MeSH tree: C18.452.648.595
If administered early, gene therapy has the potential to change the medical history of children born with metachromatic leukodystrophy (MLD), a rare and lethal neurodegenerative disease of genetic origin which leads to the ...
Apr 24, 2025
0
33
The STING protein, known for helping cells fight viral infections by generating inflammation, also appears to function as a quality control sensor for organelles that serve as cellular waste disposal systems, UT Southwestern ...
Apr 15, 2025
0
43
It is a detailed view of the brain like never seen before. In a significant technological leap, University of Florida researchers have created a powerful new computational and artificial intelligence tool that can generate ...
Mar 19, 2025
0
34
In a preclinical study, researchers at Children's Hospital of Philadelphia (CHOP) demonstrated a novel gene therapy with potentially increased effectiveness and safety for the treatment of metachromatic leukodystrophy (MLD), ...
Mar 12, 2025
0
25
The International Classification of Diseases (ICD) is a global standard for classifying diseases, deaths and health conditions and has been used for more than a century. It assigns diagnostic codes to categorize diseases ...
Feb 20, 2025
0
39
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase deficiency (MSD), an ultra-rare ...
Jan 29, 2025
0
51
Researchers at Washington University School of Medicine in St. Louis have described the neurodegeneration that occurs in the nervous system of the bowel in Batten disease, a rare and fatal genetic condition. In their latest ...
Jan 15, 2025
0
23
Researchers at Children's Hospital of Philadelphia (CHOP) announced encouraging results from the first ever gene therapy trial for Danon disease (DD), a rare, X-linked heart condition caused by a single gene mutation. The ...
Nov 19, 2024
0
22
RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
Gregory and Deborah Macres from California lost their son, Gregory, in 1997, when he was just 4 years old. Little Gregory had a severe form of a rare genetic disorder called Gaucher disease and died after unsuccessful experimental ...
Oct 14, 2024
0
40
