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 ...

The Gene Expression Core was founded in 1994 and promotes translational, multidisciplinary CF research by furnishing expertise concerning recombinant CFTR and other proteins relevant to CF disease ...

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.

A new mega-database of half a million mutations may flag new ways of treating genetic disease, scientists say.

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 ...

No, not yet, but our knowledge of the human genome sequence has enabled the development ... including various membrane transport disorders (cystic fibrosis), coagulation disorders (hemophilia ...

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 ...

An international study has identified two types of these so-called dysbioses in cystic fibrosis. They display ... Landmark Study Involving Babies in Ireland Supports Use of Cystic Fibrosis Drug in ...

A gene is a fundamental unit of heredity ... This approach has shown promise for treating conditions such as sickle cell anemia, cystic fibrosis, and certain types of cancer. The study of genes and ...

Viral and nonviral clinical trials have established proof-of-principle for gene transfer into the cystic fibrosis (CF) lung. It is currently unknown whether gene transfer efficiency is sufficient ...

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 ...