GENET archive


8-Humans: UK authority gives green light for "designer baby"

   "Prof John Burn, of the department of clinical medical sciences at
    Newcastle University, said the decision would not lead to designer
    babies. 'I would use the analogy of simply replacing the battery in
    a pocket radio to explain what we are doing,' he said. 'You are not
    altering the radio at all, just giving it a new power source.' "

                                 PART I
------------------------------- GENET-news -------------------------------

TITLE:  Designer babies to wipe out diseases approved
SOURCE: The Telegraph, UK, by Roger Highfield
DATE:   9 Sep 2005

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Designer babies to wipe out diseases approved

Scientists have been given approval for experiments that could lead
within a few years to the first genetically altered babies being born in

The controversial decision to approve a radical form of gene transplant
offers the first realistic hope of an effective treatment for an entire
class of serious genetic diseases.

A team at the University of Newcastle hopes, in as few as three years, to
combine IVF with cell and genetic surgery to wipe out diseases caused by
the equivalent of faulty batteries in cells, including muscular dystrophy.

To do this, it would create a baby that would, in effect, be a blend of
genes from one man and two women. If a girl were born in this way, her
genetic alterations would be passed to future generations to free them of
potentially deadly disorders, too.

Campaigners from the pro-life movement said the decision by the Human
Fertilisation and Embryology Authority to license the work represented an
unacceptable step towards the creation of "designer babies" - a baby with
two mothers who threatened the family unit.

Josephine Quintavalle, of Comment on Reproductive Ethics, said the
authority had disregarded public opinion. The Human Genetics Alert
watchdog said the decision marked the first step towards human genetic

The Newcastle team wants to employ its method to wipe out 50 or so
metabolic disorders linked to faults in a small set of genes outside the
nucleus of cells.

These mitochondrial genes make and run the chemical "batteries" that
power body cells. In effect, the new technique would be like changing a
battery in a computer without affecting the hard disk.

When there are faults in mitochondrial genes, which are passed only from
women to their children, the results can vary from mild to catastrophic.

Sometimes a woman with malfunctioning mitochondria will suffer only
migraine. But, for reasons that are not understood, she can pass on many
more of her mitochondrial defects to her children. The resulting
metabolic turmoil can cause the build-up of lactic acid that will damage
the brain, muscle, heart and liver. Hundreds of families in Britain
suffer from these incurable diseases.

Prof Doug Turnbull, the leader of the Newcastle team, told The Daily
Telegraph last night that he was "delighted" by the authority's decision.

When considering the application twice before, the authority cited the
Human Fertilisation and Embryology Act 1990, which prohibits "altering
the genetic structure of any cell while it forms part of an embryo".
However, its appeal committee heard that the phrase "genetic structure"
had no precise scientific meaning.

Another objection rested on the ban on any proposal to change the nuclei
of cells, the technique used to clone Dolly the sheep. But the Newcastle
method is significantly different from cloning.

Prof Azim Surani, professor of physiology and reproduction at Cambridge
University, said: "It does not involve making a copy of an existing adult.

"I also see few ethical problems, as we are dealing with the embryo at a
very early stage when the cells have not even started to divide yet."

Prof John Burn, of the department of clinical medical sciences at
Newcastle University, said the decision would not lead to designer babies.

"I would use the analogy of simply replacing the battery in a pocket
radio to explain what we are doing," he said. "You are not altering the
radio at all, just giving it a new power source."

Prof Peter Braude, of King's College London, welcomed the authority's

"If [the technique] works and is safe it will be the answer to the
prayers of those people afflicted by these awful mitochondrial genetic
disorders, for which there is no treatment."

Dr David Harrison, the head of research at the Muscular Dystrophy
Campaign, said: "The innovative approach being tested by Prof Turnbull
may lead to a treatment for mitochondrial myopathies, a group of
conditions that dramatically affects the quality and length of life."

                                 PART II
------------------------------- GENET-news -------------------------------

TITLE:  Is there a case for modifying genes?
SOURCE: The Telegraph, UK, by Roger Highfield;
DATE:   31 Aug 2005

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Is there a case for modifying genes?

Researchers could help eradicate devastating childhood metabolic
disorders if given the legislative green light, reports Roger Highfield

Behind the doors of the authority that licenses embryo research in
Britain, an intense debate is taking place. Should scientists be allowed
to carry out a new kind of genetic therapy, one that offers the first
hope that an entire class of metabolic diseases could be wiped out?

What is fascinating about the proposal, a blend of IVF and gene
transplant, is that if a baby girl is born as a result of this genetic
therapy the beneficial effects would be passed down to future generations.

The proposal is radical because scientists have agonised for at least
three decades over what is called "germ-line" gene therapy - genetic
changes passed on in eggs and sperm - and remain divided over whether it
should be allowed.

On one side are those who would outlaw it because genes can interact in
complicated ways and the eradication of certain so-called disease genes
could have unexpected side effects. They fear errors in the resulting
"designer babies" could wreak havoc in a person's genetic legacy and
pollute the human gene pool.

Against them in the debate are the pragmatists who say these are
theoretical risks and should not be allowed to outweigh the very real
potential of germ-line therapy to reduce human suffering.

But the new proposal, from a team at the University of Newcastle, is not
like the usual brand of germ-line therapy - boys would not pass on the
genetic change to their children - and marks another example where
reproductive science outpaces legislation.

In the Newcastle proposal under consideration by the Human Fertilisation
and Embryology Authority, the suffering in question is of children who
are affected by 50 or so metabolic disorders linked to faults in a small
set of genes that make and run the chemical "batteries" that power cells.

These 37 genes are contained in mitochondria, lozenge-like structures,
the legacy of when bacteria invaded the cells of our ancestors two
billion years ago to trade chemical energy for a cosy home. Only 13 of
these genes actually work in the conventional way to make proteins.
However, mitochondria are crucial.

They provide the power for our bodies, from making hearts beat to helping
us to think. And we only get our mitochondria, and their genes, from our
mothers: every one of the 15,000 trillion that power your body is the
descendant of the 400 or so mitochondria in an egg.

Compared with the "nuclear" DNA coiled up in the chromosomes in the
compartment (nucleus) in each cell, which describes the proteins that
build and operate the rest of the body, mitochondrial DNA has been
relatively neglected by scientists: there is no easy way to alter
mitochondrial genes.

When there are faults in mitochondrial genes, the results can vary from
mild to catastrophic. A woman with malfunctioning mitochondria can suffer
only the occasional migraine. But, for reasons that are not understood,
she can sometimes pass on many more of her mitochondrial defects to her

The resulting metabolic mayhem can cause the build-up of lactic acid that
will damage the brain, muscle, heart and liver. In Britain alone,
hundreds of families suffer as a result of these incurable diseases.

Why not trade the faulty mitochondria for good ones? That is precisely
what is being proposed by Prof Doug Turnbull and embryologist Dr Mary
Herbert, whose efforts in Newcastle have been backed by the Muscular
Dystrophy Campaign.

Their proposal will involve taking the pronuclei - the chromosome-
containing residues of the egg and sperm - out of a fertilised egg and
transplanting them into an egg that has been donated from another woman,
under informed consent.

The donor egg would have been "enculeated" - emptied of its DNA within
the nucleus but which still contains the cytoplasm, the jelly around the
nucleus where the mitochondria sit. When the pronuclei are inserted into
this enucleated egg, the result would be an embryo with the nuclear DNA
from the parental egg and sperm but mitochondria - and mitochondrial DNA
- from the donor egg.

The idea that this method could end mitochondrial disease has already
been successfully tested in mice by Dr Lawrence Smith at the University
of Montreal - who once worked with Prof Ian Wilmut, the head of the team
that cloned Dolly the sheep - but it would take many years to perfect in

Unfortunately for the Newcastle team, its proposal has been rejected
twice already by the HFEA, which is nervous about how any approval would
be portrayed by the media.

The cell surgery that Newcastle would use to trade faulty with normal
mitochondria could be seen as a variant of the one used to clone Dolly
the sheep. It has to be a variant because the process of "nuclear
transfer" is banned when it comes to making babies. But any proximity of
the words "cloning techniques" and "baby" is enough to make many people
nervous, particularly since Newcastle University has the first team in
the west to clone human embryos.

In reality, however, the similarities rest only in details of the
micromanipulation methods used to carry out surgery on cells that are
around one tenth of a millimetre across. Babies that would be born after
the Newcastle proposal are anything but clones; they would actually be
conceived the normal way but would be powered by mitochondria from
another woman.

Herein lies the second PR nightmare: the journalistic shorthand for
offspring resulting this way is a "baby with three parents" or a "baby
with two mothers". The Newcastle team cringes at this description, since
the nuclear DNA that really makes us who we are consists of around 25,000
genes, compared with the paltry 37 that our mothers pass to us in

The third objection rests on how this transplant of 37 genes is seen by
some as tantamount to germ-line gene therapy. At present, scientists are
only comfortable with carrying out gene transplants to fix genetic
disease so long as they do not affect eggs and sperm. And usually gene
therapy concerns fiddling with one or two genes, not 37. If the
experience in America is anything to go by, it will end up being
portrayed as germ-line therapy, even though this is not strictly true.

There was a furore when the first human mitochondrial transplants were
carried out at the Institute for Reproductive Medicine and Science of St
Barnabas in New Jersey, in a controversial fertility treatment known as
cytoplasmic transfer, before the method had been tested properly on animals.

The cytoplasm carrying the mitochondria was removed from eggs from young
donor women and injected into those of older women in a bid to make
fertilisation more likely. As a result, the child's mitochondrial DNA was
a blend of that from two women.

But there were no controls, the amount of cytoplasm that was transferred
was small, and the evidence that it worked was unconvincing. Even though
the Newcastle team has joined in the criticisms of this work, it was
crudely branded germ-line therapy in the American media brouhaha.

An HFEA spokesman said that the Newcastle proposal has been rejected so
far because the Human Fertilisation and Embryology Act 1990 prohibits the
genetic modification of the embryo. The team is expected to make a formal
appeal at the end of September. Until a decision is reached by the HFEA,
the team has told me that it does not want to discuss its proposal.

The Newcastle team has good reasons to battle on. What it wants to do is
not like conventional germ-line therapy, since it would only affect
future generations if used to free a baby girl of the disease. It is
unlikely that the original intention behind the 1990 Human Fertilisation
and Embryology Act was to prevent such research, which was science
fiction 15 years ago, marking yet another scientific reason why the 1990
Act is under review.

As if to highlight how attitudes are softening, a consultation document
recently issued by the Government recommends that research on the use of
cell nuclear replacement to treat mitochondrial disease should be
allowed, subject to licensing. The same document says that germ-line
changes should continue to be banned, suggesting that mitochondrial
transplants are not germ-line changes.

In addition to patient groups such as the Children's Mitochondrial
Disease Network, the Newcastle proposal also has powerful backers in the
scientific establishment. "I hope their appeal will be successful," said
Prof Anne McLaren of Cambridge University, a former member of the HFEA
and a leading embryologist.

One problem, she said, is that the current wording of the 1990 Act makes
any licence for this work vulnerable to legal challenge by pro-life
groups. "The HFEA is quite right to be cautious," she said, adding that
she hopes the revised Act will make this kind of treatment easier to license.

As for the claims that the resulting babies would have two mothers, Prof
McLaren commented: "I think two mothers might be even better than one
mother. Speaking as a mother, I would have no problems with that. No
problems at all."

Even full blooded germ-line therapy - alteration of nuclear DNA - has
powerful supporters, so long as it is safe. Prof James Watson, who shared
the Nobel Prize for his work on DNA's double helix, is one enthusiastic
proponent: DNA manipulation could be used to make people resistant to the
ravages of HIV, or transform slow learners into fast ones, according to
Watson, who has often voiced his backing for "simply putting right what
chance has put horribly wrong. You should be allowed to improve human life.''

At the end of 2002, the International Forum for Biophilosophy in Brussels
saw delegates backing research leading toward safe and effective human
germ-line genetic modification. They concluded that germ-line therapy
does not violate human rights, including any "so-called right to be born
with a human genome that has not been modified by artificial means".


European NGO Network on Genetic Engineering

Hartmut MEYER (Mr)
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