Researchers have developed a new variant of the gene editing technique CRISPR-Cas9 that has the potential to increase precision during gene therapy in humans. The new variant reduced unintended changes in DNA compared to its wildtype, suggesting it could play a role in gene therapies that require high precision. The study by researchers from Karolinska
The report, sponsored by ARM, Intellia Therapeutics, and CRISPR Therapeutics, and endorsed by former MEP Maria Teresa Giménez Barbat, discusses the commercial, clinical, regulatory, and ethical landscape and framework for the development of therapeutic for genome editing in Europe.
Science is producing a growing number of gene and cell therapies to treat spinal muscular atrophy, leukemia and other conditions, but payers and providers don’t have the information they need to determine what they should pay for these treatments. That’s according to Steven Pearson, MD, founder and president of Boston-based Institute for Clinical and Economic
Instead of augmenting super-soldiers, DARPA wants to boost troops’ natural defenses against engineered diseases — and even undo gene-editing altogether. The Pentagon’s research agency wants to explore the possibility of editing a soldier’s genetic makeup to protect against chemical and biological attacks. Defense Advanced Research Projects Agency director Steven Walker said Monday that he believes
The controversy over Novartis’ ultra-pricey gene therapy Zolgensma has intensified after it allegedly refused to supply the one-off therapy to a sick toddler in Belgium on compassionate grounds. According to The Brussels Times, the family of the toddler named Pia, who suffers from the ultra-rare disease spinal muscular atrophy (SMA) have been running a crowdfunding campaign
Annexon Biosciences, a clinical-stage biopharmaceutical company developing a pipeline of novel therapies for patients with classical complement-mediated disorders of the body, eye and brain, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for ANX005 for the treatment of Guillain-Barré Syndrome (GBS), a rare, acute, antibody-mediated autoimmune disease impacting
Source https://www.nature.com/articles/s41467-019-12281-8#Sec14 Highly specific Cas9 nucleases derived from SpCas9 are valuable tools for genome editing, but their wide applications are hampered by a lack of knowledge governing guide RNA (gRNA) activity. Here, we perform a genome-scale screen to measure gRNA activity for two highly specific SpCas9 variants (eSpCas9(1.1) and SpCas9-HF1) and wild-type SpCas9 (WT-SpCas9) in
Wake Forest Institute for Regenerative Medicine scientists have fine-tuned their delivery system to deliver a DNA editing tool to alter DNA sequences and modify gene function. The improved “hit and run” system works faster and is more efficient. “With this new method we are able to package together in one lentiviral capsid both essential components
We often take our immune and nervous systems for granted. We assume that our immune system will protect us from diseases and when pathogens invade our body. Likewise, we assume that our nervous system will take information from the environment, relay it to our brain and then allow our brain to move muscles. But what
Monash University researchers, along with industry partner Haemokinesis, have developed the world’s first blood incubator using laser technology. This technology can slash blood incubation time to just 40 seconds, compared to the current industry gold standard of five minutes. The study shows that laser incubation can improve pre-transfusion testing for millions of patients undergoing blood
