Steven C Strutt, Rachel M Torrez ... Jennifer A Doudna
Divergent Cas9 enzymes direct site-specific single-stranded RNA cleavage, reducing infection by RNA phage in vivo and enabling programmable, PAM-independent repression of gene expression in bacteria.
Lentiviral protein transduction offers a new approach for administration of custom-designed nucleases, leading to high-efficacy targeted gene editing and reduced off-target activity after virus-directed delivery of programmable nuclease proteins.
Sara E Vazquez, Elise MN Ferré ... Mark S Anderson
Humans with mutations in the AIRE gene exhibit common autoantibodies targeting ovarian and intestinal antigens, including intestinal dysfunction-associated antibodies to enteroendocrine transcription factor RFX6.
An empirically derived scoring system for identifying optimal communication modules of aptazymes accelerates the development of ligand-responsive RNA genetic switches functional in mammalian cells.
Fabian Wehnekamp, Gabriela Plucińska ... Don C Lamb
The ability to follow single objects in 3D in a living organism with high spatiotemporal resolution opens new possibilities for quantitative biophysical studies of complex systems in their physiological context.
Arjun Khakhar, Colby G Starker ... Daniel F Voytas
A fungal bioluminescence pathway can be reconstituted in planta to create luminescence in many plant species without external substrate addition, and be used to design customizable reporters of gene-expression.
A deep mutational scan of the ADAR2 deaminase domain creates a map for designing tailored ADAR2 variants, and splitting the deaminase between residues 468 and 469 enables highly transcript-specific RNA editing.