May the result be a promising one. Let us all pray the almighty.
With regards
Jaison Bellarmine-----Original message-----
From: Wahid Raza
Sent:  2011:10:28, 3:34  pm
To: accessindia
Subject: [AI] Fwd: A Gene therapy trial news from U.K.


Hi all,
pasting below article, which get from another list.
Regards
Wahid

---------- Forwarded message ----------

hi friends,
The following is the news published 27-10-2011 on park forest about the 1st
patient got treatment this week. let cheack it... this is for info only.
****************************************************************************************
United Kingdom--(ENEWSPF)--27 October 2011.  The first patient to receive
gene therapy for an incurable type of blindness was treated at the John
Radcliffe Hospital in Oxford this week as part of a trial led by Oxford
University.

*Story continues below:*

*eNews Park Forest is an independently owned and operated electronic
publication and has no affiliation whatsoever with the governing bodies of
the Village of Park Forest.*

If successful, the advance could lead to the first-ever treatment for
choroideraemia, a progressive form of genetic blindness that first arises in
childhood and is estimated to affect over 100,000 people worldwide.

‘This disease has been recognised as an incurable form of blindness since it
was first described over a hundred years ago. I cannot describe the
excitement in thinking that we have designed a genetic treatment that could
potentially stop it in its tracks with one single injection,’ says Professor
Robert MacLaren, professor of ophthalmology at the University of Oxford and
an honorary consultant at the Oxford Eye Hospital and Moorfields Eye
Hospital, who is leading the trial.

Jonathan Wyatt, 63, an arbitration lawyer from Bristol had the surgery at
the Oxford Eye Hospital based at the John Radcliffe – the main NHS centre
for this trial. He is the first of 12 people in this initial human trial
that will receive the novel gene therapy.

Mr Wyatt was diagnosed with choroideraemia in his late teens and has
suffered progressive sight loss ever since. He now sees only blackness
except for a small area of a few degrees in diameter in the centre of his
vision.

Choroideraemia is a genetic disease that leads to progressive degeneration
of the retina in the eye. It generally affects males only and there is no
treatment. The diagnosis is usually made in childhood and leads to blindness
in men by their forties. It occurs due to deficiency of the REP1 gene
located on the X chromosome.

The novel gene treatment was developed by Professor MacLaren at Oxford
University in collaboration with Professor Miguel Seabra at Imperial College
London. It is designed to provide the gene missing in people with
choroideraemia to stop the deterioration that gradually leads to blindness.

It uses a virus essentially as a delivery vehicle that ferries DNA including
the missing gene into the right part of the eye. The virus has been
engineered to infect the light-sensitive cells in the retina known as
photoreceptors. There the gene is switched on and becomes active.

With this particular gene therapy, the treatment could provide a one-off
permanent correction of the disease because the gene is thought to remain in
the retinal cells indefinitely.

‘This trial represents the world’s first ever attempt to treat this disease
and the first time that gene therapy has been directed towards the
light-sensitive photoreceptor cells of the human retina,’ says Professor
MacLaren. ‘This represents a major breakthrough and is highly significant
for patients who are losing sight from other photoreceptor diseases, such as
retinitis pigmentosa.’

The trial will see 12 patients undergo surgery in which the gene therapy is
injected into one eye. The other eye would then act as a control against
which to assess any treatment effect. The researchers would however aim to
go on to treat the second eye, should the treatment be proven to be
effective.

The aim of the trial is primarily to assess safety, but it will also gain
initial data on how effective the treatment is. The researchers estimate
that it will take two years to know whether or not the degeneration has been
stopped completely by the gene therapy.

‘While safety appears so far to be fine, the efficacy of the gene therapy
will only be evident after 24 months. We need this time to measure any
effect as the degeneration caused by choroideraemia is slow,’ explains
Professor MacLaren.

The clinical trial is funded by a grant awarded to the University of Oxford
by the Health Innovation Challenge Fund – a translational award scheme
funded jointly by the Wellcome Trust and the Department of Health.

Professor Seabra, who played a key role at Imperial College London in
identifying the gene causing choroideraemia and in eliciting the mechanism
of cell death in the retina, comments: ‘The ability to offer a gene
replacement treatment for these patients was the final objective of 20 years
of intense research in my laboratory. This is a moment of fulfilment for us
and a dream come true for all choroideraemia patients.’*Professor Robert
MacLaren and Dr Zinkernagel carry out the gene therapy treatment. Credit:
BBC News**Jonathan Wyatt, a barrister from Bristol and the first patient to
receive gene therapy for choroideraemia. Credit: BBC News*
Source: ox.ac.uk
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