For the first time, researchers have shown that ordinary human cells can change their original function. This may give new hope for type 1 diabetes patients.
Progress reported on understanding why Hispanic children are more likely to develop acute lymphoblastic leukemia and to die of the disease.
The group of Dr. Cerón at IDIBELL used the model organism Caenorhabditis elegans to optimize the technique, leading to the development of the method called Nested CRISPR. This cloning-free method involves the insertion of long DNA fragments in two steps.
Researchers at the University of Waterloo have developed a new way to prevent and treat Chlamydia, the most common sexually transmitted bacterial infection in the world.
Researchers have combined the largest description of ST3GAL5 (GM3 synthase) deficiency using detailed natural history data from 104 individuals of Amish ancestry born between 1986 and 2017 with a definite or probable diagnosis of ST3GAL5 deficiency. The study examined objective measures of biochemistry, auditory function, brain development, and caregiver burden. GM3 synthase is encoded by ST3GAL5, and is essential for synthesis of the most biologically relevant gangliosides in mammals.
A research team has described the first case of a patient affected by dysfunctions in a nucleoside transporter of the SLC28 gene family, which brings a set of genes which were not related to human pathologies in the scientific bibliography so far.
Experimental gene therapy cassettes for Duchenne muscular dystrophy have been modified to deliver better performance. The cassettes, which carry the therapy into muscle cells, contain newer versions of a miniaturized treatment gene. Earlier versions of the treatment cassettes did significantly enhance muscle function in previous lab studies, but did so incompletely. That's partly because the huge dystrophin gene has to be condensed to fit inside the transport virus.
Researchers at The Ottawa Hospital and the University of Ottawa have discovered a new way to treat the loss of muscle function caused by Duchenne muscular dystrophy in animal models of the disease. As reported in Cell Stem Cell, the team restored muscle stem cell function that is impaired in Duchenne muscular dystrophy, resulting in efficient regeneration of the muscle and preventing the progressive loss of muscle strength characteristic of the disease.
University of Otago bioethicists are calling for a more robust system of ethical governance in human gene-editing in the wake of the Chinese experiment aiming to produce HIV immune children.
Damaged fibres in the brain or spinal cord usually don't heal at all. Neuroscientists from Bochum have high hopes for new methods based on gene therapy.