Released on: September 2, 2008, 8:14 am

Press Release Author: BeyondPR

Industry: Healthcare

Press Release Summary: Research published this week in the journal Nature gives
millions of deaf and hard of hearing people new hope of new gene or drug treatments
for deafness and has been welcomed as a significant breakthrough by the country’s
only medical research charity for deaf people, Deafness Research UK.

Press Release Body: Research published this week in the journal Nature gives
millions of deaf and hard of hearing people new hope of new gene or drug treatments
for deafness and has been welcomed as a significant breakthrough by the country’s
only medical research charity for deaf people, Deafness Research UK.

Deafness Research UK Research Advisory Panel member, Professor Guy Richardson,
praised the work as “a technical tour de force, and very convincing proof of the
principle that gene therapy could, at least in certain cases, be used to cure
deafness”.

There are nine million deaf and hard of hearing people in the UK and in most cases
deafness results from loss of sensory cells in the inner ear known as “hair” cells.
The cells can be damaged and lost through ageing, noise, genetic defects and certain
drugs and, because the cells don’t regenerate, the result is progressive - and
irreversible - hearing loss. Damage to these cells can also lead to tinnitus which
affects around 5 million people in the UK.

The latest research, by a team at the Oregon Health & Science University, shows that
a key gene known as Atoh1 (also known as Math1) can not only cause cells to develop
into hair cells but that these cells function like normal hair cells.

Vivienne Michael, Chief Executive of Deafness Research UK said: “This is an
important and exciting step along the road towards an effective medical treatment
for deafness. Deafness Research UK has a long history of supporting research into
the repair and regeneration of the sensory cells in the inner ear, including work on
the Atoh1 gene and on the use of stem cells to restore hearing. We will continue to
work towards getting a cure for unwanted deafness into the clinic.”

John Brigande, Assistant Professor of Otolaryngology at the Oregon Hearing Research
Center in the OHSU School of Medicine said “Our work shows that it is possible to
produce functional auditory hair cells in the mammalian cochlea.”

Brigande and colleagues were able to produce hair cells by transferring a key gene
called Atoh1 (also known as Math-1) into ‘progenitor’ cells in the inner ear of
developing mice. This type of cell becomes specialised to perform different
functions during development, according to the instructions they receive from genes.
The gene Atoh1 is known to turn progenitor cells into hair cells, but it was not
previously known whether the hair cells would work normally if Atoh1 was introduced
artificially.

To find out, the team inserted Atoh1 into progenitor cells along with a fluorescent
protein molecule that is often used in research as a marker, to make cells easily
visible. They were then able to see that the gene transfer technique resulted in
mice being born with more hair cells in the cochlea than are normally found.

Crucially, Dr. Anthony Ricci, Associate Professor of Otolaryngology at the Stanford
University School of Medicine, demonstrated that the gene-treated hair cells
function like ordinary hair cells.

Stem cells are a type of progenitor and so can be instructed by genes to become a
specific cell-type. One obstacle in the way of stem cell research has been ethical
objection to the use of embryonic stem cells. However, researchers at Sheffield
University supported by Deafness Research UK are currently working on extracting
stem cells from the bone marrow and blood that are found in the umbilical cord, with
the aim of turning them into hair cells that could be inserted into the cochlea. The
umbilical cells are in rich supply and avoid the ethical issues surrounding the
embryonic cells.

ENDS






Notes to editors
About Deafness Research UK
• Deafness Research UK is the country’s only charity dedicated to finding new cures,
treatments and technologies for deaf, hard of hearing and other hearing impaired
people.
• The charity supports high quality medical research into the prevention, diagnosis
and treatment of all forms of hearing impairment including tinnitus.
• The Deafness Research UK Information Service provides free information and advice
based on the latest scientific evidence and informed by leading experts. The
Information Service can be contacted on Freephone 0808 808 2222
• For more information on research into deafness, tinnitus and other hearing
conditions, log on to the website at www.deafnessresearch.org.uk where you can
access a wide range of information. Alternatively you can e-mail Deafness Research
UK at info@deafnessresearch.org.uk
• One in seven people in the UK – almost nine million people - suffer hearing loss.
• Deafness Research UK was founded in 1985 by Lord (Jack) and Lady Ashley of Stoke.
• In January 2008, Action for Tinnitus Research (ATR) was linked with Deafness
Research UK under a uniting direction order under Section 96 (6) of the Charities
Act 1993.



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Ref: DRUK0088 – Gene therapy