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Spastic hereditary paraplegia type 50 (SPG50) is a typical ultra-rare disease (incidence is less than 1 in 50,000). The disease is caused by biallelic pathogenic variants in the AP4M1 gene, which encodes a subunit of the AP-4 protein complex. It is a progressive neurodegenerative disease. Patients usually begin to show symptoms in infancy, resulting in developmental delay, speech disorders, epileptic seizures, and gradual paralysis of the limbs. In their teens, most patients are wheelchair-dependent and show severe cognitive impairment, usually dying in adulthood.
In a new study, researchers from the Institute of Biochemistry at the University of Kiel have found a way to inhibit the function of the cancer-causing protein MYC. This could be used to develop new drugs. The relevant research results were recently published in the journal Gut, with the title of the paper "Targeting MYC effector functions in pancreatic cancer by inhibiting the ATPase RUVBL1/2".
Pathological α-synuclein (α-syn) can spread between cells in part by binding to lymphocyte activation gene 3 (Lag3). Recently, in a research report published in the international journal Nature Communications, scientists from Johns Hopkins University School of Medicine and other institutions have identified a potential new biological target involving Aplp1, a cell surface protein that can drive the spread of α-synuclein that causes Parkinson's disease.
Host cells are the most commonly used key starting materials for the production of recombinant proteins, antibody drugs or vaccine drugs. The types of host cells used for the production of biological products mainly include bacterial cells (such as Escherichia coli), yeast cells (such as Saccharomyces cerevisiae) and mammalian cells (including Chinese hamster ovary (CHO) cells and human embryonic kidney (HEK) cells), etc.
In vivo genome correction is expected to produce lasting disease cures. However, effective stem cell editing remains challenging. In a new study, researchers from the University of Texas Southwestern Medical Center, Case Western Reserve University School of Medicine and ReCode Therapeutics have developed a method to deliver gene editing tools into the lungs to repair CFTR gene defects associated with cystic fibrosis. In the process, they overcame problems that have hindered previous therapies and believe that their method will soon be able to be used to treat human patients. The relevant research results were recently published in the journal Science, with the title "In vivo editing of lung stem cells for durable gene correction in mice".
Recently, the latest research results published by the research team of Washington University in St. Louis (WUSTL) in the journal Science Immunology showed that after knockout the important cancer-promoting receptor TREM2 on macrophages, the intestinal microenvironment will also change to an inflammatory state. Among them, Ruminococcus gnavus will proliferate significantly, promoting the proliferation and activation of CD4+T cells in the intestine, and migrating to the tumor site in large numbers, making PD-1 inhibitor treatment more effective.
The MYC gene is a regulatory and proto-oncogene that is overexpressed in most prostate cancers (PCa). A large number of research results have shown that abnormal expression of microRNAs is involved in the occurrence and progression of human prostate cancer. Recently, in a research report entitled "Inhibitory effect of miR-377 on the proliferative and invasive behaviors of prostate cancer cells through the modulation of MYC mRNA via its interaction with BCL-2/Bax, PTEN, and CDK4" published in the international journal Genes & Cancer, scientists from the Pasteur Institute of Iran and other institutions revealed the inhibitory effect of miR-377 on prostate cancer cells through research.
The ecosystem surrounding a tumor, also known as the tumor microenvironment, includes immune cells, tissues, blood vessels, and other cells that interact with each other and with tumor cells. Over time, tumors shape this ecosystem in their own interests, monopolizing all nutrients and protecting them from the host's immune attack. In order to better understand the role of the ecosystem in cancer risk, occurrence, and treatment.
Recently, researchers from Yale University and other institutions published a research paper titled "In vivo AAV-SB-CRISPR screens of tumor-infiltrating primary NK cells identify genetic checkpoints of CAR-NK therapy" in the journal Nature Biotechnology. After years of technical research, the research team used the AAV-transposon system to integrate the CRISPR library into the genome, overcoming the obstacles of low efficiency of primary NK cell transduction and gene editing. Through four parallel CRISPR screening experiments and single-cell sequencing data, the study discovered a new target, CALHM2, which provides a new therapeutic target for NK cell tumor immunity.
In a new study, researchers from the University of California, Los Angeles discovered that a protein called MYCT1 plays a key role in regulating the self-renewal of human hematopoietic stem cells (HSCs) by helping them sense and interpret signals from their environment. This discovery brings scientists one step closer to developing a method to expand hematopoietic stem cells in laboratory dishes. This will make life-saving hematopoietic stem cell transplants more accessible and improve the safety of hematopoietic stem cell-based therapies, such as gene therapy. The relevant research results were recently published in the journal Nature, with the title of the paper "MYCT1 controls environmental sensing in human haematopoietic stem cells".
