Researchers accidentally reprogrammed mature neurons into dopamine-producing neurons—a discovery that could spark new therapies for brain diseases.
CRISPR-GO allows scientists to reorganize the genome—taking specific pieces and placing them at new locations in a cell’s nucleus.
Spark Therapeutics announced encouraging preclinical data on SPK-3006, its gene therapy to treat the muscle disorder Pompe.
Two separate teams used modified versions of CRISPR-Cas9 to correct rare but lethal inherited liver diseases.
Some microRNAs could serve as prognostic markers of Crohn's and open the possibility of developing personalized therapies.
A mouse study and a human population study find chemotherapy might compound cognitive deficits in cancer patients with the APOE4 gene.
An NCI team is reporting promising results from a small trial of a dendritic-cell vaccine in patients whose tumors overexpress HER2.
Insights into how bacteria hibernate and use metals to evade destruction by antibiotics could be useful in drug development.
Two dual EGFR/HER2 inhibitors from Boehringer Ingelheim and Puma showed in vitro activity against AML cancer cells.
In a small study, HIV patients who got two broadly neutralizing antibodies maintained viral suppression for 21 weeks.