Category Archives: cystic fibrosis

East Coast Truckers take youngsters on a special day out

 With a chorus of honking horns and lots of smiling faces, a convoy of trucks set off from County Hall, in Norwich, for an extra special day out.

The East Coast Truckers set off from County Hall on their charity convoy. Oliver Walsh with his driver Arron Peters.

19:50 28 August 2016

The East Coast Truckers children’s convoy took place today, and saw more than 60 trucks take scores of excited youngsters on a trip from Norwich to Pleasurewood Hills and Great Yarmouth.

They were also joined by members of the emergency services for the 70-vehicle procession which created a great spectacle on the county’s roads.

The East Coast Truckers have become famous for the annual event – now in its 31st year – which aims to give children with disabilities or from disadvantaged backgrounds a day they will never forget.

Find out more

 

 

Cystic fibrosis drug offers hope to patients

  • 17 May 2015
  • From the section Health

A “groundbreaking” cystic fibrosis therapy could profoundly improve patients’ quality of life, say doctors.

Patients often die before their 40s as mucus clogs and damages their lungs and leaves them prone to infection.

Big Advance Against Cystic Fibrosis: Stem Cell Researchers Create Lung Surface Tissue in a Dish

Harvard stem cell researchers at Massachusetts General Hospital (MGH) have taken a critical step in making possible the discovery in the relatively near future of a drug to control cystic fibrosis (CF)

 

ScienceDaily (Apr. 5, 2012) — Harvard stem cell researchers at Massachusetts General Hospital (MGH) have taken a critical step in making possible the discovery in the relatively near future of a drug to control cystic fibrosis (CF), a fatal lung disease that claims about 500 lives each year, with 1,000 new cases diagnosed annually.


Beginning with the skin cells of patients with CF, Jayaraj Rajagopal, MD, and colleagues first created induced pluripotent stem (iPS) cells, and then used those cells to create human disease-specific functioning lung epithelium, the tissue that lines the airways and is the site of the most lethal aspect of CF, where the genes cause irreversible lung disease and inexorable respiratory failure.

That tissue, which researchers now can grow in unlimited quantities in the laboratory, contains the delta-508 mutation, the gene responsible for about 70 percent of all CF cases and 90 percent of the ones in the United States. The tissue also contains the G551D mutation, a gene that is involved in about 2 percent of CF cases and the one cause of the disease for which there is now a drug.

The work is featured on the cover of this month’s Cell Stem Cell journal. Postdoctoral fellow Hongmei Mou, PhD, is first author on the paper, and Rajagopal is the senior author.

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