A team of medical researchers affiliated with a host of institutions in the U.S. has used base editing to restore the natural production of the SMN protein in mice, effectively curing spinal muscular atrophy (SMA) in the ...
Scientists in Cambridge and Berlin have used a form of gene therapy to increase levels of the so-called "cold shock protein" in the brains of mice, protecting them against the potentially devastating impact of prion disease.
Mar 24, 2023
0
16
Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades.
Mar 22, 2023
0
1443
Living organisms have a knack for persisting in the face of challenges. For instance, when genes malfunction, organisms may be able to compensate by activating redundant genes with similar functions, called paralogs.
Feb 21, 2023
0
54
Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For patients who have facioscapulohumeral ...
Feb 17, 2023
0
873
Professor Megumu K. Saito and his colleagues have revealed that SMN, a protein involved in spinal muscular atrophy (SMA), promotes the functional maturation of mitochondria by regulating miRNA expression along with MYOD1 ...
Feb 17, 2023
0
33
A new study has observed differences in the shape, structure and mechanical properties of nuclei in trained and untrained individuals, potentially helping to explain the anti-aging effects of exercise.
Jan 18, 2023
0
3
Newborn screening (NBS) for spinal muscular atrophy (SMA), when combined with early treatment, results in better movement ability in affected children, including the ability to walk, when compared to children who are diagnosed ...
Jan 18, 2023
0
15
Muscular dystrophy is a debilitating disease that causes progressive weakening and loss of muscles. Fukuyama congenital muscular dystrophy (FCMD), the second most common form of childhood muscular dystrophy in Japan, is a ...
Dec 12, 2022
0
29
A number of new neurologic medications for diseases like multiple sclerosis (MS), Parkinson's disease and migraine have received U.S. Food and Drug Administration (FDA) approval over the past decade. However, with most having ...
Nov 30, 2022
0
2
