ESPE Yearbook of Paediatric Endocrinology

To read the full abstract: Nature, Vol. 560, p. 291, 10 August 2018For the first time, a drug based on the mechanism gene-silencing through RNA interference (RNAi) has received regulatory approval. We can expect that this is a forerunner of a new class of drugs targeting disease-causing genes.It has been known for 20 years that short RNA molecules can attach ...