Gene therapy for thalassemia ends need for transfusions in young children

Over 90 percent of patients with transfusion-dependent thalassemia, an inherited blood disorder, no longer needed monthly blood transfusions years after receiving gene therapy, according to an international Phase 3 clinical trial that for the first time included children younger than 12 years of age. Twenty-two patients were evaluated (ranging in age 4-34 years), including pediatric patients.

Quelle: Sciencedaily

T-cell responses may help predict protection against SARS-CoV-2 infection in individuals with and without cancer

T-cell responses directed against the receptor-binding domain of the SARS-CoV-2 spike protein were associated with protection from SARS-CoV-2 infection in vaccinated individuals with or without cancer, with lower T-cell responses observed in patients with blood cancers, according to a new study.

Quelle: Sciencedaily

Defeating leukemia cells by depriving them of energy

Acute myeloid leukemia, which affects blood and bone marrow cells, is a particularly dangerous form of cancer. More than half of patients under the age of 60 die. This proportion rises to 85% for patients over 60. A team has now identified a previously unknown mechanism that could lead to the development of new therapies. The selective activation of AMPK, a key enzyme in the energy balance of tumor cells, would indeed lead to their death by triggering the cells stress response. Moreover, the scientists have successfully exploited this energy gap in an animal model of the disease: a combination of two drugs — one of which is already on the market — has indeed shown promise. However, their effectiveness has yet to be confirmed on leukaemia stem cells, which have the ability to escape many treatments to restart tumor growth.

Quelle: Sciencedaily

Researchers identify biomarkers to predict patient response to immunotherapy treatment for melanoma

A team of researchers has discovered blood biomarkers that can potentially predict patient response in the treatment of melanoma.

Quelle: Sciencedaily

Metabolism found to regulate production of killer cells

Researchers have discovered that metabolic changes affect how blood cells are formed during embryonic development. They found a previously unknown metabolic switch with a key role in how different types of blood cells develop. This means blood cell formation can be directed towards producing natural killer cells in the laboratory to ultimately be used in a new anti-cancer treatment.

Quelle: Sciencedaily

Identification of a unique ’switch‘ for blood vessel generation

Researchers discover a ’switch‘ specific to transcription factors that induce the genes essential for blood vessel development in postnatal periods. Mouse models further showed that the modifiers responsible for the switch are critical for postnatal angiogenesis.

Quelle: Sciencedaily

Suppressing the spread of tumors

When tumors spread, cancer cells migrate to other parts of the body through the blood or lymphatic vessels. Scientists have now found a new protein that prevents cancer cells from doing so by making them stick more tightly to their surroundings. Their findings could in the future help doctors determine the aggressiveness of a tumor and fine-tune the therapy.

Quelle: Sciencedaily

Researchers identify key regulator of blood stem cell development

A protein that masterminds the way DNA is wrapped within chromosomes has a major role in the healthy functioning of blood stem cells, which produce all blood cells in the body, according to a new study.

Quelle: Sciencedaily

New blood test combined with image-based prostate cancer screening reduces harms and costs

The combination of a novel blood test and magnetic resonance imaging (MRI) can reduce overdiagnosis of low-risk cancers as well as societal costs in prostate cancer screening, according to a cost-effectiveness study.

Quelle: Sciencedaily

Magnesium is essential for the immune system, including in the fight against cancer

The level of magnesium in the blood is an important factor in the immune system’s ability to tackle pathogens and cancer cells. Researchers have reported that T cells need a sufficient quantity of magnesium in order to operate efficiently. Their findings may have important implications for cancer patients.

Quelle: Sciencedaily

Obscure protein is spotlighted in fight against leukemia

Acute myeloid leukemia (AML) is a cancer of white blood cells. Researchers discovered that AML cancer cells depend on a protein called SCP4 to survive. They think the previously little-known protein is involved in a metabolic pathway the cancer cells need to survive. SCP4 provides researchers with a potential new therapeutic approach for this aggressive cancer.

Quelle: Sciencedaily

New graft strategy may improve outcomes for blood stem cell recipients

Removing one type of T cell from donor blood used for stem cell grafts could greatly reduce a serious complication called graft-versus-host disease in patients with leukemia, according to a new study.

Quelle: Sciencedaily

First time genome editing made possible on cells lining blood vessel walls

Researchers have developed a unique nanoparticle to deliver genome editing technology, including CRISPR/Cas9, to endothelial cells, which are cells that line blood vessel walls. This is the first time that vascular endothelial cells could be reached for genome editing, since the usual way to deliver CRISPR/Cas9 — through a virus — does not work for this cell type.

Quelle: Sciencedaily

Scientists retool CAR T cells to serve as ‘micropharmacies’ for cancer drugs

Immunotherapies called chimeric antigen receptor (CAR) T cells use genetically engineered versions of a patient’s own immune cells to fight cancer. These treatments have energized cancer care, especially for people with certain types of blood cancers. Now, scientists have developed new CAR T cells that can do something their predecessors cannot: Make drugs.

Quelle: Sciencedaily

Study confirms nutrient’s role in childhood blood cancer

A molecular building block of many animal proteins, the amino acid valine, plays a key role in cancerous growth seen in T cell acute lymphoblastic leukemia, a new study shows.

Quelle: Sciencedaily

Cannabis use could cause harmful drug interactions

Using cannabis alongside other drugs may come with a significant risk of harmful drug-drug interactions, new research suggests. The researchers looked at cannabinoids–a group of substances found in the cannabis plant — and their major metabolites found in cannabis users‘ blood and found that they interfere with two families of enzymes that help metabolize a wide range of drugs prescribed for a variety of conditions. As a result, either the drugs‘ positive effects might decrease or their negative effects might increase with too much building up in the body, causing unintended side effects such as toxicity or accidental overdose. While more research needs to be done, the authors said one early takeaway from these studies is that it’s important to be careful when using cannabis with other prescription drugs.

Quelle: Sciencedaily

Research reveals how aging cells can be an underlying cause of kidney damage

A study in mice has found that stress and tissue damage initiated by angiotensin II, a molecule that is known to increase blood pressure and stiffening in the linings of blood vessels, leads to cellular senescence, a process by which a cell ages and permanently stops dividing but does not die. Importantly, when the researchers eliminated senescent cells from the mice, tissues returned to a normal state in spite of a continued infusion of angiotensin II.

Quelle: Sciencedaily

For stem cells, bigger doesn’t mean better

A new study suggests that enlargement of stem cells contributes to age-related decline in function. The researchers found that blood stem cells, which are among the smallest cells in the body, lose their ability to perform their normal function — replenishing the body’s blood cells — as they grow larger. When the cells were restored to their usual size, they behaved normally again.

Quelle: Sciencedaily

Team engineers new way to get medication past blood-brain barrier

A team of researchers has developed a new technique to open the blood-brain barrier temporarily to deliver medication to the brain. Getting medication past the brain’s unique and protective blood vessels, known as the blood-brain barrier, is one of the biggest challenges in treating brain and central nervous system diseases, according to researchers. The technique uses light and nanoparticles to pry open temporarily these barriers — called tight junctions — to allow medication to reach its target.

Quelle: Sciencedaily

Noninvasive brain biopsy shows improved sensitivity in tumor detection

A team of researchers has developed a noninvasive diagnostic method that may one day replace the biopsy with a simple blood test.

Quelle: Sciencedaily

Landmark study points to source of rapid aging, chronic inflammation in people living with HIV

In a groundbreaking study of people living with HIV, researchers found that elusive white blood cells called neutrophils play a role in impaired T cell functions and counts, as well as the associated chronic inflammation that is common with the virus.

Quelle: Sciencedaily

Chemo helps breast cancer cells get their ‘foot in the door’ to the lungs

A new study adds to the evidence that chemotherapy enhances cancer’s spread beyond the primary tumor, showing how one chemo drug allows breast cancer cells to squeeze through and attach to blood vessel linings in the lungs.

Quelle: Sciencedaily

Researchers identify new drug target for blood cancer, potentially solid tumors

Researchers have shown for the first time how mutations affecting a cellular process called RNA splicing alter cells to develop myelodysplastic syndrome (MDS) and other hematologic malignancies and solid tumors.

Quelle: Sciencedaily

Cancer chemotherapy drug reverses Alzheimer’s symptoms in mice

A drug commonly used to treat cancer can restore memory and cognitive function in mice that display symptoms of Alzheimer’s disease, new research has found. The drug, Axitinib, inhibits growth of new blood vessels in the brain — a feature shared by both cancer tumors and Alzheimer’s disease. This hallmark represents a new target for Alzheimer’s therapies. Mice that underwent the therapy not only exhibited a reduction in blood vessels and other Alzheimer’s markers in their brains, they also performed remarkably well in tests designed to measure learning and memory.

Quelle: Sciencedaily

A global assessment of cancer genomic alterations in epigenetic mechanisms

Muhammad A Shah, Emily L Denton, Cheryl H Arrowsmith, Mathieu Lupien and Matthieu Schapira

Abstract

Background

The notion that epigenetic mechanisms may be central to cancer initiation and progression is supported by recent next-generation sequencing efforts revealing that genes involved in chromatin-mediated signaling are recurrently mutated in cancer patients.

Results

Here, we analyze mutational and transcriptional profiles from TCGA and the ICGC across a collection 441 chromatin factors and histones. Chromatin factors essential for rapid replication are frequently overexpressed, and those that maintain genome stability frequently mutated. We identify novel mutation hotspots such as K36M in histone H3.1, and uncover a general trend in which transcriptional profiles and somatic mutations in tumor samples favor increased transcriptionally repressive histone methylation, and defective chromatin remodeling.

Conclusions

This unbiased approach confirms previously published data, uncovers novel cancer-associated aberrations targeting epigenetic mechanisms, and justifies continued monitoring of chromatin-related alterations as a class, as more cancer types and distinct cancer stages are represented in cancer genomics data repositories.

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