid lipofuscinosis research.

The NCLs (neuronal ceroid lipofuscinosis) are forms of neurodegenerative disease that affect people of all ages and ethnicities but are most prevalent in children. Commonly known as Batten disease, this debilitating neurological disorder is comprised of 13 different subtypes that are categorized based on the particular gene that is mutated (CLN1-8, CLN10-14). The pathological mechanisms underlying the NCLs are not well understood due to our poor understanding of the functions of NCL proteins. Only one speci...

p.G360R Is a Pathogenic GLA Gene Mutation Responsible for a Classic Phenotype of Fabry Disease.

The authors report the case of a classic phenotype of Fabry disease in a 60-year-old male patient presenting with left ventricular hypertrophy and stroke. Genetic analysis revealed 2 GLA-gene variants, i.e., p.R356Q and p.G360R. This clinical case highlights that the finding of 2 or more GLA gene variants in a Fabry patient should lead to a careful evaluation in order to determine their exact role in the condition. This case also provides the first clinical evidence that the p.G360R mutation is pathogenic a...


Quick Search