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

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 ...

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.

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 ...

CF is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. An estimated 105,000 people have been diagnosed with CF across 94 countries, according to the CF ...

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 ...

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 ...