Gene Therapy Advance In Beta-Thalassemia

[solankyno1]

Gene therapy’s roller coaster ride of euphoria and setback is on the upside. A new viral vector treatment for the anemia called beta-thalassemia works in vitro, and shows no sign of toxicity, according to a paper published in EMBO Molecular Medicine.

The results “will pave the way forward” to clinically apply the treatment with the vector, called GLOBE LV, the researchers stated in the paper.

The team of Italian scientists used the therapy on human hematopoietic stem cells unable to produce beta-globin, the cause of beta-thalassemia.The disease interferes with hemoglobin production, and can be fatal if the patient doesn’t receive treatment. The viral vector, produced from HIV-1, contains genetic material correcting the defect. Once these treated stem cells developed into red blood cells, they were found to contain sufficient hemoglobin.

While other attempts at gene therapy for the disease have been previously made, the researchers say the results didn’t have enough efficiency to succeed

Thalassimia comes in two main types, alpha- and -beta varieties. It causes the production of abnormal hemoglobin, which not only reduces oxygen-carrying capacity, but can cause damage to internal organs.

Thalassemia is prevalent in people of Mediterranean origin, and some other populations. It’s a big problem in Oman.

Gene therapy has followed the traditional path of biotech treatments: enthusiasm to excess in the beginning, disillusionment and despair after setbacks, and renewed interest as incremental progress adds up. For example, an improved version of the gene therapy treatment for “bubble-boy syndrome” is now being tested. A few years ago, treatment for the disease was halted after some of those treated developed leukemia. The improved version is thought to have removed the leukemia-causing effect.

Here’s a blog post of mine from early 2009 discussing the revival of interest in gene therapy.

Source : Gene Therapy Advance In Beta-Thalassemia NCTimes.com Blogs

[Jyoti]

sounds good, hope it will become even better sounding soon. Shall I say, best of luck to all of us?