I think we have reached a turning point ...
soon (I hope) a comment is a bit ' more personal.
gene therapy to treat Leber congenital dell'amaurosi
The New England Journal of Medicine have published the results of the first intervention gene therapy on ' man to treat Leber congenital amaurosis, a severe form of hereditary blindness.
The intervention was successful and is the result of a long and complex work of an international team that made the part of the Children's Hospital of Philadelphia and Naples, Telethon Institute of Genetics and Medicine (Tigem) and the Department of Ophthalmology of the Second University of Naples.
The Leber congenital amaurosis is a form of retinopathy of genetic origin and cause blindness or serious eye injury in the early months of life.
currently no cure, but gene therapy could be the right strategy to cure or at least against this disease.
During the clinical trial, doctors have injected into the subretinal space of the eye a viral vector containing a correct copy of the altered gene that causes the disease . The normal gene, it was plugged in the retina and produced the missing protein in individuals malati.I results been very positive. For some months after the treatment, all patients were observed significant improvements in visual function : improved retinal response to light, the ability to run tests of mobility and the perception of the visual field.
Moreover, the treatment did not cause toxic effects. Participated in this first clinical trial of a girl 19 years of Pavia and twins 26 years of Agrigento.
The Italian part of the research (funded by the Telethon Foundation) was coordinated by Alberto Auricchio, Tigem researcher and associate professor at the University Federico II of Naples. Also for the Tigem, have also collaborated Enrico Maria Surace and Sandro Banfi, which dealt with genetic screening of patients eligible for intervention.
The part about the recruitment and follow-up of patients were coordinated by Francesca Simonelli Department of Ophthalmology of the Second University of Naples, while the synthesis of viral vector and the surgery took place at Children's Hospital Philadelphia, under the supervision of Jean Bennett .
These results were presented in Rome by Alberto Auricchio and Francesca Simonelli, the presence of Andrea Ballabio (Tigem Director) and Susanna Agnelli (President of the Telethon). Now researchers
procederanno col testare l'assenza di tossicità anche a dosaggi più alti e su un numero maggiore di pazienti e valutando gli effetti a lungo termine .
La terapia genetica dimostra di essere efficace e destinata a migliorarsi sempre più e, con la recente scoperta di vettori in grado di trasferire nell'organismo geni di grandi dimensioni, si candida ad essere sfruttata anche per altre malattie causate dall'alterazione di geni di grandi dimensioni come la fibrosi cistica, la malattia di Stargardt, la distrofia muscolare di Duchenne e l'emofilia di tipo A.
Redazione MolecularLab.it (29/04/2008)
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