At the end of 2023, the FDA approved Casgevy for sickle cell disease, its first approval for a therapeutic that used the genome editing tool clustered regulatory interspaced palindromic repeats ...
One person's side effect could be another person's treatment if we expand our perspective on small molecule drug targets, according to a new study published November 5, 2025, in npj Precision Oncology ...
Small interfering RNA (siRNA) drugs are a class of therapeutic agents that silence specific genes associated with inherited diseases. However, siRNA drugs have challenges because siRNAs often silence ...
For more than 70 years, hydralazine has helped doctors lower dangerously high blood pressure during medical emergencies, ...
In a new study published in the Clinical and Translational Medicine, the research group of Prof. Chen-Yu Zhang and Prof. Xi Chen at Nanjing University evaluated the on- and off-target effects of ...
CRISPR-Cas9 gene editing technology is widely used to to help study genes of interest and modify disease-associated genes. However, the system is associated with adverse effects, including mutations ...
One person's side effect could be another person's treatment if we expand our perspective on small molecule drug targets, according to a new study published November 5, 2025, in npj Precision Oncology ...
Zidesamtinib targets ROS1+ NSCLC patients with prior TKI treatment, addressing CNS-penetrant treatment challenges and ...
At the 2025 Society for Neuroscience meeting, scientists discussed the adverse side effects of GLP-1 agonists and new ...
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