Nat. Biotechnol. 36, 857–864 (2018).

The genetic disease phenylketonuria (PKU) prevents affected individuals from metabolizing phenylalanine; this metabolic function can be returned to the gut with engineered bacteria.

The inability of individuals with phenylketonuria to metabolize phenylalanine results in its accumulation in the brain and blood, neurological deficits and emotional and cognitive problems. It is currently treated with a prescription diet in conjunction with therapies.

Researchers from Synlogic, Inc. engineered a bacterial strain, activated in the anaerobic environment of the gut, that was able to metabolize phenylanaline in monkeys and mice. This approach shows promise for translation to humans and potentially also for treatment of other metabolic disorders.