Cystic fibrosis (CF) is a single-gene disorder with insufficient treatment options and a target organ, the lung that is relatively easily accessible. Thus, it is not surprising that in the early ...

Using the Human Genome Sequence to Study Disease ... For example, cystic fibrosis is typically characterized as a single-gene disease associated with recessive mutations in the CFTR gene.

Collins: Well, cystic fibrosis is a very well-known example ... contains within it enough cells to be able to look at the DNA sequence of you or me or a newborn baby. So it would be realistic ...

In their 2019 paper, Liu’s team used prime editing to alter the gene mutations causing sickle cell disease ... a lung biologist and research director at Cystic Fibrosis Trust, who was not involved in ...

An explanation of the cause and effects of the genetic disorder cystic fibrosis and how it can be treated using gene therapy. There is a discussion about gene therapy which includes an interview ...