Sentences with phrase «immune gene therapy trials»

Not exact matches

Scientists believe that is what happened during a 1999 French gene therapy trial on a group of 10 young children with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.

After this incident, and the death in 1999 of a young man in a clinical trial, apparently because of an immune reaction to the treatment, gene therapy research grounds nearly to a halt.

The proposed clinical trial, in which researchers would use CRISPR to engineer immune cells to fight cancer, won approval from the Recombinant DNA Advisory Committee (RAC) at the U.S. National Institutes of Health, a panel that has traditionally vetted the safety and ethics of gene therapy trials funded by the U.S. government and others.

Scientists believe that's what happened during a 1999 French gene therapy trial on a group of 10 infants with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.

The LCA2 trials are a rare success for the field of gene therapy, which has also cured children with the immune disorder known as bubble boy disease.

As for worries about harmful effects, researchers say AAV8 isn't likely to disrupt healthy genes, a side effect that stalled a promising gene - therapy trial for an immune - deficiency disorder in 2002.

In the current trial, two patients experienced an immune response to the gene therapy that did not result in safety concerns, and were treated with steroids.

The results drew attention in part because they come from the lab of James Wilson at the University of Pennsylvania, who led a 1999 trial in which a teenager died from an immune reaction to a different gene therapy vector.

Previous hemophilia gene therapy trials have been frustrated by an immune response to the gene therapy product that limited the success of the therapy.

They currently have a trial under way at the U-M Health System which tests a two - part gene therapy approach in patients with brain tumors called gliomas in an effort to get the immune system to attack the tumor.

These are skills that we implement for other international trials of gene therapy for rare genetic diseases of the immune system, blood, muscle, vision or liver... We will continue the current study with the objective of providing treatment for patients.»

Other blood disorders that have shown significant benefit from targeted gene therapy in small trials include hemophilia (specifically, factor IX deficiency), severe beta - thalassemia (deficiency for the adult beta - globin gene) and leukemia, where the patient's immune cells were treated to enable them to recognize cancer cells and destroy them.

Researchers reported promising preliminary outcomes for the first four children enrolled in a U.S. gene therapy trial for Wiskott - Aldrich syndrome (WAS), a life - threatening genetic blood and immune disorder, at the 57th annual meeting of the American Society of Hematology.

The researchers used the new technique to mutate the genes CXCR4 and CCR5, which encode receptor molecules that different strains of the HIV virus use to sneak in and infect immune cells and which have been targeted in previous cell therapy trials.