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Last Updated: Tuesday, 24 June, 2003, 12:20 GMT 13:20 UK
Q&A: Gene therapy
The UK Government has announced millions of pounds for gene therapy research.

BBC News Online looks at the issues behind the recommendations.

What could gene therapy do for patients?

It could "correct" the genetic fault which causes conditions including muscular dystrophy and haemophilia. The possibilities offered by gene therapy are expanding all the time.

For example, just one gene is responsible for the life-threatening condition cystic fibrosis, in which organs in the body, particularly the lungs and pancreas, are clogged with thick, sticky mucus.

Scientists identified the gene in 1989, and they have been working on gene therapy for the condition ever since.

Laboratory and animal tests have been successful. Initial tests on patients have proved promising.

Jim Littlewood of the Cystic Fibrosis Trust, said: "Gene therapy for Cystic Fibrosis is a template for therapy for other diseases."

The UK Government announced 2.5m for CF genetic research and 3m for research into other single gene disorders.

How does gene therapy work?

Scientists try to correct the faulty genes responsible for a condition by "smuggling" working copies into the cells and hoping they will replace the problem originals.

They do this by using a virus, such as the common cold agent, to carry the replacement DNA into the cells.

The treatment has been successfully used, as in the case of the so-called "bubble boy" Rhys Evans, who suffered from a genetic condition which prevented him from developing a working immune system.

However, authorities in the United States have found that hundreds of experiments have failed and have caused a number of deaths.

And last year, authorities in France suspended gene therapy trials after a child developed leukaemia after undergoing treatment.

It is possible problems occur because the new DNA inserts itself into the chromosome at random positions, so if it lodges in the wrong position, it could trigger changes in the body that could lead to disease.

Are there other applications of genetic knowledge?

Yes. Scientists are discovering more about how individuals' genetic make-up determines how they respond to existing treatments.

There is also the potential to develop new, individually tailored treatments.

The government is also to ask the Human Genetics Commission to consider the case for screening babies at birth and storing information about their genetic profile so healthcare could be tailored to their individual needs and genetic make-up when they are older.

The commission is being asked to report back by 2004

If genes are identified for particular conditions, can doctors test to see who has faulty versions?

Yes. There are already many genes which can be tested for, including cystic fibrosis and breast cancer.

But there have been concerns there are not enough safeguards in place to ensure such testing does not lead to discrimination by employers, doctors and insurance companies.

There is already a ban on insurance companies using information from genetic testing, which will remain in place until 2006.

The government has promised to assess whether further regulation is needed.

The White Paper will also make it illegal to test a person's DNA without their consent.

The only exceptions will be if it is for medical treatment and gaining consent is impossible or it is part of a criminal investigation.

How widely available are genetic testing kits?

There has been concern about tests being available direct to the public without guidance, which genetics watchdogs have warned could provide false reassurance or cause unnecessary alarm.

There are currently no restrictions over which kits can be sold direct to the public in the UK.

But in April this year, the Human Genetics Commission, which advises the government on genetic issues, said the majority of tests should only be carried out under the supervision of a doctor.

Most tests are only available through specialist genetic testing services in the NHS.

But some, including tests which claim to predict the genetic risk of developing conditions such as heart disease, osteoporosis, asthma and some cancers are sold through alternative healthcare providers in the UK, along with recommendations about what supplements or medicines could help prevent the diseases.

Many more tests are marketed in the US, including one which can check for breast cancer risk. These are often available to patients in the UK over the Internet.

Dr Reid raised concerns that a lack of regulation in the US could be creating a "genetic underclass", and pledged this would not happen in the UK.

Millions for genetic technology
24 Jun 03  |  Health
Gene testing: The pros and cons
24 Jun 03  |  Health
Gene therapy trials halted
03 Oct 02  |  Health

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