Although the therapeutic utility of CRISPR-based approaches has yet to be demonstrated, venture dollars are flowing into new companies developing the platform. Proof of concept may come faster than expected as new findings show that a CRISPR-based therapeutic can correct a mutation in adult mice with genetic liver disease.
TARGETS & MECHANISMS
Heart failure accounts for over half the deaths in patients with Friedreich's ataxia, but there are no therapies for the neurodegenerative disease. Now, a team of French researchers has shown that i.v. frataxin gene therapy could prevent or reverse heart failure in a mouse model of the disease.
Findings that link high IL-15 levels in tumors with increased local T cell proliferation and favorable patient outcomes position the cytokine as a robust prognostic and complement ongoing efforts to develop IL-15 as a therapeutic in colorectal cancer and other malignancies.
New evidence that miR-25 inhibition can help treat heart failure contradicts earlier results. Although the current study shows that blocking miR-25 restores SERCA2A -- and thus improves cardiac contractility -- discrepancies between the studies need to be resolved before a therapy can be developed.
Treating type 2 diabetes with a DPP-4 peptide vaccine; inhibiting Nav1.6 to alleviate optic neuritis; using cysteine dietary supplementation to prevent neurotoxicity associated with Huntington's disease; and more...
Detection of Plasmodium falciparum infection with ultrasound-based separation of red blood cells; blood-based DNA methylation signature for progressive supranuclear palsy and frontotemporal dementia; loss-of-function SMARCA4 mutations associated with small-cell carcinoma of the ovary, hypercalcemia type; and more...