A new Tel Aviv University study finds that gene deletion or deficiency in neurons is responsible for the abnormal hypersocial behavior associated with Williams syndrome (WS), a rare disorder affecting 1 in 10,000 people around the world.
A subset of the stem cells in hair follicles have the potential to regenerate the coating that insulates neurons in mice, reports Thomas Hornyak of the VA Maryland Health Care System and the University of Maryland School of Medicine and colleagues, in a new study published 24th April in PLOS Genetics. The study offers a new direction for finding therapeutic options for certain neurodegenerative diseases.
University of California San Diego School of Medicine researchers found that treating patients with personalized precision medicine that combined therapies to target multiple alterations improved outcomes in patients with therapy-resistant cancers.
Since the CRISPR genome editing technology was invented in 2012, it has shown great promise to treat a number of intractable diseases. However, scientists have struggled to identify potential off-target effects in therapeutically relevant cell types, which remains the main barrier to moving therapies to the clinic. Now, a group of scientists at the Gladstone Institutes and the Innovative Genomics Institute (IGI), with collaborators at AstraZeneca, have developed a reliable method to do just that.
Gene therapy offers the promise of a cure for beta-thalassemia and a new study has shown that it is associated with fewer complications and hospital admissions over 2 years than treatment by allogeneic hematopoietic stem cell transplantation (HSCT).
What if doctors had a remote control that they could use to steer a patient's own cells to a wound to speed up the healing process? Although such a device is still far from reality, researchers reporting in the ACS journal Nano Letters have taken an important first step: They used near-infrared light and an injected DNA nanodevice to guide stem cells to an injury, which helped muscle tissue regrow in mice.
A small clinical trial has shown that gene therapy can safely correct the immune systems of infants newly diagnosed with a rare, life-threatening inherited disorder in which infection-fighting immune cells don't develop or function normally. Eight infants with the disorder, called X-linked severe combined immunodeficiency (X-SCID), received experimental gene therapy co-developed by NIH scientists. They experienced substantial improvements in immune system function and normal growth up to two years after treatment.
Gene therapy developed at St. Jude Children's Research Hospital has cured infants born with X-linked severe combined immunodeficiency (SCID-X1). The children are producing functional immune cells, including T cells, B cells and natural killer (NK) cells, for the first time. The gene therapy involved collecting patients' bone marrow, then using a virus as a vector to insert a correct copy of the IL2RG gene into the genome (DNA) of patients' blood stem cells.
A new study from BC Children's Hospital, the University of British Columbia (UBC) and an international team of researchers published in the New England Journal of Medicine is the first to identify a rarely-seen type of DNA mutation as the cause of an inherited metabolic disorder. Inherited metabolic disorders -- where the body can't break down specific nutrients from food leading to a range of serious health problems -- are often caused by a defective gene.
11,000 people are predicted to die from acute myeloid leukemia (AML) in 2019. Recently, drug developers designed a new treatment to target the cancer's mutated genes. But, these drugs don't always work. Now, in a new study, researchers investigate both sides of the drug-body relationship to better understand why certain AML treatments--and other cancer treatments--may not work as expected.