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The Unit 714 conducts basic and translational research on rare dermatological
Highlights
diseases, such as Epidermolysis Bullosa, Netherton syndrome, congenital Pachyo-

nychia, Xeroderma Pigmentosum and Kindler syndrome, among others. During
2013 a national clinical trial was awarded and we become partners of the GENE- ó

GRAFT European project, the first clinical trial of Gene Therapy for Epidermolysis

Bullosa which is entirely based on a strategy published and patented by our group.
Additionally, our Unit collaborated with various Spanish hospitals in the treatment

of chronic ulcers (bioengineered skin/cell therapy) in the context of COMPAS-

SIONATE USE. Also noteworthy is the transfer of the know-how for the production
of dermal substitutes to the Cell Therapy Unit of the Hospital Niño Jesus. Also at

translational level must be highlighted the activities of our unit in the molecular

diagnosis of the different forms of epidermolysis. This activity is recorded in OR-
PHANET and currently funded by CIEMAT. Finally, U714 has participated in creat-

ing patient REGISTRIES and carrying out epidemiological studies in collaboration

with ISCIII-Rare Disease Institute, other CIBERER Units and clinical researchers
from various hospitals.

Concerning transfer activity during 2013, it is remarkable the creation of a SPIN- 13
20
OFF and the exploitation of two of our patents for the treatment of skin and bone T
rare diseases.
OR
P
In 2013, U714 has been funded by Public National (1 project from SAF, 1 project RE
L
from FIS, 1 project from the Ministry of Health and Social Policy: Clinical Trial and A
NU
1 INNPACTO project) and Regional grants (2 projects from CAM) as well as pri- N
vate grants (1 project from Ramn Areces Foundation). The group also received A
R /
European funding (Eranet project outsourcing). Finally, is worthy to mention the E
granting of two intramural projects (ACCI).
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