News Column

Studies from G. Farinelli et al Further Understanding of Gene Therapy (Lentiviral vectors for the treatment of primary immunodeficiencies)

August 21, 2014

By a News Reporter-Staff News Editor at Gene Therapy Weekly -- Investigators discuss new findings in Biotechnology. According to news reporting originating from Milan, Italy, by NewsRx correspondents, research stated, "In the last years important progress has been made in the treatment of several primary immunodeficiency disorders (PIDs) with gene therapy. Hematopoietic stem cell (HSC) gene therapy indeed represents a valid alternative to conventional transplantation when a compatible donor is not available and recent success confirmed the great potential of this approach."

Our news editors obtained a quote from the research, "First clinical trials performed with gamma retroviral vectors were promising and guaranteed clinical benefits to the patients. On the other hand, the outcome of severe adverse events as the development of hematological abnormalities highlighted the necessity to develop a safer platform to deliver the therapeutic gene. Self-inactivating (SIN) lentiviral vectors (LVVs) were studied to overcome this hurdle through their preferable integration pattern into the host genome."

According to the news editors, the research concluded: "In this review, we describe the recent advancements achieved both in vitro and at preclinical level with LVVs for the treatment of Wiskott-Aldrich syndrome (WAS), chronic granulomatous disease (CGD), ADA deficiency (ADA-SCID), Artemis deficiency, RAG1/2 deficiency, X-linked severe combined immunodeficiency (gamma chain deficiency, SCIDX1), X-linked lymphoproliferative disease (XLP) and immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome."

For more information on this research see: Lentiviral vectors for the treatment of primary immunodeficiencies. Journal of Inherited Metabolic Disease, 2014;37(4):525-533. Journal of Inherited Metabolic Disease can be contacted at: Springer, Van Godewijckstraat 30, 3311 Gz Dordrecht, Netherlands. (Springer -; Journal of Inherited Metabolic Disease -

The news editors report that additional information may be obtained by contacting G. Farinelli, Ist Sci San Raffaele, HSR TIGET, I-20132 Milan, Italy. Additional authors for this research include V. Capo, S. Scaramuzza and A. Aiuti (see also Biotechnology).

Keywords for this news article include: Biotechnology, Milan, Italy, Europe, Immunology, Gene Therapy, Bioengineering, Immunodeficiency

Our reports deliver fact-based news of research and discoveries from around the world. Copyright 2014, NewsRx LLC

For more stories covering the world of technology, please see HispanicBusiness' Tech Channel

Source: Gene Therapy Weekly

Story Tools Facebook Linkedin Twitter RSS Feed Email Alerts & Newsletters