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While the original version made use of tiny fat droplets to carry the DNA, the new one makes use of a hybrid virus.
The researchers have modified a monkey lentivirus — a kind of virus that can insert genetic material in the host cell — to carry the external ‘targeting’ proteins of a virus that naturally infects the lung.
“Normally with gene therapy, you are delivering small amounts to the brain or the eye, but for the lung you will need a considerable amount of viral vectors, especially if you’re going to nebulize it because maybe half of the vector will be lost just from the nebulization and, obviously, breathed out.”If the therapy proves able to modify enough lung cells, the partners expect the therapy would only need to be given every 3-5 years, or even just once.
It would also remove the need for any other medications for lung function.
With this technology it would be possible to revert mutations directly in the CFTR gene, although this kind of treatment would need to be tailored to each specific mutation causing cystic fibrosis.
back in 2015 to target cystic fibrosis using CRISPR/Cas9.
The function of the pancreas, liver and bowel is also affected — a single mutation ends up damaging the whole body.
Currently available treatments are still not able to fully address the complexity of the disease.“The problem is that the most success has been observed in cystic fibrosis patients with only specific classes of mutation, and since there are nearly 2,000 different mutations in 6 different classes, many patients still have no treatment.”“New treatments are urgently needed for cystic fibrosis patients who do not benefit from CFTR repair drugs.
In particular, the company targets ion channel proteins that control fluid secretion and mucus clearance from the airways.There is still a long way to go before this kind of practice becomes part of the standard care for patients with cystic fibrosis.But as scientists learn more about the multiple levels of complexity of this disease, the future is looking brighter for patients with the condition.The cause of cystic fibrosis is very straightforward. People diagnosed with cystic fibrosis have a mutation in a gene called CFTR.This gene encodes a protein that is responsible for transporting chloride to the surface of cells.
Only one other research group, at the University of Iowa, is working on a gene therapy for cystic fibrosis, but it is not as advanced as its UK counterpart.