Important findings from an animal study have prompted the exploration of a gene therapy for Duchenne muscular dystrophy in an ongoing human trial.
Results for "gene therapy"
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.
General-purpose ultrasound therapy also monitors brain activity in real time
Ultrasound therapy, which stimulates neural pathways, just became easier to evaluate, opening the door for better clinical data.
Delivering CRISPR gene-editing therapy: What’s holding us back?
Genomic editing tools can reverse disease causing mutations to provide cures — once we can conquer the remaining barriers.
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