Sentences with phrase «patient gene in a cell»

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The companies» R&D will focus on on a gene mutation present in a wide swath of patients with ALS, a degenerative nervous system disease that eats away at nerve cells and weakens muscles.

Risk Versus Reward: The Value of Cell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developmCell Therapy for Patients and Investors Source: Streetwise Reports (4/25/18) The cell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developmcell therapy space, encompassing disruptive new treatment including stem cell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage developmcell therapy, immunotherapy and gene editing, has begun to mature, with a handful of product approvals and others in late - stage development.

In this special section of Science, expert contributors retrace the long and tortuous path leading to the mapping and identification of the BRCA1 gene; discuss the ways in which BRCA mutation status has been integrated into the clinical management of patients in high - risk families; and highlight the role of the BRCA proteins in preserving the structural and numerical integrity of chromosomes throughout the cell cycle, a function that may explain their tumor suppressor activitIn this special section of Science, expert contributors retrace the long and tortuous path leading to the mapping and identification of the BRCA1 gene; discuss the ways in which BRCA mutation status has been integrated into the clinical management of patients in high - risk families; and highlight the role of the BRCA proteins in preserving the structural and numerical integrity of chromosomes throughout the cell cycle, a function that may explain their tumor suppressor activitin which BRCA mutation status has been integrated into the clinical management of patients in high - risk families; and highlight the role of the BRCA proteins in preserving the structural and numerical integrity of chromosomes throughout the cell cycle, a function that may explain their tumor suppressor activitin high - risk families; and highlight the role of the BRCA proteins in preserving the structural and numerical integrity of chromosomes throughout the cell cycle, a function that may explain their tumor suppressor activitin preserving the structural and numerical integrity of chromosomes throughout the cell cycle, a function that may explain their tumor suppressor activity.

Researchers from Duke University had previously used CRISPR to correct genetic mutations in cultured cells from Duchenne patients, and other labs had corrected genes in single - cell embryos in a laboratory environment.

«Fibroblast growth factor receptor inhibitors are new therapies being developed in clinical trials for patients whose cancer cells have genetic alterations in this family of genes,» says Roychowdhury, a member of the OSUCCC — James Translational Therapeutics Program.

Novel abnormalities in the FGFR gene, called FGFR fusions, were identified in a spectrum of cancers, and preliminary results with cancer cells harboring FGFR fusions suggested that some patients with these cancers may benefit from treatment with FGFR inhibitor drugs, according to data published in Cancer Discovery, a journal of the American Association for Cancer Research.

Since patients (and mice) with Usher 1c also have balance problems caused by hair - cell damage in the vestibular organs, the researchers also tested whether gene therapy restored balance.

The gene - edited cells will then be multiplied in the lab and re-introduced into the patient's bloodstream.

Lu's team will extract immune cells called T cells from the blood of the enrolled patients, and then use CRISPR — Cas9 technology — which pairs a molecular guide able to identify specific genetic sequences on a chromosome with an enzyme that can snip the chromosome at that spot — to knock out a gene in the cells.

Today's findings augment recent research also published in Nature (Dec. 7, 2016) detailing the team's development of a «stemness biomarker» — a 17 - gene signature derived from leukemia stem cells that can predict at diagnosis which AML patients will respond to standard treatment.

So we sequenced a gene involved in cell growth and found a correlation in about 85 percent of the patients: If you had a certain mutation in the EGFR gene, you responded to the drug; if you didn't have the mutation, you didn't.

«This suggests that Runx2 levels in myeloma cells may be a gene predictor of a patient's prognosis, good or bad,» Yang said.

Whether investigating fat cells, immunotherapy or use of the CRISPR - Cas 9 gene - editing tool, which a federal panel recently approved for a select number of patients suffering from three types of cancers, including multiple myeloma, approaches beyond attacking cancer cells are needed in the fight against many cancers.

By providing a woman's family history of these cancers, including the ages they were diagnosed, the programs calculate a probability that the patient carries a harmful mutation in BRCA1 or BRCA2 (genes involved in controlling malignant cell growth).

Another problem with Kobinger's approach is that patients will require repeated treatments, because the added gene ends up in surface cells that die off after a few months.

CTL119 manufacturing begins with a patient's own T cells, some of which are removed and then reprogrammed in Penn's Clinical Cell and Vaccine Production Facility with a gene transfer technique designed to teach the T cells to target and kill tumor cells.

Researchers at the Center for Cell and Gene Therapy at Baylor College of Medicine, Texas Children's Hospital and Houston Methodist have developed an alternative treatment in which virus - specific cells protect patients against severe, drug - resistant viral infections.

Among patients with non-small cell lung cancer (NSCLC) fueled by ALK gene alterations who were being treated with crizotinib (Xalkori), a decrease in the number of circulating tumor cells (CTCs) harboring increased copies of the ALK gene over the first two months of treatment was associated with increased progression - free survival.

The team integrated three, complementary gene sequencing approaches to look for mutations in tumor cells from SS patients: whole - genome sequencing in six subjects, sequencing of all protein - coding regions (exomes) in 66 subjects, and comparing variation in the number of copies of all genes across the genome in 80 subjects.

Although the correct gene did manufacture Factor 9 in the patients for a few weeks, soon their immune systems had wiped out the new cells containing AAV — and the precious gene for Factor 9.

When Fishel and Kolodner heard of the accumulation of mutations in cancer cells from patients with familial colon cancer, they suspected that the gene responsible would be similar to the bacterial and yeast genes they had studied.

Some patients with non-small cell lung cancer (NSCLC) have changes in the anaplastic lymphoma kinase (ALK) gene, which can drive the development of their cancer.

Among patients with advanced non-small cell lung cancer without a mutation of a certain gene (EGFR), conventional chemotherapy, compared with treatment using epidermal growth factor receptor tyrosine kinase inhibitors, was associated with improvement in survival without progression of the cancer, but not with overall survival, according to a study in the April 9 issue of JAMA.

The Epigenetics Research group used these cells to perform genome - wide profiling across more than 20,000 individual genes in these patients.

When the team measured gene expression first in the stem cells, and then re-evaluated the cells once they had become neurons, very specific differences emerged between the cells derived from bipolar disorder patients and those without the condition.

In eight of the patients, normal cells showed one mutated NF1 gene and one normal gene, while cancerous cells had only the mutated form.

Epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) are the preferred treatment option for patients with advanced non-small cell lung cancer (NSCLC) who have mutations in the EGFR gene.

«If you look at a set of lung cancer patients, like we did in the paper, who develop brain metastases, they all have those two genes in their primary lung cancer,» said Sheila Singh, the study's supervisor, associate professor at the Michael G. DeGroote School of Medicine, scientist with the Stem Cell and Cancer Research Institute at McMaster University and neurosurgeon at McMaster Children's Hospital.

In recent studies of cancer patients who received a bone marrow transplant, genes from the marrow's white blood cells were found in the patient's tumor cellIn recent studies of cancer patients who received a bone marrow transplant, genes from the marrow's white blood cells were found in the patient's tumor cellin the patient's tumor cells.

Further research could test these cancer stem cell gene expression at the RNA and protein level in circulating tumor cells and biopsies from patients on trial.

By performing a genome - wide screen in breast cancer cells, Dr. Oesterreich and her colleagues identified a gene called HOXC10 as one that the cancer seems to modify to allow continued tumor growth in patients whose cancer becomes resistant to traditional therapies.

A new gene therapy treatment has restored some sight in a handful of blind patients suffering from Leber's congenital amaurosis, a syndrome in which, because of a broken or missing gene called RPE65, retinal photoreceptor cells malfunction and eventually die.

Surprisingly, they found that although the patterns of gene expression — as shown by the RNA sequencing — differed between the hepatocellular carcinomas and the liver cancers with biliary phenotype and depended on the histological type, the overall pattern of mutations in the cells was actually similar between the tumors — of either type — that had emerged in patients who had had infections with either hepatitis C or B, and were different in patients without such infections.

About half of melanoma patients harbor an identical tumor - specific mutation in the BRAF gene, which encodes a protein kinase that helps drive cell growth.

Examination of gene expression in patients with non-small cell lung cancer (NSCLC) showed the area adjacent to tumors is rich with cancer markers.

The gene mutations driving cancer have been tracked for the first time in patients back to a distinct set of cells at the root of cancer — cancer stem cells.

The investigators also measured gene expression of postvaccinated cervical cells in three of the patients and found increased expression of several genes (CXCR3, Tbet and IFNβ) associated with activation of the immune system.

Dr. Eric Olson (right) shows the dystrophin protein (red) produced in gene - edited heart muscle cells taken from a DMD patient's blood.

Some of those patients have already seen firsthand how gene editing can correct defects in their cells.

MammaPrint surveys 70 genes in tumor cells, checking whether they're turned on or off in an individual patient.

Lee says the research team will measure the amount of methylation in patients» cells when they begin their treatment and the presence of genes associated with irinotecan resistance, among other possible biomarkers.

Easily accessible from nasal biopsies, these cells — which belong to nerve tissues and can differentiate into neurons — constitute an interesting model to identify the genes and proteins whose expression is deregulated in patients with ASD.

In the study, which began in 2011, an adeno - associated virus vector was used to carry a functional copy of the affected gene to muscle cells in the diaphragms of nine patientIn the study, which began in 2011, an adeno - associated virus vector was used to carry a functional copy of the affected gene to muscle cells in the diaphragms of nine patientin 2011, an adeno - associated virus vector was used to carry a functional copy of the affected gene to muscle cells in the diaphragms of nine patientin the diaphragms of nine patients.

In 2006, researchers discovered the genetic defect behind FOP: A mutated version of the gene ACVR1, which in patients produces an overactive form of a cell surface protein called a transmembrane receptoIn 2006, researchers discovered the genetic defect behind FOP: A mutated version of the gene ACVR1, which in patients produces an overactive form of a cell surface protein called a transmembrane receptoin patients produces an overactive form of a cell surface protein called a transmembrane receptor.

The method, successfully tested in heart muscle cells from patients, offers an efficient alternative to the daunting task of developing an individualized molecular treatment for each gene mutation that causes DMD.

The patients had malfunctioning RPE65 genes, which code for a protein that ensures the photoreceptor cells in the retina work smoothly.

To treat hereditary blood diseases, doctors could take a sample of bone marrow cells from a patient, correct the faulty gene, and then grow healthy cells in the bioreactor and transplant them back into the patient.

Using biopsies of the patients» tumors collected before the start of treatment and at the time patients developed resistance, the researchers performed large - scale genomic analyses to search for mutations specific to the cancer cells in all of each patient's 20,000 genes.

Hutchinson - Gilford progeria is caused by a spontaneous mutation during conception in a gene called LMNA, which encodes a protein called prelamin A. Progeria patients experience a buildup of an abnormal version of prelamin A in their cells that, among other changes, distorts the nucleus and alters gene expression.

In response to flu virus, the activity of type I and type III interferon genes was abnormally low in the patient's dendritic cells, the team reports online today in SciencIn response to flu virus, the activity of type I and type III interferon genes was abnormally low in the patient's dendritic cells, the team reports online today in Sciencin the patient's dendritic cells, the team reports online today in Sciencin Science.