Salk technology at the heart of gene therapy success

November 6, 2009

Salk technology at the heart of gene therapy success

November 6, 2009

LA JOLLA, CA—Using a gene therapy delivery system developed in the laboratory of Inder Verma at the Salk Institute for Biological Studies, an international team of researchers successfully treated two children with adrenoleukodystrophy or ALD, in which the fatty insulation of nerve cells degenerates. The genetic disorder leads to progressive brain damage and results in death within two to five years after diagnosis.

Although the children have not been cured, their condition has been stable for the last two years, and they are cancer-free, according to Patrick Aubourg, the French researcher who led the study published in the Nov. 6, 2009 issue of the journal Science. Verma was not involved in the gene therapy trial.

(ALD is the rare, inherited fatal disease in children that was the focus of the 1993 Oscar-nominated film “Lorenzo’s Oil” starring Nick Nolte and Susan Sarandon.)

Verma, Ph.D., a professor in the Laboratory of Genetics and holder of the Irwin Mark Jacobs Chair in Exemplary Life Science pioneered the use of stripped-down versions of HIV, the virus that causes AIDS, to ferry intact versions of genes that are defective or missing to cells throughout the body.

Prior to this innovation, viruses used as gene delivery vehicles could only infect actively dividing cells, which left out the vast majority of cells in the body, drastically limiting its utility. Verma’s modified HIV virus, however, a member of the lentivirus family, is capable of infecting non-dividing cells and delivering genes efficiently into a wide variety of cells.

The new gene therapy vector not only solved the efficiency problem but also appears to positively impact the safety concerns that had dashed gene therapy hopes years earlier when the only human gene therapy treatment that had been considered to be largely successful, turned out be riskier than realized. Out of 20 children treated for X-linked severe combined immune deficiency (X-SCID) – commonly known as the “bubble boy” syndrome – 18 were cured but three developed leukemia and one died of it.

About eight years ago Verma was first approached by Aubourg and the Salzman sisters, who had been pushing scientists and doctors to design a gene therapy study after they had learned that three of their children suffered from ALD, to consider making his lentiviral vector available.

Eventually, the virus was produced by CellGenesys, who had licensed the gene therapy vector technology from the Salk Institute, and Aubourg successfully treated two boys from Spain.

关于索尔克生物研究所：
索尔克生物学研究所是世界顶尖的基础研究机构之一，其国际知名的教职员工在一个独特的、协作和富有创造性的环境中，致力于探索生命科学的基本问题。索尔克科学家们既专注于科学发现，也致力于培养下一代研究人员，通过研究神经科学、遗传学、细胞生物学和植物生物学及相关学科，为我们理解癌症、衰老、阿尔茨海默病、糖尿病和心血管疾病做出了开创性贡献。.

学院取得了许多成就，获得了包括诺贝尔奖和美国国家科学院院士在内的无数荣誉。该研究所由脊髓灰质炎疫苗先驱 Jonas Salk 博士于 1960 年创立，是一家独立的非营利组织和建筑地标。.