Implementation of new pricing and business structures and improved licensing and manufacturing processes could reduce the per-patient cost of gene therapy tenfold.
Category: genetics – Page 44
Summary: Researchers have developed the first 3D map of gene regulation in the human brain, offering insights into how early brain development influences lifelong mental health. This map, focusing on regions tied to memory and emotional regulation, reveals how chromatin structure controls gene activity, especially during key developmental stages.
These findings may help identify when and where genetic variants linked to autism and schizophrenia disrupt normal development. By understanding these early influences, scientists hope to improve neurodevelopmental disorder research and stem-cell models, potentially paving the way for earlier intervention strategies.
Scientists have used gene-editing techniques to boost the repair of nerve cells damaged in multiple sclerosis, a study shows. The innovative method, which was tested in mice, supports the development of cells that can repair the protective myelin coating around nerves, restoring their ability to conduct messages to the brain.
The findings, now published in Nature Communications, offer a potential route for future treatments to stop disability progression, experts say.
Our bodies have the ability to repair myelin, but in multiple sclerosis (MS), and as we age, this becomes less effective. There are currently no treatments to boost this process.
Mamma bears press pause on their early pregnancies, so that their cubs are born closer to a food filled spring. Researchers led by a team in Germany have now found this same pregnancy pause button exists in human cells too.
“Although we have lost the ability to naturally enter dormancy, these experiments suggest that we have nevertheless retained this inner ability and could eventually unleash it,” says molecular geneticist Nicolas Rivron from the Austrian Academy of Sciences (IMBA).
“Triggering a dormant state during an IVF procedure could provide a larger time window to assess embryo health and to synchronize it with the mother for better implantation inside the uterus.”
A research team led by the University of California, Irvine has engineered an efficient new enzyme that can produce a synthetic genetic material called threose nucleic acid. The ability to synthesize artificial chains of TNA, which is inherently more stable than DNA, advances the discovery of potentially more powerful, precise therapeutic options to treat cancer and autoimmune, metabolic and infectious diseases.
“It’s exciting to see the high efficiency and versatility of eePASSIGE, which could enable a new category of genomic medicines,” added Gao. “We also hope that it will be a tool that scientists from across the research community can use to study basic biological questions.”
Prime improvements
Many scientists have used prime editing to efficiently install changes to DNA that are up to dozens of base pairs in length, sufficient to correct the vast majority of known pathogenic mutations. But introducing entire healthy genes, often thousands of base pairs long, in their native location in the genome has been a long-standing goal of the gene-editing field. Not only could this potentially treat many patients regardless of which mutation they have in a disease-causing gene, but it would also preserve the surrounding DNA sequences, which would increase the likelihood that the newly installed gene is properly regulated, rather than expressed too much, too little, or at the wrong time.
Researchers have identified a plausible geological setting in which nucleic acids, the fundamental building blocks of genetic material, could have replicated on their own, potentially giving rise to life on Earth.
The study, published as a reviewed preprint in the journal eLife, shows how a simple interaction between gas flow and water in a narrow rock channel could create the physical conditions necessary for nucleic acid replication.
The work offers insights that may interest scientists exploring the origins of life as well as applications in nucleic acid research and diagnostics.
Summary: Scientists have identified how genetic variants influence the risk of neurological and psychiatric disorders, including schizophrenia and autism. Using live neural cells and DNA sequencing, researchers discovered thousands of “non-coding” genetic variants with context-dependent functions, activated during brain development.
These variants act like switches, turning genes on or off depending on cellular pathways. This research offers new insights into the biological mechanisms behind psychiatric disorders and could lead to personalized treatments based on genetic profiles.
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Summary: Neural stem cells, which create new neurons in the brain, become less active with age due to elevated glucose levels. Researchers found that by knocking out the glucose transporter gene GLUT4 in older mice, they could significantly increase the production of new neurons.
This discovery opens up potential pathways for both genetic and behavioral interventions to stimulate brain repair, including the possibility of a low-carbohydrate diet. The findings could help treat neurodegenerative diseases and aid in brain recovery after injury.