Gene editing to correct genetic faults and removing the risk of hereditary diseases may soon become reality. Researchers have come up with a new gene editing technology that could help patients with sickle cell anaemia and up 89 percent of other genetic defects. This new technique is called the “prime editing” and is being developed
Source NIH Initial investment aims to advance accessible and scalable candidate interventions into clinical trials within 10 years. The National Institutes of Health plans to invest at least $100 million over the next four years toward an audacious goal: develop affordable, gene-based cures for sickle cell disease (SCD) and HIV. The Bill & Melinda Gates
Treatment approved for approximately 90% of patients with cystic fibrosis, many of whom had no approved therapeutic options Source FDA The U.S. Food and Drug Administration today approved Trikafta (elexacaftor/ivacaftor/tezacaftor), the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. Trikafta is approved for patients 12 years and older
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,
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
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
Researchers have discovered that a key cell type involved in liver injury and cancer consists of two cellular families with different origins and functions. The research by academics from the Universities of Edinburgh and Bristol and funded by the Wellcome Trust and Medical Research Council, is published today [Tuesday 15 October] in Nature Communications. The distinguishing
A California “human biohacking” bill calls for warnings on do-it-yourself genetic-engineering kits. Source Technology Review It’s going to be illegal in California to sell “gene-therapy kits” unless they carry a warning that says not to use them on yourself. Just one wrinkle: we’re not sure any such kit exists. Not yet, anyway. The consumer protection
