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Q: Where in the treated cells is CFTR actually produced?
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What cells are affected by cystic fibrosis?

The protein affected by cystic fibrosis is called the cystic fibrosis transmembrane conductance regulator or CFTR. CFTR acts as a channel that transports negatively charged chloride ions into and out of cells. This helps control the flow of water in tissues. CFTR also regulates the function of other channels that transport positive sodium ions into and out of cells.


When was CFTR - AM - created?

CFTR - AM - was created in 1962.


How can genetic engineering improve cystic fibrosis?

They isolated functional CFTR gene. Then they inserted the CFTR gene into nondisease-causing adenovirus virus. Last, they infected the patient´s airway cells with virus carryinhg the healthy CFTR gene.


How many introns are in CFTR?

none, CFTR is intron free.


What is the causes of cystic fibrosis?

Cystic fibrosis is caused by a mutation on the recessive allele of the CFTR gene. It is caused by a deletion of three adenine bases, so the CFTR protein produced has 1479 amino acids instead of 1480, although only one amino acid is missing this has an effect on the tertiary structure of the protein produced. The CFTR protein normally allows Chloride ions out of the epithelial cells of the airways, producing a more negative water potential outside of the cell and so water would usually move out of the cell and flush away the mucus. Since this protein is non-functional, water remains in the cell. Therefore this produces thick, sticky and viscous mucus, that can trap pathogens and cause disease for example.


What does a CFTR gene stand for?

It is the gene for cystic fibrosis. CFTR means Cystic Fibrosis Transport Regulator.


What do scientists currently believe is the cause of Cystic Fibrosis How are the faulty gene inherited and what direct effect does that have on the affected cells?

The faulty gene causes the misfolding of a protein called the Cystic Fibrosis Transmembrane conductance Regulator (CFTR). CFTR is responsible for the movement of chloride and sodium ions into and out of cells. The lack of salt and water on the surface of the cells causes the mucus to become extremely thick and sticky which builds up in and clogs organs.


People with cystic fibrosis inherit defective genetic information and cannot produce normal cftr proteins scientists have used gene therapy to insert normal DNA segments that code for the missing cft?

Offspring of someone with altered lung cells will inherit the normal CFTR gene.


What role does cftr play in the body?

The CFTR gene provides instructions for making a protein called the cystic fibrosis transmembrane regulator.


How does cystic fibrosis effect the cell membrane?

Cystic fibrosis is caused by defective CFTR protein. CFTR is an ion channel that transports chloride ions across epithelial cell membranes.


What happens to The protein called CFTR in cystic fibrosis?

Its name is CFTR and is a membrane channel for chlorine ions. Genetic defects in CFTR brings to an increased concentration of chlorine ions that lead to a greater viscosity of mucus in several mucosae in lungs and pancreas.


Where is the national headquarters of CFTR?

The national headquarters of the CFTR is located in the city of Boston. Boston is a city that can be found in the state of Massachusetts, which is part of the United States.