MeSH tree: C18.452.132.100.435
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
Scientists have conducted the largest and most diverse study to date on how recessive genetic changes contribute to developmental disorders. They found that most undiagnosed cases that are due to recessive causes are linked ...
Sep 23, 2024
0
47
Researchers from Children's Hospital of Philadelphia (CHOP) have tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase deficiency (MSD), a disorder that affects the brain, lungs, ...
Sep 9, 2024
0
45
Cancer cells seldom start off stealthy. Quite to the contrary, they announce their presence to the immune system by planting chemical red flags right on their membranes. Once alerted, the body's defenses can swoop in, destroying ...
Sep 9, 2024
0
117
About 37 million people in the United States have chronic kidney disease and studies show that genetics may explain between 10% and 20% of cases in adults (and as many as 70% of cases in children).
Jul 29, 2024
0
31
University of Queensland researchers are suggesting that a new diagnosis should be added to the official world diseases list to better identify those with gaming disorders.
Jun 14, 2024
0
36
Metachromatic leukodystrophy is a rare genetic disorder that mainly affects young children and results in severe neurological symptoms accompanied by a loss of motor and intellectual capacities. At Paris Brain Institute, ...
Apr 22, 2024
0
43
Chronic kidney disease (CKD) is extremely prevalent among adults, affecting over 800 million individuals worldwide. Many of these patients eventually require therapy to supplement or replace kidney functions, such as dialysis ...
Apr 3, 2024
0
56
The U.S. Food and Drug Administration has approved Orchard Therapeutics' Lenmeldy (atidarsagene autotemcel) as the first gene therapy for the treatment of children with metachromatic leukodystrophy (MLD).
Mar 25, 2024
0
26
