Thursday 16 October 2014

Gene therapy for Cystic Fibrosis

Original paragraph:   

       
Scientists found that a laboratory-created "super" virus could be used to deliver a healthy version of a gene into cells affected by cystic fibrosis, ridding them of the disease.

The discovery is the latest boost for a technique called gene therapy, which scientists hope can attack diseases at their root.
Previous studies have shown that scientists can "turn off" the faulty gene which causes cystic fibrosis by replacing it with a healthy copy.
Tests on human lung cells with cystic fibrosis showed that they were free from the disease after being treated with the virus.
Viruses are one of two key ways that scientists hope they can carry healthy genes into sufferer's cells.
Human trials have already begun into the other method in which liposomes, natural fatty substances which stick to the outside of cells and encourage viruses to enter them, are used to transfer the genes.
The disease, which affects the lungs and causes breathing difficulties, has few known treatments and average life expectancy is just 31.
The scientists modified a harmless virus, known as adeno-associated virus (AAV), which is carried by 90 per cent of the population, into a highly infectious form several hundred times stronger than normal.
The new virus is more effective at entering lung cells than its natural ancestor and better at evading becoming detected by the body's natural defence systems.
The researchers now hope to test the newly created virus in the first ever animal models of the disease, pigs bred last year, and then in humans.
The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene.
Although the results are positive in human lung tissue in a laboratory, it remains to be seen if it would work in the patient.”

 Key ideas:



  •  Scientists found a virus that could be used a new healthy gene towards the cells that are affected by cystic fibrosis
  • Tests on human lung cells showed that they were free from the disease after being treated with the virus.
  • Viruses are the key scientists hope can carry healthy genes
  • average life expectancy is 31
  • they have created a modified virus known as adeno-associated virus (AAV)
  • The virus is effective at entering lung cells and better at becoming detected by the body's natural defence systems.  
  • The virus only carries a shortened version of the CF gene and it remains to be seen if it has the same function as the whole gene.
The picture describes its procedure on how gene therapy is used to cure such diseases and how new healthy genes are transmitted into the body . 
http://www.bio.miami.edu/dana/pix/gene_therapy.jpg

Source in MLA:
"Cystic Fibrosis Sufferers Offered Hope by Gene Therapy Treatment." The Telegraph. Telegraph Media Group, 28 Sept. 0016. Web. 14 Oct. 2014. <http://www.telegraph.co.uk/health/healthnews/4639764/Cystic-fibrosis-sufferers-offered-hope-by-gene-therapy-treatment.html>.

Paraphrasing:
Through a technique called gene therapy , researches are truly convinced that it has the capability to attack diseases that are inherited. For a disease such as Cystic Fibrosis, scientists have discovered a harmless virus known as adeno-associated virus (AAV) as one of the solution considered since viruses was one of the main method hoping it could replace affected cells of such sickness into healthy ones. As stated, the average life expectancy of a person who received Cystic Fibrosis is 31 where human trials were now done in a procedure which includes fatty substances as well as liposomes as a path to lead the certain virus and transport new genes. In result, individuals whom received tests for cystic fibrosis were then free from the disease due to its effectiveness and its ability to be easily detected by our body's natural defence systems. However , we may not know how this treatment may impact various individuals with different body situations.



 The interview talks in detail about gene therapy as a possible treatment.
  https://www.youtube.com/watch?v=oC0q7s2udkQ


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