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Home » Molecular scissors for blistering skin disease

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Molecular scissors for blistering skin disease

State: 
Declined
Contact Details
I am the event manager: 
I am the event manager
Event Manager Info
1. Event Manager Organisation: 
Newcastle University
2. Event Manager First Name: 
Julia
3. Event Manager Surname: 
Dr Reichelt
4. Event Manager Address line 1: 
Institute of Cellular Medicine
5. Event Manager Address line 2: 
Medical School
6. Event Manager Address line 3: 
Newcastle upon Tyne
7. Event Manager Postcode: 
NE2 4HH
8. Event Manager Telephone number: 
01912225074
9. Event Manager Email address: 
julia.reichelt@ncl.ac.uk
Your event
10. Which of the following areas of science does you event best fit into?: 
Medical Sciences
Event type and format
11. Do you have a preferred day for your event to take place on?: 
Saturday 7 September 2013
Sunday 8 September 2013
17. Length: 
2 hour
18. What will the format of your event be?: 
Talks/presentations - 1 person presenting new science or for a specific audience with Q & A; or a TED talk
19. Please give any details about the format of your event you feel are relevant.: 
Dr Julia Reichelt gives a short presentation followed by a discussion (Q&A). This is supported by an exhibition of a number of posters, each presented by a scientist or a student. The poster presentation gives the audience an opportunity to get additional information at their own pace and ask the presenters further questions in a relaxed environment. A 3D model of the 'molecular scissors' helps the audience understand the science behind these novel tools for gene therapy.
20. Location/venue: 
Venue and location to be allocated by the Festival team
21. Venue requirements: 
We require a seminar room or small lecture theatre with screen/projector for the oral presentation. Within this room (or adjacent to it) we require a reception area which provides space for 5 poster boards (including sufficient space for about 6 people at each poster), and a small table. We require 5 poster boards (portrait format) and a small table (e.g. bistro-style) to accomodate the 'molecular scissor' 3D model. Campus location would be preferred.
22. Audience Level: 
Adults with some knowledge of the topic
Event details
12. Event title: 
Molecular scissors for blistering skin disease
13. Description of the core message of your event and key topics to be covered (c.100 words): 
Inherited blistering skin diseases are extremely painful and often life-threatening. There are currently about 5000 people living with this type of disorder in the UK, and there is no cure available. This short talk introduces blistering skin diseases and the gene therapy approach currently being developed by my research team to cure patients suffering from the blistering skin disease EBS. A number of posters presented by scientists and students, provide further information about the structure of the skin and how mutant genes cause blistering skin diseases. These scientists will explain how we aim to destroy disease-causing mutant genes using ‘molecular scissors’ and why our gene therapy targets skin stem cells.
14. Does the event have particular local relevance?: 
Yes
15. If so, please indicate what this is: 
The BBC documentary 'The boy whose skin fell off' which describes the life and suffering of a young man from a village close to Newcastle - Jonny Kennedy - with an inherited blistering skin condition. He passed away due to the disease aged only 36. This devastating disease is still incurable. We are currently developing a gene therapy for blistering skin diseases using 'molecular scissors' to destroy the disease-causing gene in the patients' skin stem cells providing a permanent cure.
16. How does your event relate to this year's theme of 'Making waves'?: 
If our gene therapy approach using ‘molecular scissors' to destroy the disease-causing gene in inherited blistering skin diseases works it shows proof of principle and can be adapted to treat other inherited diseases. In this way, our approach will have larger impact and be 'making waves'.
Press interest
23. Do you consider that the event will attract press interest?: 
Yes
24. If yes, please summarise up to 4 key points you think will be of press interest: 
Stem cells, gene therapy, 'molecular scissors' (designer nucleases such as TALE and zinc-finger nucleases), inherited blistering skin diseases.
25. Do you have any other comments you would like to make or information you would like to include?: 
The Newcastle group is the only research group in the UK using 'molecular scissors' to develop a treatment for blistering skin diseases. Treatment of skin stem cells will ensure a permanent cure of the disease as this is the cell type which is responsible for skin regeneration. We have recently shown that 'molecular scissors' work in mouse skin stem cells (Höher et al, 2012, Stem Cell Reports and Reviews 8:426-34) which led to a press release by our funder, the British Skin Foundation, and subsequent press coverage. 'Molecular scissors' have great potential for various types of gene therapies. The first clinical trial using 'molecular scissors' aiming to prevent disease progression in HIV patients is currently ongoing in the USA. Newcastle University, with its North East Stem Cell Institute (NESCI) including a running 'Good Manufacturing Practice' (GMP) facility, and its close connection to the Royal Victoria Infirmary (RVI) is an ideal site for our research, for subsequent first in man trials, and, future applications of our therapy approach in patients.

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