Revolutions in gene therapy are rapidly changing the landscape of modern medicine. Revolutions in gene therapy are rapidly changing the landscape of modern medicine, forcing society and science alike ...
In a world-first trial, scientists used a one-off CRISPR gene edit to switch off a liver “fat brake” gene, slashing stubborn LDL cholesterol and triglycerides in patients whose levels refused to budge ...
Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...
NEW ORLEANS — New data from a first-in-human trial of a CRISPR-based gene therapy for cholesterol disorders are giving lipids experts reason for optimism about the approach for treating cardiovascular ...
In a step toward the wider use of gene editing, a treatment that uses Crispr successfully slashed high cholesterol levels in a small number of people. In a trial conducted by Swiss biotech company ...
A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated children, offering a lasting, donor-free solution to ADA-SCID, known as the ...
A blood stem cell gene therapy co-developed by UCLA’s Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications ...
Long-term efficacy has been shown for γ-retroviral–mediated gene therapy for ADA-SCID, but insertional oncogenesis remains a risk, and T-cell leukemia was reported in one patient. 12 In contrast to ...
Scientists may have discovered the first therapy for Huntington’s disease, a brain disorder that until now has had no effective treatments. Researchers at the University of College London (UCL) ...
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