The editing in human cells and in mice of the survival motor neuron 1 gene (SMN1) restored the levels of SMN protein that the mutation of the SMN2 gene produces in spinal muscular atrophy (SMA). Scientists from the Broad Institute in Boston and The Ohio State University reversed the mutation using the base editing technique. “This base editing approach to treating SMA should be applicable to all SMA patients, regardless of the specific mutation that caused their SMN1 loss,” the lead author David Liu, a professor and director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute of Harvard and MIT, told BioWorld.
Current prophylactic and therapeutic approaches for SARS-CoV-2 are effective, but the need for new approaches with broad activity makes virus-host interactions an essential piece to look at.
Two molecules that affected the cell cycle only of acute myeloid leukemia (AML) cells could be used as a clinical strategy against this pathology. Scientists at Memorial Sloan Kettering Cancer Center and Harvard University have discovered that DEG-35 and DEG-77 arrested the cell cycle and promoted cell differentiation and apoptosis in these cells.
Cholecystokinin (CCK) is a peptide hormone found predominantly in the gastrointestinal tract and throughout the central nervous system (CNS), and which has been also shown to stimulate the secretion of calcitonin, insulin and glucagon and to act as a natriuretic kidney peptide. Researchers from Harvard University and affiliated organizations aimed to assess the function of CCK in obesity-induced airway hyperresponsiveness (AHR) and asthma.
Harvard University’s Wyss Institute for Biologically Inspired Engineering licensed its Erapid electrochemical sensing platform to IQ Group Global to integrate with the Australian consortium’s transistor technology in a SARS-CoV-2 test. The combined solution could greatly simplify serological testing for the virus and help monitor immunity in individuals and populations over time.
I2O Therapeutics Inc., of Allston, Mass., raised $4 million in seed funding to further its work on oral formulations of therapies typically limited to injections, such as biologics, large molecules and peptide-based pharmaceuticals.
A team at the Broad Institute of Harvard and MIT has developed a genome editing method that could, in principle, correct 90% of the roughly 75,000 currently known genomic changes that are associated with genetic diseases.
Synthetic biology is seeing rapid advances, but the medical applications have thus far remained largely elusive. But now researchers from the Wyss Institute for Biologically Inspired Engineering at Harvard University and Harvard Medical School (HMS) have developed a tool that can track specific populations of bacteria in the gut of living organisms and document population changes over time.
David Southwell, a seasoned pharma exec and CEO of newly funded Tscan Therapeutics Inc., called from a small, unadorned conference room on the Harvard campus to talk about his new company. He's used to being on the clinical development side, where the accommodations are a bit more posh, but nonetheless he's pleased to be investigating T-cell therapy for cancer patients.
