One of the challenges in designing genetic and cellular strategies is getting the therapy to the right place. This is even more complicated when it comes to the nervous system. The brain is a complex organ that contains the most differentiated and inaccessible cells in human biology. It is an impassable safe, protected by the blood-brain barrier.
Gene therapy technology makes it possible to select diseased or mutated cells from a patient, modify them in the laboratory and reintroduce them to the body to treat different disorders. This is known as ex vivo autologous gene therapy. The difference with allogeneic cell techniques is whether the donor is oneself (autologous) or a compatible person (allogeneic), which would provide healthy cells that do not need genetic modification.
The discovery of DNA was a milestone in the history of science that led to a breakthrough in biomedical research. By associating disease and genetics, genome correction techniques were ultimately developed that are supposed to work in the same way that antibiotics and antivirals block pathogenic microorganisms: by directly attacking the causes of disease.
The most ambitious objective of any treatment is to eradicate the disease, acting on its origin to cure it instead of treating its symptoms. This is the purpose of the gene therapy against type 2 diabetes (T2D) and obesity that Fractyl Health Inc. is developing. Scientists from the Lexington, Mass.-based company have designed a strategy based on glucagon-like peptide-1 (GLP-1) to transform pancreatic cells and reverse the disease.
The human genome, the sequence that represents the DNA of our species, was built with a single individual as a model. This all-in-one standard didn’t include the gene variations that make us different or explain why some people develop certain diseases. Four simultaneous studies from the Human Pangenome Reference Consortium have published a sequence based on 47 individuals, beginning to capture the genetic diversity that defines humans.
One way to prevent the effect of a molecule is to use the cell’s own machinery to break it down. This is what the PROTAC technology does, an acronym for proteolysis targeting chimera, or BacPROTAC, when applied to bacteria. A study led by Austrian and German scientists has demonstrated the effectiveness of this technique in eliminating the tuberculosis pathogen Mycobacterium tuberculosis (Mtb). The finding opens the door to the BacPROTAC strategy as an alternative to the development of drugs against this microorganism.
Patients suffering from neuropathic pain, a chronic condition, have few treatment options and often develop tolerance to existing pain therapy that decreases its effectiveness. Now, a group of scientists from the University of Alabama at Birmingham (UAB) and the Baylor College of Medicine have described the pathophysiological mechanism of initiation, transmission and maintenance of neuropathic pain and identified a potential therapeutic target to treat it efficiently.
Cells of Saccharomyces cerevisiae, a yeast used as a model for human mitosis, age in two ways. Both genomic instability and the decline of mitochondria cause cells to degenerate and die. The choice of one type or another depends on a network of genes that can be adjusted by bioengineering.
The map of the genetic activity of the risk genes that affect the central nervous system (CNS) reveals the molecular signatures associated with the neurological pathologies in this organ. A study by researchers at McGill University in Canada and the Allen Institute for Brain Science in Washington compared 40 brain diseases with this technology and classified them into five groups whose members shared the same transcriptional pattern.
The brain is plastic throughout life, but never more so than from birth to young adulthood. It increases its volume by developing dendrites and axons that connect neurons in to each other, forming new pathways to process the information that it will store. Those connections require maintenance. And if a connection is unsuccessful, better to delete it than to keep it. This is known as synaptic pruning and occurs from childhood to the age of 20. Now, a group of scientists from the University of Cambridge and Fudan University has described a neuropsychopathological (NP) factor that explains why inappropriate pruning in adolescence is related to mental health disorders.
