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Figure 1: Expansion mutations in four SCA10 families. Figure 4: Correlation between the size of expanded SCA10 ATTCT repeat and the age of onset. Figure 5: Sca10 is widely expressed in brain ...
Early research shows that editing expanding trinucleotide repeats halts the lengthening process that causes neurological ...
Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and ...
3 Sobell Department of Motor Neuroscience and Movement Disorders, UCL Institute of Neurology ... to the genetic testing for HD causative mutations, that is, IT15 gene trinucleotide-repeat expansion.
The ASO for myotonic dystrophy type 1 was well tolerated and improved splicing and function in a study presented at ASGCT.
† Department of Chemistry and International Institute for Nanotechnology, Northwestern University, Evanston, Illinois 60208, United States § ‡ Translational Research Institute and § Department of ...
Department of Physics, North Carolina State University, Raleigh, North Carolina 27695-8202, United States ...
For the first time, doctors have treated a baby born with a rare, life-threatening genetic disorder with a gene-editing therapy scientists tailored to specifically repair his unique mutation.
The defending champion Florida Panthers ousted the Tampa Bay Lightning 6-3 in Game 5, wrapping up a convincing series win that bodes well for their repeat bid. The Panthers, healthy after dealing ...
Finals MVP Jonquel Jones knows the Liberty’s 2025 season will have its share of ups and downs as the quest to repeat begins Saturday in Downtown Brooklyn. AP Photo by Abbie Parr “We try to ...
A radar image of a speed offender caught in central Switzerland last month revealed that the culprit was not only a duck, but likely a "repeat offender," local authorities said. Police in the town ...
Nearly 19 million children in the United States have at least one parent with a substance use disorder, according to a new study published Monday in JAMA Pediatrics. And a significant number of ...