New treatment for cystic fibrosis
Cystic fibrosis, a devastating illness that clogs a victim’s lungs and renders them susceptible to infection, is a genetic disease—but one that may see an unorthodox new treatment in 2002.
Sufferers of the disease possess a damaged version of the CFTR gene, which helps lung cells to maintain proper fluid balance. Most past attempts at therapies have involved trying to insert functioning fragments of CFTR DNA directly into these cells. But these trials have met with dismal success rates because DNA is notoriously difficult to introduce into living tissue.
The CFTR gene—like all genes made out of DNA—has to be copied by the cell into RNA before it is useful. So U.S. researchers took advantage of this basic bit of biology and tricked CFTR-defective lung cells into accepting bits of RNA that code for the correct version of the damaged gene.
In quantitative studies, the researchers determined that 16% of all CFTR RNA inside the lung cells came from the foreign source and was therefore functional. Other research has suggested that a level of 8% is all that is required to rid the body of symptoms.
Better than bacon
Researchers in the U.S. have created a quartet of cloned piglets that have the potential to be organ donors for humans.
Scientists have always wanted to use pigs as transplant fodder because their organs are physiologically similar to those of humans and they are in plentiful supply.
But using regular pig organs in a transplant will almost certainly lead to rejection by the human host.
Cells in the human immune system use little sugary molecules that poke up from the surfaces of cells to recognize—and destroy—foreign tissue.
So scientists used genetic engineering techniques to destroy the gene that makes one particular cell-surface sugar molecule in cells from fetal pig tissue. In a procedure similar to that used to clone Dolly, the modified chromosomes from the fetal cells were injected into an egg cell. Over 3,000 of these engineered eggs were implanted into surrogate moms. Out of those 3,000, only seven piglets were born and four ultimately survived.
PPL Therapeutics, the Scottish firm that made Dolly, announced they’d developed their own method of making similar pigs; the announcement sent their stock price soaring by 46 per cent.