Targeted Antisense Drug Shows Promise Against ALS
Antisense approach shows promise against caused by fused in sarcoma genetic mutation, based on research from Columbia University.
Novel Strategy Can Help Sensitize Glioma Cells To Radiotherapy
Glioma brain cancer cells with mutated ATRX protein can be better targeted with the use of radiation sensitizers to help ensure cancer cell destruction during radiotherapy, according to University of Michigan researchers.
Pfizer and OPKO Suffer FDA Setback with Pediatric Growth Hormone Deficiency Therapy
Complete letter of response from FDA sets back Pfizer and OPKO Health's hopes for imminent U.S. market approval for their pediatric growth hormone deficiency treatment somatrogon.
Scientists Develop Safer Gene Editing Drug Delivery System
The particles allow for the safer delivery of gene-editing therapies than other methods that can lead to prolonged expression in transduced cells—which increases the frequency of off-target editing—and viral vector integration into the genome of transduced cells, both of which can lead to oncogenesis.
Penn and CBM Ink Potential $100M Gene Therapy Manufacturing Deal
The University of Pennsylvania’s Gene Therapy Program (GTP) has signed a collaboration agreement with contract manufacturer The Center for Breakthrough Medicines (CBM) worth an estimated $75-$100 million.
High Speed Cell Sorter Allows Genome-Scale Studies of Complex Phenotypes
Isolating individual cells with complex phenotypes is a technological challenge, now researchers present a high-speed cell sorter that uses fluorescence imaging to enable genome-scale studies of complex phenotypes.
Immunotherapy Can Induce Remission in Young Children with Peanut Allergy
Landmark NIH allergy study shows immunotherapy successfully desensitizes 71% of young children with severe peanut allergy, with 21% also achieving remission.
NIH Earmarks $170M for the Nutrition for Precision Health Program
The new program, a part of the All of US Research Program, will seek to develop algorithms to predict responses to each individual's diet. The effort will recruit 10,000 people from diverse backgrounds as it looks to create tools to provide individuals with personal dietary recommendations.
Gene Therapy for Severe Form of Epilepsy Shows Promise
Scientists from the University of Virginia School of Medicine describe in a study involving mouse models how a newly developed gene therapy can treat Dravet syndrome, a severe form of epilepsy, and potentially prolong survival for people with the condition.
Tau Interactome Map Suggests How Mutated Brain Protein Spreads
Researchers at the Buck Institute and collaborators have mapped the tau interactome using proteomics. They also uncovered new findings about how the diseased protein spreads in the brain and how it impacts mitochondria.
