CFTR, the Odd ABC Transporter Responsible for Cystic Fibrosis
Dr. Chen is the William Ford Professor at Rockefeller and also a Howard Hughes Medical Institute Investigator. She studies ATP-binding cassette (ABC) transporters, a diverse group of membrane proteins integral to almost every biological process. In humans, ABC transporters make up one of the largest gene families, and more than a dozen genetic diseases have been traced to ABC transporter defects. ABC transporters are also central to multidrug resistance in many pathogenic bacteria and in tumor cells. In humans, ABC transporters make up one of the largest gene families, and more than a dozen genetic diseases have been traced to ABC transporter defects. ABC transporters are also central to multidrug resistance in many pathogenic bacteria and in tumor cells. By pursuing structural and mechanistic studies of ABC transporters, she hopes to understand how nature utilizes the chemical energy of ATP hydrolysis to perform work – transporting substrates against their chemical gradients. Currently, she is focusing on the following questions: 1. How anti-cancer drugs are ejected by ABC transporters? 2, How antigens are transported and loaded onto MHC-I molecules? and 3, The root cause of cystic fibrosis.
Dr. Chen's WALS lecture will detail her lab's effort to understand the root cause of cystic fibrosis, the most common lethal genetic disorder in populations of Northern European descent, affecting one out of every 2500 newborns. Cystic fibrosis is caused by mutations in a single gene, the cystic fibrosis transmembrane conductance regulator (CFTR). CFTR belongs to the ABC superfamily. Yet whereas other ABC transporters utilize the chemical energy of ATP hydrolysis to transport substrates against their chemical gradients, CFTR conducts anions down their electrochemical gradient. By combining structural biology, electrophysiology, and medicinal chemistry in studying CFTR, Chen's lab hopes to contribute to treating—and eradicating—this devastating disease.
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