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New drug regimen is safe and effective in children with cystic fibrosis

An international, open-label Phase 3 study, co-led by Susanna McColley, MD, from Ann & Robert H. Lurie Children's Hospital of Chicago, found that a regimen of three drugs (elexacaftor/tezacaftor/ivacaftor) that targets the genetic cause of cystic fibrosis was safe and effective in 6-11-year-olds with at least one copy of F508del mutation in the CFTR gene, which is estimated to represent almost 90 percent of the cystic fibrosis population in the United States.

19 Mar 2021

Scientists use clinical data to study the molecular groups of atypical teratoid rhabdoid tumor

Atypical teratoid rhabdoid tumor (ATRT) is a rare brain tumor that predominantly occurs in young children. Scientists at St. Jude Children's Research Hospital used data from two clinical trials to study the molecular groups of ATRT and correlate them with clinical outcomes.

19 Mar 2021

Rare pulmonary disease discovered in rural Alberta

A rare pulmonary disease that is linked to bats has made Alberta home, according to new research led by provincial lab scientists.

19 Mar 2021

Malfunctioning blood-brain barrier is linked to benign forgetfulness of aging

Have you forgotten where you laid your keys? Ever wondered where you had parked your car? Or having trouble remembering the name of the new neighbor? Unfortunately, these things seem to get worse as one gets older.

19 Mar 2021

Chemical cocktail shows promise for producing muscle stem cells

A UCLA-led research team has identified a chemical cocktail that enables the production of large numbers of muscle stem cells, which can self-renew and give rise to all types of skeletal muscle cells.

19 Mar 2021

Study reveals biomarkers for the prognosis of gastric adenocarcinoma

Low plasma levels of protein TGFB1 and polymorphisms in gene TGFB1 act as biomarkers for the prognosis of gastric adenocarcinoma, according to a study led by the University Complutense of Madrid.

18 Mar 2021

Study explores how CAR T immunotherapy could be used to treat solid tumors

A study recently published in the journal Science looked into how CAR T immunotherapy could be used to treat solid tumors in addition to leukemias. The programming of CAR T cells opens avenues for applying cell therapies to, for example, breast cancer or ovarian cancer in the future.

18 Mar 2021

Study investigates how genetic variants contribute to increased risk of lung cancer

Lung cancer is the leading cause of cancer death in the U.S. for both men and women. While risk for this disease can be influenced by environmental and lifestyle factors like smoking, studies estimate that 18% of lung cancer cases are due to inherited genetic variants.

18 Mar 2021

Anti-inflammatory therapies may help prevent heart disease in people with age-related blood condition

Therapies that soothe inflammation could be an effective way to prevent heart disease in people with a common age-related blood condition, according to a new study from researchers at Columbia University Vagelos College of Physicians and Surgeons.

18 Mar 2021

Researchers identify inflammatory mechanism related to bone damage in rheumatoid arthritis

In a study aimed at investigating the mechanism responsible for exacerbating rheumatoid arthritis in smokers, researchers at the Center for Research on Inflammatory Diseases, linked to the University of São Paulo in Brazil, discovered a novel path in the inflammatory process associated with the bone damage caused by rheumatoid arthritis.

18 Mar 2021

Novel cancer-fighting gene therapy enters phase I clinical trial

Researchers from the MDC and Charité have developed a new gene therapy based around a special T-cell receptor that helps the immune system effectively recognize and fight cancer cells.

18 Mar 2021

Study confirms that higher level of maternal folate increases the child’s birthweight

Imagine that you are going to conduct research on how different factors affect a child in the womb. Bad habits such as smoking, drinking alcohol or poor eating habits are factors that may affect the child.

18 Mar 2021

Gene expression of viral entry factors may explain the differences of COVID-19 severity

A new study uncovers which cell types can be infected by SARS-CoV-2 due to their viral entry factors. The study also suggests that increased gene expression of these viral entry factors in some individuals partially explains the differences of COVID-19 severity reported in relation to age, gender and smoking status.

18 Mar 2021

Study shows how coronavirus variants spread more quickly

The fast-spreading UK, South Africa, and Brazil coronavirus variants are raising both concerns and questions about whether COVID-19 vaccines will protect against them.

18 Mar 2021

Long noncoding RNA plays a vital role in mobilizing immune response after organ transplantation

A long noncoding RNA whose function was previously unknown turns out to play a vital role in mobilizing the immune response following a bone marrow transplant or solid organ transplantation.

17 Mar 2021

A complete genetic and clinical analysis of individuals with SCN2A-related disorders

Researchers from Children's Hospital of Philadelphia affiliated with the CHOP Epilepsy Neurogenetics Initiative (ENGIN) have compiled a complete genetic and clinical analysis of more than 400 individuals with SCN2A-related disorder, which has been linked to a variety of neurodevelopmental disorders, including epilepsy and autism.

17 Mar 2021

New method for growing embryos outside the womb

To observe how a tiny ball of identical cells on its way to becoming a mammalian embryo first attaches to an awaiting uterine wall and then develops into nervous system, heart, stomach and limbs: This has been a highly-sought grail in the field of embryonic development for nearly 100 years.

17 Mar 2021

Researchers find a better way to identify cells and understand the mechanisms of diseases

In researching the causes and potential treatments for degenerative conditions such as Alzheimer's or Parkinson's disease, neuroscientists frequently struggle to accurately identify cells needed to understand brain activity that gives rise to behavior changes such as declining memory or impaired balance and tremors.

17 Mar 2021

Researchers explore potential role of TARM1 protein in pathogenesis of rheumatoid arthritis

TARM1 is a receptor protein whose role in the functioning of the immune system is unknown. In a new study, scientists from Japan have explored the potential role of TARM1 in the pathogenesis of rheumatoid arthritis by analyzing mouse models.

17 Mar 2021

Insights into elusive protein complex can help find novel treatments for chromosomal disorders

The cells in our body are constantly fighting off the threat of cancer by repairing damaged DNA. In a new study, scientists from Tokyo University of Science investigate the structure of an elusive protein complex that plays a key role in the activation of the "Fanconi anemia pathway" involved in DNA repair, and report on the factors governing its stability.

17 Mar 2021

Genetic News and Research

New drug regimen is safe and effective in children with cystic fibrosis

Scientists use clinical data to study the molecular groups of atypical teratoid rhabdoid tumor

Rare pulmonary disease discovered in rural Alberta

Malfunctioning blood-brain barrier is linked to benign forgetfulness of aging

Chemical cocktail shows promise for producing muscle stem cells

Study reveals biomarkers for the prognosis of gastric adenocarcinoma

Study explores how CAR T immunotherapy could be used to treat solid tumors

Study investigates how genetic variants contribute to increased risk of lung cancer

Anti-inflammatory therapies may help prevent heart disease in people with age-related blood condition

Researchers identify inflammatory mechanism related to bone damage in rheumatoid arthritis

Novel cancer-fighting gene therapy enters phase I clinical trial

Study confirms that higher level of maternal folate increases the child’s birthweight

Gene expression of viral entry factors may explain the differences of COVID-19 severity

Study shows how coronavirus variants spread more quickly

Long noncoding RNA plays a vital role in mobilizing immune response after organ transplantation

A complete genetic and clinical analysis of individuals with SCN2A-related disorders

New method for growing embryos outside the womb

Researchers find a better way to identify cells and understand the mechanisms of diseases

Researchers explore potential role of TARM1 protein in pathogenesis of rheumatoid arthritis

Insights into elusive protein complex can help find novel treatments for chromosomal disorders

Sequencing & Genomics: Elevating Science and Healthcare eBook

Bringing real-time analytics to downstream bioprocess monitoring

Designing fluidic architecture for scalable diagnostic platforms

Evaluating new approaches for rare disease genomics

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