MeSH tree: C16.320.322
The genetic disorder Fragile X syndrome occurs when individuals don't make the Fragile X protein known as FMRP. Essential for normal brain development, FMRP helps control when and where proteins are made in the brain, supporting ...
Aug 28, 2025
0
16
Texas Children's is pleased to announce that a three-year-old girl has been successfully treated with the first-ever FDA-approved gene therapy treatment for AADC deficiency.
Aug 19, 2025
0
31
A cell therapy preserves muscle structure and function in laboratory mice with a type of disease similar to Duchenne muscular dystrophy, according to new research from the Smidt Heart Institute at Cedars-Sinai.
Aug 4, 2025
0
21
Findings from St. Jude Children's Research Hospital demonstrate that virtual memory T cells, a specialized group of immune cells, provide nonspecific immunity for infants early in life.
Jul 31, 2025
0
19
The U.S. Food and Drug Administration's (FDA) top vaccine official is stepping down after just three months in a role that upset drug companies, patient groups and some political leaders.
Jul 31, 2025
0
14
A study evaluating a pioneering lentivirus (LV)-mediated gene therapy trial for classical Fabry disease showed promising results over five years, indicating a potential breakthrough in treatment for the genetic disorder.
Jul 25, 2025
0
104
Researchers led by UC Davis Health scientist Sanchita Bhatnagar have developed a promising gene therapy that could treat Rett syndrome. The therapy works on reactivating healthy but silent genes responsible for this rare ...
Jul 23, 2025
0
157
UC Davis MIND Institute researchers Randi and Paul Hagerman are calling for increased awareness and screening for fragile X-associated conditions. In a new paper published in the New England Journal of Medicine, the husband-and-wife ...
Jul 18, 2025
0
23
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated that inhibiting GLUD1 ...
Jul 16, 2025
0
10
Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular ...
Jul 15, 2025
0
14
