Topic List : Genetics
Child’s life saved by experimental drug
Four-year-old Zoe Harting is doing well after participating in a phase-2 clinical trial of the first drug for a deadly genetic disease, spinal muscular atrophy type 1.
Nusse wins $3 million Breakthrough Prize
The developmental biologist was honored for helping to decode how Wnt signaling proteins affect embryonic development, cancer and the activity of tissue-specific adult stem cells that repair damage after injury or disease.
Tracking cancer evolution in the blood
Monitoring cancer DNA in blood can predict recurrence and prognosis and drive treatment decisions. A Stanford study of 92 lymphoma patients suggests similar techniques may work for other tumors.
Paving the way for gene therapy
Using the CRISPR gene-editing technique in stem cells, Stanford researchers repaired the gene that causes sickle cell disease, and the mended stem cells were successfully transplanted into mice.
Gene therapy for blistering skin disease
A trial in which genetically altered skin was grafted onto patients’ chronic wounds marks the first time that skin-based gene therapy has been demonstrated to be safe and effective in humans.
Telomere destruction activates DNA damage response
A new study shows that telomeres shorten without cell division in a mouse model of Duchenne muscular dystrophy. Subsequent DNA damage responses and mitochondrial dysfunction are likely cause of heart failure.
Dietary approach to depleting stem cells
A new study shows that a diet deficient in valine effectively depleted the blood stem cells in mice and made it possible to perform a blood stem cell transplantation on them.
A baby with a mosaic heart
Researchers have solved the mystery of an infant with severe long QT syndrome, found to be caused by a lethal genetic defect in only 8 percent of her cells.
RNA molecule aids DNA damage response
Stanford researchers have found that a tumor suppressor known as p53 is stabilized by a regulatory RNA molecule called DINO. The interaction helps a cell respond to DNA damage and may play a role in cancer development and premature aging.
Cell, gene medicine lab opens
Making cell- or virus-based therapies for use in humans requires a rigid set of quality-control standards outlined by the Food and Drug Administration. A new Stanford facility will allow promising new therapies to be tested in the clinic.