Unraveling the 50-year mystery, gene therapy is expected to treat classic genetic diseases April 04, 2018 Source: WuXi PharmaTech Both sickle cell anemia and beta-thalassemia are hereditary anemia due to mutations in the HBB gene encoding the adult hemoglobin beta subunit. Patients with these diseases may need to receive blood transfusions or other treatments for life. Studies have shown that re-expressing fetal hemoglobin in adults can alleviate the symptoms of anemia. However, the gene encoding fetal hemoglobin ceased to be expressed after the birth of the fetus. In a recent study published in Nature Genetics, researchers at the University of New South Wales (UNSW) used CRISPR gene editing technology to add rare gene mutations in nature to genes encoding fetal hemoglobin. So that these genes can be expressed in adult patients. This finding has the potential to produce gene therapies for the treatment of multiple blood diseases due to mutations in the HBB gene. ▲Image source: 123RF Hemoglobin in patients with sickle cell anemia or beta-thalassemia cannot function properly with oxygen due to mutations in the HBB gene. However, a small number of patients with genes encoding fetal hemoglobin carry naturally occurring genetic mutations. These genetic mutations allow the fetal hemoglobin, which was originally stopped after birth, to continue to be expressed in the adult stage, thereby alleviating the symptoms of anemia. When researchers used CRISPR/Cas9 gene editing technology to add genetic mutations in nature to genes expressing fetal hemoglobin, they found that the gene-edited fetal hemoglobin gene was significantly increased in red blood cells. Further studies have found that proteins encoded by genes named BCL11A and ZBTB7A bind directly to the promoter region of the gene encoding fetal hemoglobin, thereby inhibiting the expression of fetal hemoglobin. Gene mutations that allow the sustained expression of fetal hemoglobin disrupt the binding of BCL11A and ZBTB7A proteins to the promoter region, thereby relieving inhibition of fetal hemoglobin expression. “In the past 50 years, scientists have been trying to find out why the fetal hemoglobin gene is shut down, and thus developing ways to restart these genes,†said the head of the study, Merlin of the UNSW School of Biotechnology and Biomolecular Sciences. Professor Crossley said: "Our research has solved this secret." “Our innovative approach can be said to be a pioneer in 'organic gene therapy,'†Dr. Crossley added. “The reason why it is organic is because we have not introduced any new DNA into the cell. We just introduced it through genetic engineering. There are already benign mutations in nature that can improve the symptoms of patients." "We expect it to be a safe and effective treatment and can be used to treat a range of common genetic blood diseases including thalassemia and sickle cell anemia." Gene therapy based on the use of CRISPR gene editing technology to increase fetal hemoglobin expression has entered the clinical stage. In December last year, CRISPR Therapeutics submitted to the FDA a clinical trial application for the treatment of beta-thalassemia with CTX001. CTX001 therapy collects patients' red blood cells, uses CRISPR gene editing technology to improve fetal hemoglobin expression, and then injects these genetically modified red blood cells into patients to achieve therapeutic effects. The clinical phase 1/2 trial to test the safety and efficacy of CTX001 therapy will begin this year. Reference materials: [1] Solution to 50-year-old mystery could lead to gene therapy for common blood disorders [2] Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding [3] CRISPR Therapeutics Submits First Clinical Trial Application for a CRISPR Gene-Edited Therapy, CTX001 in β-thalassemia Bathroom Safety & Home Health Care
Elderly assistance armrest
Assist handrails are mainly used to install in elderly families, disabled families, elderly care institutions, disabled service institutions, medical institutions, hotels and other places to help the disabled elderly, disabled people and patients maintain physical balance.
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It refers to the devices that are installed in corridors, bedrooms, bathrooms, bathrooms, staircases and balconies of the elderly care institutions and have continuity and specific functions to support the actions of the elderly. The disabled elderly should use the hand grip action to maintain their body balance or support their body. The assist handrail installed on the wall is generally divided into horizontal, vertical and inclined installation, and also divided into high, middle and low installation. It is a kind of assistive device suitable for aging environment.
Unraveling the 50-year mystery, gene therapy is expected to treat classic genetic diseases