A study published in the journal Molecular Therapy by Christian Mueller, PhD, shows that using nuclease-free gene editing to correct cells with the mutation that causes alpha-1 antitrypsin deficiency leads to repopulation of a diseased liver with healthy cells. It has the potential to prevent liver and lung damage from forming in very young alpha-1 patients.
Virotherapy capable of destroying tumor cells and activating anti-tumor immune reactions, and the use of engineered hematopoietic stem cells (HSCs) to deliver replacement genes that have the potential to cure blood diseases are among the key areas of gene therapy being advanced by German researchers.
Three of the most deadly cancers -- glioblastoma, sarcoma and ovarian -- get critical funding for clinical trials from Alliance for Cancer Gene Therapy.
During the conference, researchers will discuss how Droplet Digital PCR helps them screen stem cells for harmful mutations as well as how Droplet Digital PCR provides early detection of transplant rejection and delivers absolute quantification of gene expression to investigate cancer.
In a new study, researchers from the University of Copenhagen have identified the two genes whose mutation cause a serious cancer form found in the liver. The result sets concrete goals for future treatment of the otherwise incurable disease.
A new study demonstrates success in using a shortened form of the CEP290 gene for gene therapy in a mouse model of Leber congenital amaurosis type 10 (LCA10), a retinal degenerative disorder that causes childhood blindness.
Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes Duchenne muscular dystrophy, a severe muscle-wasting disease.
The first gene therapy for cancer will transform approaches to cancer treatments, but it poses ethical challenges for policy-makers, as described by three Hastings Center scholars in the October issue of Health Affairs.
Most causes of untreatable blindness occur due to loss of the millions of light sensitive photoreceptor cells that line the retina, similar to the pixels in a digital camera.
New research published in the Journal of Physiology today suggests that enhancing breathing via the brain may limit deficiencies in respiratory capacity in Duchenne muscular dystrophy patients.