Gene therapy restores hearing in children with hereditary deafness; ongoing trials show promise for widespread application.
Results for "gene therapy"
CRISPR gene therapy for sickle cell disease and β-thalassemia gets UK approval
In a world first, UK authorities grant regulatory approval for a CRISPR gene therapy targeting sickle cell disease and β-thalassemia.
Making progress with a gene therapy for muscular dystrophy
Important findings from an animal study have prompted the exploration of a gene therapy for Duchenne muscular dystrophy in an ongoing human trial.
The first CRISPR gene therapy to cure sickle-cell disease
In 2019, CRISPR gene-editing therapy was used for the first time to treat sickle cell disease.
Gene Therapy: Virus-like Delivery System Takes the Heat out of Melanoma
Targeted viral-based gene therapies come with their own risks. A new gene delivery system mimics viral therapies to infect cancer cells with vesicular stomatitis virus matrix protein, with a similar transfection efficiency as the virus itself.
Non-Toxic Targeted Gene Therapy in Obese Rats
Obesity is a growing epidemic around the world.
Gene therapy: Non-Viral Nanoparticles as Delivery Systems
Researchers from Case Western Reserve University present an efficient non-viral gene delivery system for safe gene therapy to treat genetic eye disorders.
Click Chemistry for Gene Therapy
A reducibly-degradable hyperbranched polymer PEI-SS-HP composed of LMW PEI via a disulfide-containing linkage is demonstrated as a promising gene vector.
Unlocking personalized leukemia therapy using epigenetic biomarkers
A specific epigenetic signature in patients who respond to chemotherapy offers potential to improve treatment.
Regenerating nerves with an mRNA therapy
Stimulating neuron growth using a new mRNA therapy, researchers hope to treat chronic pain caused by disease, injury, or chemotherapy.
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