A more efficient approach to gene therapy that could lower costs and improve patient outcomes has recently been developed by a team from Scripps Research. This work, published on October 17 in the journal Blood, offers a potential alternative to the standard process of delivering gene therapy, which is expensive, time-consuming, and requires many steps to administer healthy
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease and Lou Gehrig’s disease, is a rare condition characterized by the degeneration of nerve cells that control voluntary muscles. The disease leads to gradually worsening symptoms that include muscle weakness, twitching, and stiffness. As more of these nerve cells, or motor neurons, are lost, muscles decrease in size
Further action needed to facilitate development of safe, effective treatments Source The Pew Over the last two decades, cell therapies (which involve the transplantation of whole cells into a patient), gene therapies (which use genetic material to manipulate a patient’s cells), and other medical treatments intended to repair or replace damaged, diseased, or dysfunctional cells,
Cancer: important reason for poor response to checkpoint inhibitors identified. The aim of immunotherapies is to enable the immune system once again to fight cancer on its own. Drugs known as checkpoint inhibitors are already in clinical use for this purpose. However, they are only effective in about one third of patients. Based on analysis
Scientific research follows processes to validate, refute prior evidence Source The Badger Herald A panel of science writers and experts discussed the importance of understanding uncertainty in the scientific process at the Discovery Building Wednesday night. The University of Wisconsin’s 2019 Science Writer in Residence Christie Aschwanden’s scientific journalism has appeared in FiveThirtyEight, the Washington Post,
Unique Rice platform helps bioscientists learn how ectoderm cells begin to differentiate Source Rice University During embryonic development, the entire nervous system, the skin and the sensory organs emerge from a single sheet of cells known as the ectoderm. While there have been extensive studies of how this sheet forms all these derivatives, it hasn’t been
Source NIH Director’s Blog Exercise can work wonders for your health, including strengthening muscles and bones, and boosting metabolism, mood, and memory skills. Now comes word that staying active may also help to lower your odds of developing cancer. After reviewing the scientific evidence, a panel of experts recently concluded that physical activity is associated with reduced
NIH Director Francis Collins describes evolution of existing collaborations and how the two agencies might facilitate development of gene therapy, such as by having a common template for regulatory review. Source Pink Sheet The National Institutes of Health is looking to build on its partnerships with the US Food and Drug Administration to tackle new
The U.S. Food and Drug Administration today approved Reyvow (lasmiditan) tablets for the acute (active but short-term) treatment of migraine with or without aura (a sensory phenomenon or visual disturbance) in adults. Reyvow is not indicated for the preventive treatment of migraine. “Reyvow is a new option for the acute treatment of migraine, a painful
Discoveries Could One Day Lead to New Drugs to Prolong Healthy Human Lifespan The discovery in the 1990s that a mutation in a single gene of an experimental worm could double its lifespan set off a stampede of research on the molecular biology of aging and triggered hopes that drug therapies or other interventions could
