Newsgroup: it.scienza.medicina
Titolo: Terapia genica

Salve a tutti!
Studio medicina e a corso di biologia molecolare mi è stata fornita una
"panoramica" delle diverse metodologie applicabili nella terapia genica.
Ecco la domanda: delle numerose strade promettenti che si conoscono e si
stanno sperimentando per correggere il genoma o il trascritto, ve ne
sono alcune già applicabili? Viene già utilizzata una qualsiasi forma di
terapia genica per alcune patologie?
Grazie in anticipo
Luigi

 terapia genica per alcune patologie?
Per ora, non al di fuori di studi controllati.
Saluti,
Piersante Sestini


Ciao,
tempo fa avevo letto da qualche parte qualcosa riguardo a questo tipo di 
terapia e ADA.... eccoti trovata qualche referenza:
[Gene therapy for adenosine deaminase (ADA) deficiency: review of the 
past, the present and the future] Nippon Rinsho 2001 Jan;59(1):72-5 
(ISSN: 0047-1852)
ADA deficiency is the first disease being treated by gene therapy. Since 
the first trial of gene therapy performed ten years ago, more than 10 
patients including our case with ADA deficiency have been treated by the 
gene therapy with different clinical protocols. In contrast to the 
recent successful report for X-SCID patients, however, no curative 
effect of gene therapy for ADA deficiency has been achieved at the 
moment. In this chapter, I would like to review the past, the present 
and the future of gene therapy for ADA deficiency, and discuss an issue, 
especially PEG-ADA therapy, regarding the prospect for stem cell gene 
therapy for the disease.
Advances in gene therapy for ADA-deficient SCID.Curr Opin Mol Ther 2002 
Oct;4(5):515-22 (ISSN: 1464-8431)
Adenosine deaminase (ADA)-deficient severe combined immunodeficiency 
(SCID) was the first inherited disease treated with gene therapy. The 
pilot gene therapy studies demonstrated the safety, therapeutic 
potential and limitations of ADA gene transfer into hematopoietic cells 
using retroviral vectors. This review describes the latest progress in 
ADA-SCID dinical trials using peripheral blood lymphocytes (PBLs) and 
hematopoietic stem cells (HSCs). PBL gene therapy was able to restore 
T-cell functions after discontinuation of ADA enzyme replacement 
therapy, but only partially corrected the purine metabolic defect. The 
development of improved HSC gene transfer protocols, combined with low 
intensity conditioning, allowed full correction of the immunological and 
metabolic ADA defects, with clinic benefit. These results have important 
implications for future applications of gene therapy in other disorders 
involving the hemapoietic system.
Piu' di cosi' non ti posso aiutare (PS non sono medico, ma biologa), se 
non suggerirti ulteriori ricerche in PubMed o medline.... :-)
Ciao
Guya

 terapia genica per alcune patologie?
Ti segnalo questo review, eccone il link:
http://www.hosppract.com/genetics/9910mmc.htm
The Prospects for Gene Therapy 
STEPHEN L. ECK 
University of Pennsylvania 
Gene therapy will probably not be inordinately complex.  Although
extremely high-tech in its design and manufacture, it is likely to be
easy to administer in most practice settings. It will be widely used for
classic genetic and common diseases alike. 
Spero di esserti stato d'aiuto, fammi sapere
-- 
gigi
studente ignorantello 2o anno medicina