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Recently, in a research report published in the international journal Proceedings of the National Academy of Sciences, scientists from the University of Alabama at Birmingham and other institutions have clarified the cause and molecular mechanism of the serious birth defect called CHARGE syndrome (CHARGE joint deformity, nostril atresia deformity) through research. In the article, the researchers successfully inactivated the CHD7 gene in mouse embryonic neural crest cells, and then tracked how the inactivation of the CHD7 gene in the developing cardiac neural crest cells induced serious defects in the right ventricular outflow tract and large arteries of the heart, and lead to perinatal death of the fetus. Heart defects and other birth defects in embryos are similar to human CHARGE syndrome defects. Currently, known CHD7 mutations can induce approximately 70% of human CHARGE syndrome.
In a new study, researchers from St. Jude Children's Research Hospital in the United States visually observed the structure of previously unknown ABL kinase, thereby providing new insights for the design of targeted therapies for adult and childhood cancer patients. This research will advance the understanding of drug resistance produced by cancer-targeted drugs. The relevant research results were published online in the Journal of Science.
Recently, in a research report published in the international journal Nature Genetics, scientists from Cambridge University and other institutions discovered for the first time that a four-stranded DNA structure (G-quadruplexes, G-quadruplexes) may play a key role in the occurrence of specific types of breast cancer. The results of related studies may provide potential new targets for the development of personalized breast cancer therapies.
Hematopoiesis is affected by biological stresses, such as infections, inflammation, and specific drugs. Recently, in a research report published in the international journal of Blood, scientists from Tokyo Medical and Dental University identified a new type of cell surface marker through research, which can help accurately analyze the response of the hematopoietic process to biological stress.
In the past 18 months, 11 gene editing research and development projects have entered the clinical development stage in the United States or the European Union, 6 of which are based on the CRISPR-Cas gene editing system. Recently, a review published in Nature Reviews Drug Discovery conducted an in-depth inventory of the gene editing R&D pipeline.
Recently, a research report published in the journal of Nature Metabolism, scientists from the University of Cincinnati developed a new method to target the molecular processes that activate specific protein complexes. Related research may be expected to help develop new therapies for treating tumor diseases.
In a new study, researchers from the Francis-Crick Institute in the United Kingdom identified key genes for the survival of Toxoplasma gondii in mice by using a CRISPR-based gene screening method. Related research results were recently published in the journal of Nature Communications. This study provides a flexible new approach to extending the use of CRISPR screening, which also allows for the simultaneous testing of hundreds of genes in Toxoplasma in mice, thereby greatly reducing the number of research animals used.
A team of researchers from Canada, the United States, and Sweden found that editing a gene involved in the production of proteins that promote muscle strength can alleviate symptoms in a mouse model of muscular dystrophy. In their paper published in the journal of Nature, the team described their experiments with mice and the new knowledge they learned.
Recently, researchers from the University of Cambridge's Welkom Foundation's Sanger Institute and Open Targets have used the CRISPR/Cas9 technology to destroy more than 300 cancer types from 30 cancer types. Each gene in a cancer model finds thousands of key genes necessary for cancer to survive. The researchers then developed a new system to prioritize the 600 most promising drug targets for developing treatments. These results accelerate the development of targeted therapies and bring scientists closer to building a Cancer Dependency Map (a detailed manual on precise cancer treatment rules) designed to help more patients receive effective treatment. The relevant research results were published online in the journal of Nature.
CRISPR gene editing technology is a revolutionary approach to treating hereditary diseases. However, this tool has not been used to effectively treat long-term chronic diseases. A research team led by Dr. Dongsheng Duan of the University of Missouri School of Medicine has identified and overcomes the barriers to editing CRISPR genes, which may provide the basis for continuous treatment using this technology.
