Scientists have created genetically-engineered mice with artificial human chromosomes in every cell of their bodies, as part of a series of studies showing that it may be possible to treat genetic diseases with a radically new form of gene therapy.
In one of the unpublished studies, researchers
made a human artificial chromosome in the
laboratory from chemical building blocks rather
than chipping away at an existing human
chromosome, indicating the increasingly
powerful technology behind the new field of
synthetic biology.
The development comes as the Government
announces today that it will invest tens of millions
of pounds in synthetic biology research in Britain,
including an international project to construct all
the 16 individual chromosomes of the yeast
fungus in order to produce the first synthetic
organism with a complex genome.
A synthetic yeast with man-made chromosomes
could eventually be used as a platform for
making new kinds of biological materials, such as
antibiotics or vaccines, while human artificial
chromosomes could be used to introduce
healthy copies of genes into the diseased organs
or tissues of people with genetic illnesses,
scientists said.
Researchers involved in the synthetic yeast
project emphasised at a briefing in London earlier
this week that there are no plans to build human
chromosomes and create synthetic human cells
in the same way as the artificial yeast project. A
project to build human artificial chromosomes is
unlikely to win ethical approval in the UK, they
said.
However, researchers in the US and Japan are
already well advanced in making “mini” human
chromosomes called HACs (human artificial
chromosomes), by either paring down an
existing human chromosome or making them
“de novo” in the lab from smaller chemical
building blocks.
Natalay Kouprina of the US National Cancer
Institute in Bethesda, Maryland, is part of the team
that has successfully produced genetically
engineered mice with an extra human artificial
chromosome in their cells. It is the first time such
an advanced form of a synthetic human
chromosome made “from scratch” has been
shown to work in an animal model, Dr Kouprina
said.
“The purpose of developing the human artificial
chromosome project is to create a shuttle vector
for gene delivery into human cells to study gene
function in human cells,” she told The
Independent. “Potentially it has applications for
gene therapy, for correction of gene deficiency in
humans. It is known that there are lots of
hereditary diseases due to the mutation of certain
genes.”
Synthetic biology is loosely defined as designing
new kinds of life-forms or making new genetic
arrangements that do not exist in nature, which
could provide practical benefits to society, notably
in medicine, manufacturing or environmental
monitoring.

independent.co.uk/news/science/exclusive-mice-with-human-chromosomes--the-genetic-breakthrough-that-could-revolutionise-medicine-8701357.html