Diseases, Conditions, Syndromes
Two approaches to treat Lysosomal Storage Diseases
Enzyme therapy proves effective in treating LSDs, whilst gene therapy is an upcoming contender.
Apr 15, 2014
0
0
Page 5 - News tagged with gene replacement
Genetics
Researchers achieve gene therapy milestone for potential Cockayne syndrome treatment
Researchers working with UMass Chan Medical School's Translational Institute for Molecular Therapeutics announced progress in developing a vector to deliver gene replacement therapy in mice models with Cockayne syndrome, ...
Jan 30, 2023
0
5
Immunology
Gene therapy restores immune function in children with rare immunodeficiency
An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
0
6
Cardiology
Correction of cardiovascular symptoms of MPS I in animal model
REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, ...
Sep 30, 2014
0
0
Genetics
Will short-term and long-term treatments for single-gene diseases survive?
Two weeks and several political disasters ago, the House of Representatives passed the American Health Care Act of 2017, and soon lists of "pre-existing conditions" festooned news feeds. We all ticked off a few. But the lists, ...
May 22, 2017
0
0
Diseases, Conditions, Syndromes
Fight back against MRSA to be started with a sniff
An innovative anti-bacterial spray that will kill MRSA is being developed by Norwich Research Park scientists thanks to funding from the University of East Anglia.
Mar 21, 2013
0
0
