Researchers at the University of Saskatchewan (USask) and University of Calgary have found unusually high rates of Spinal Bulbar Muscular Atrophy, also known as Kennedy's Disease, a rare neuromuscular disorder, among Indigenous ...
Aug 10, 2021
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Johns Hopkins Medicine researchers, in collaboration with national and international researchers, have identified a genetic mutation in a small number of children with a rare type of inflammatory bowel disease. The discovery ...
Jul 30, 2021
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An experimental drug reported in Nature Communications suggests that a "path is clearly achievable" to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent ...
Jul 19, 2021
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A "perfect storm" of genetic mutations, toxic proteins and a defect in natural cell recycling has been uncovered in University of Queensland research that could lead to treatments for neurodegenerative diseases such as Alzheimer's ...
Jul 8, 2021
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An international group of clinicians and scientists from MIT and Lund University, among others, have analyzed how individual genetic changes affect the heart muscle. The researchers have created a new computer tool that could ...
Jun 15, 2021
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Researchers have developed a new approach to gene therapy that leans on the common pain reliever acetaminophen to force a variety of genetic diseases into remission.
Jun 14, 2021
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We don't know enough about biological processes in people who are aging with HIV—a new study sheds light on the matter.
Jun 8, 2021
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A rare mutation that causes Parkinson's disease-like symptoms interrupts the flow of dopamine in the brain, suggests a study in fruit flies published today in eLife.
May 25, 2021
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Apic Bio, a gene therapy company developing treatment options for patients with rare genetic diseases and co-founded by UMass Medical School's Robert H. Brown Jr., DPhil, MD, announced that the FDA has cleared its investigational ...
Apr 22, 2021
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Researchers at Beam Therapeutics have developed a redesigned base editor that shows considerable promise in directly repairing the single-base mutation that causes sickle-cell disease (SCD). Many strategies are being pursued ...
Apr 20, 2021
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