The cystic fibrosis gene: a molecular genetic perspective

LC Tsui, R Dorfman - Cold Spring Harbor …, 2013 - perspectivesinmedicine.cshlp.org
LC Tsui, R Dorfman
Cold Spring Harbor perspectives in medicine, 2013perspectivesinmedicine.cshlp.org
The positional cloning of the gene responsible for cystic fibrosis (CF) was the important first
step in understanding the basic defect and pathophysiology of the disease. This study aims
to provide a historical account of key developments as well as factors that contributed to the
cystic fibrosis transmembrane conductance regulator (CFTR) gene identification work. A
redefined gene structure based on the full sequence of the gene derived from the Human
Genome Project is presented, along with brief reviews of the transcription regulatory …
The positional cloning of the gene responsible for cystic fibrosis (CF) was the important first step in understanding the basic defect and pathophysiology of the disease. This study aims to provide a historical account of key developments as well as factors that contributed to the cystic fibrosis transmembrane conductance regulator (CFTR) gene identification work. A redefined gene structure based on the full sequence of the gene derived from the Human Genome Project is presented, along with brief reviews of the transcription regulatory sequences for the CFTR gene, the role of mRNA splicing in gene regulation and CF disease, and, various related sequences in the human genome and other species. Because CF mutations and genotype-phenotype correlations are covered by our colleagues (Ferec C, Cutting GR. 2012. Assessing the disease-liability of mutations in CFTR. Cold Spring Harb Perspect Med doi: 10.1101/cshperspect.a009480), we only attempt to provide an introduction of the CF mutation database here for reference purposes.
perspectivesinmedicine.cshlp.org