A lipid nanoparticle-based gene therapy for cystic fibrosis restores biological function in a preclinical model...
This film documents a PET event discussing how to improve diagnosis, treatment and support for people affected by rare and inherited metabolic diseases...
A topical CRISPR-based genome-editing therapy has been developed to address disease-causing mutations responsible for inherited human skin disorders...
Professor Jim Al-Khalili interviews Professor George Church in this episode of The Life Scientific. From bringing back mammoths to the Human Genome Project... but does he ask probing enough questions? Davy Tennison finds out for BioNews...
A novel gene insertion therapy to treat a rare liver disorder in newborns has received regulatory designations in the USA and the UK...
Ancient viral DNA sequences in the mouse genome may offer insight into a severe muscle-wasting disease in humans…
A patient with B-cell acute lymphoblastic leukaemia has become the first adult to receive a pioneering cell therapy on the NHS...
CRISPR/Cas9 genome editing has been used in mice to investigate two key genes' roles in male fertility and sperm production...
A new genome editing approach has been shown to safely and efficiently remove the source of transthyretin amyloidosis in mice, demonstrating greater therapeutic opportunities for genome editing...
A heart attack patient has become the first person to be treated in a clinical trial of an experimental gene therapy, which aims to strengthen blood vessels after coronary bypass surgery...
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