Many interesting papers have been published recently; here are our top three picks. They cover an innovative gene therapy trial, a new experimental approach for oncology, and the development of novel tools to map gene enhancers.

1. For the first time, an advanced CRISPR model has corrected a pathogenic mutation inside patients’ bodies. The disease, called alpha-1 antitrypsin deficiency, affects the lungs and liver. The biotech company conducting the clinical trial is Beam Therapeutics. The technique, known as base editing, functions more like a proofreader than a pair of genetic scissors. Science and The New York Times have both reported on this breakthrough.
2. A group at Tel Aviv University used CRISPR to disable a single gene in head and neck cancer cells, eliminating 50% of the tumors in an animal model. According to the authors, this approach had not been attempted before because it was believed that inactivating a single key gene would not be sufficient to achieve a therapeutic effect. The experiment was published in Advanced Science.
3. Finally, two American groups (Stanford and the University of California at San Francisco) used CRISPR to study hierarchies and interactions in gene regulatory networks. As explained in Nature Biotechnology, the human genome exhibits complex dynamics, with millions of regulatory elements orchestrating when, where, and how genes are turned on or off. On average, each gene is associated with about fifty sites that enhance its transcription — so-called enhancers. Our understanding of these networks has been limited because, until now, we lacked the right technologies to study them. New CRISPR-based approaches now make it possible to map them using “combinatorial perturbations.”