RNA editing is the next frontier in gene therapy—here's what you need to know
The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA ...
Second Gene Therapy Shows Promise for Syndrome Involving Blindness, Deafness
Harvard Medical School researchers have taken another decisive step in their efforts to develop a gene therapy for people ...
sicklecellanemianews7 天
Cyclosporin H boosts uptake of gene therapy in SCD newborn stem cells
Cyclosporin H boosts a new lentivirus-delivered gene therapy with enhanced anti-sickling properties in blood stem cells from ...
Neuroscience News10 天
Dual-Gene Therapy Shows Promise for Hearing and Vision Loss
Researchers have developed a dual-gene therapy approach to treat Usher syndrome type 1F, a rare condition causing deafness and progressive blindness.
Discovery enables effective use of gene therapy for muscular dystrophies and other large ...
Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
News Medical6 天
Assessment of gene therapy viral vectors in RPE cells
This research evaluates the effects of various gene therapy viral vectors on RPE cells to determine efficacy for therapeutic ...
Medical Xpress on MSN8 天
CRISPR-based therapy shown to be safe, effective for people with transthyretin amyloidosis
A new type of therapy that 'edits' a gene in patients with a rare heart condition has been shown to be safe and effective, according to research from UCL and the Royal Free Hospital.