ChemDiv shares reviews on Nature and Science Journal articles that we found the most exciting this month. Here is the first part of the July reviews. Today we observe Genetic Engineering, Drug research, Neurophysiology, and Immunology.
Kaiser, J. Gene editor injected into the body treats disease. Science 373, 16 (2021)
CRISPR is a family of DNA sequences found in the genomes of such organisms as bacteria and archaea. CRISPR sequences form the basis of a technology known as CRISPR-Cas9 that can be used to edit genes within organisms.CRISPR excels at fixing disease mutations in lab-grown cells. But using CRISPR to treat most people with genetic disorders requires clearing enormous hurdles. In a new study, in a medical first, researchers have injected a CRISPR drug into the blood of people born with a disease that causes fatal nerve and heart disease. In three of them, it nearly shut off the production of a toxic protein by their livers. The preliminary data are generating hope about CRISPR as a one-time, lifelong treatment of genetic disorders.
Shahrudin, S., Ding, S. Boosting stem cell immunity to viruses. Science 373, 160-161 (2021)
Stem cells are cells from which all other cells in the body with specialized functions are generated. Mammalian stem cells exhibit deficiencies in innate immunity, so they rely on RNA interference for antiviral protection. Mammalian antiviral RNAi is initiated by Dicer, which processes replicative intermediates into small interfering RNAs that cleave viral RNA. However, it remains unclear how stem cells activate antiviral RNA interference. In their new study, scientists show that mouse and human stem cells have a specialized Dicer isoform for virus-derived small interfering RNAs production to initiate potent antiviral RNA interference. This further indicates that small interfering RNAs therapy may be viable for RNA viruses such as SARS-CoV-2.
Servick, K. Brain signals ‘speak’ for person with paralysis. Science 373, 263 (2021)
On pages of Science, researchers report about a system that reads electrical signals from speech production areas of the brain. This was the first demonstration of the potential of this system for the disabled. The patient has lost the ability to control the muscles involved in speech after a stroke. Researchers used a computational model that interpreted brain activity in a brain region involved in producing speech and helped the man to produce sentences through a system. This system is also known as a deep-learning algorithm or a type of machine learning that works based on the structure and function of the human brain.
McAlpine, C.S., Park, J., Griciuc, A. et al. Astrocytic interleukin-3 programs microglia and limits Alzheimer’s disease. Nature 595, 701–706 (2021)
Glia, also called glial cells or neuroglia, are non-neuronal cells in the nervous system that do not produce electrical impulses. Communication between glial cells is essential for neuronal and brain health. Interleukin-3 is a multifunctional cytokine that has been implicated in inflammatory and autoimmune diseases. In their new study scientists show, in humans and mice, that interleukin-3 programs microglia to ameliorate the pathology of Alzheimer’s disease. IL-3 elicits the programming of microglia to endow them with an acute immune response program, and these changes restrict Alzheimer’s disease pathology. Such findings identify IL-3 as a node for therapeutic intervention in Alzheimer’s disease.
Monteys, A.M., Hundley, A.A., Ranum, P.T. et al. Regulated control of gene therapies by drug-induced splicing. Nature (2021)
Gene therapy is a technique that modifies a person’s genes to treat or cure disease. Although approaches for gene therapies have undergone substantial advancement over recent years, it has based on complex constructs that cannot be completely controlled. In the article, scientists report a universal switch element that enables precise control of gene replacement or gene-editing after exposure to a special small molecule, which can reach both peripheral tissues and the brain. Such molecules are called small-molecule inducers, and now they are currently in human use. A new switch system can be adapted for cell-biology applications, animal studies, and the application of gene therapies in humans.