Chimeric antigen receptor (CAR) T cells crossed the finish line as a cancer therapy, when the FDA approved Kymriah (tisagenlecleucel, formerly CTL-019, Novartis AG), in August.
The hallucinogenic drug psilocybin has a long history of religious and medicinal use, and is currently being tested in combination with therapy to treat a number of psychiatric conditions including addictions, obsessive compulsive disorder and treatment-resistant major depression.
A Gordian knot-like approach may be feasible in gene therapy for Rett syndrome. Researchers at the British University of Edinburgh have demonstrated that treating mice with a "radically truncated" gene that encoded about a third of the full-length MeCP2 protein, which is mutated in Rett syndrome, allowed the animals to survive long term with minimal symptoms.
Editing the myocilin gene, the most frequently mutated gene in primary open-angle glaucoma, reduced glaucoma symptoms in an animal model. Primary open-angle glaucoma is the most frequent form of glaucoma in the U.S., affecting roughly 3 million people.
One of the challenges for weight loss is that the body is evolutionarily programmed to worry about famines, not feasts. As a result, losing weight, if not exactly easy, is achievable for many individuals. Keeping that weight off, though, is a formidable challenge, because weight loss sets off powerful compensatory mechanisms in the body.