Stem cells could combine with gene therapy to fight genetic diseases

Stem cells could combine with gene therapy

Research has combined stem cell technology and gene therapy for the first time, which could lead to genetic disorders being treated by medical insurance customers’ own cells.
The study, published in Nature, claims to have used cells from people with a genetic liver condition in order to generate a cell type called induced pluripotent stem cells, which have the ability to transform into other types of cells.


Researchers claimed it was a critical step towards being able to devise treatments, however, they warned that more safety tests needed to be carried before the technology could be used in the mainstream.
Currently, stem cells created from any medical insurance policyholder with a genetic disorder can’t be used to cure diseases as the cells are also likely to contain the corrupted genetic code that the original patient has.

Use of genetic technology to precisely remove mutations is a step forward in being able to develop personalised stem cells that could successfully treat human diseases. It is also suggested that the results of the study could help overcome some of the stumbling blocks that the research has previously faced.

The cirrhotic liver disease is being worked on by the Wellcome Trust Sanger Institute and the University of Cambridge. The disorder is caused by a change to a pair of letters within the genetic code, which is made up of six billion letters.
As a result of the disorder, antitrypsin – a protein which helps protect the body from damage – cannot escape from the liver, where it is made, and sufficiently provide protection.


It is one of the most common genetic diseases in the world, affecting one in every 2,000 people in Europe.
Currently, the only solution for the disorder is to undergo a liver transplant, however, this requires a lifetime of drugs in order to prevent an organ rejection.

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