retrovirus carrying a new version of the genome, in the artist's representation
In Europe, waiting to receive the certificate, gene therapy called Strimvelis. If approved, it would be the first therapy using gene editing for the treatment of deadly disease.
The disease, which treats therapy, called severe combined immunodeficiency (SCID, alymphocytosis, Glyantsmana-Rinikera syndrome). It is also known as "bubble boy syndrome" - people with this congenital defect virtually no immune system, it is extremely vulnerable to infectious diseases and have to be in a sterile environment.
One of the patients had known David Vetter, whose case was widely reported in the media in 1970. In 1976, based on his life was made an American television film "The Boy in the Plastic Bubble" (traditional translation of the name - "Under the hood") with John Travolta.
British Pharmacological Therapy holds giant GlaxoSmithKline, which bought it in 2010 at the Milan Institute of Gene Therapy. It has already been tested successfully in Italy for 18 sick children, and the first treatment took place 15 years ago. All of them are still alive. Total registered about 14 cases of birth of children with this disease in Europe per year.
In early April, the European Commission approved the pre-treatment. It is expected that the problems with the certification from GSK will not, and if so it will be able to sell this treatment in 27 European countries and in the United States to start the licensing process. The successful implementation of such a large company plans to open a new era in medicine.
"The idea that you no longer need to worry about anything and just be normal, leads people in excitement, - said Marcia Boyle [Marcia Boyle], founder and president of immunodeficiency, whose son is one of the 200 children living today with this severe disease. - I'm a little skeptical of gene therapy, as we have long awaited her appearance, but the fool mother nature was not so easy »
The traditional options for dealing with SCID is a bone marrow transplant (a complex and expensive operation, requiring the availability of suitable donors, and often resulting in death) or the use of an enzyme deputies, one ampoule which is worth $ 5,000.
The new treatment system by editing the genes, doctors removed stem cells from the patient's bone marrow, edit gene "in vitro", introducing a new copy in a retrovirus, which then delivers it to cells of the body.
According to the Alliance of regenerative medicine, genetic studies are booming, companies engaged in this activity in 2015 received a total of about $ 10 billion investment. Currently there are more than 5,000 different genetic diseases caused by a defect in a single gene only, and are in the development of several hundreds of options for their treatment by gene editing. 70 kinds of therapy are in the final stages of testing.