Sentences with phrase «based gene therapy trial»

(In the early 2000s, five children who participated in a retrovirus - based gene therapy trial for severe combined immunodeficiency developed leukemia.)

Not exact matches

He and his lab, for example, along with the company Neurologix based in Fort Lee, N.J., are conducting a clinical trial to test a similar gene - therapy treatment for Parkinson's disease.

AveXis, Inc., a clinical - stage gene therapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXStherapy company developing treatments for patients suffering from rare and life - threatening neurological genetic diseases, announced in July 2016 that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for the treatment based on preliminary clinical results from the trial of AVXSTherapy Designation for the treatment based on preliminary clinical results from the trial of AVXS - 101.

Boston Children's Hospital has offered non-exclusive licenses to for - profit entities on a patent developed by Orkin's laboratory regarding BCL11A, a genetic switch regulating hemoglobin production that is expected to form the basis of clinical trials for gene therapy and gene editing for sickle cell disease and thalassemia.

After preclinical studies, a gene therapy trial for SCID - X1 was initiated, based on the use of complementary DNA containing a defective γc Moloney retrovirus — derived vector and ex vivo infection of CD34 + cells.

It is the first time that a gene therapy based on genetically modified stem cells is tested in a multicenter, international clinical trial that shows a reproducible and robust therapeutic effect in different centers and different countries.

In the coming months, four US clinics will recruit people with HIV to a trial of a therapy based on gene editing.

The present research provides a theoretical backbone for gene introduction research previously conducted by trial - and - error, and is expected to contribute to the elucidation of the molecular basis of DNA medicines and gene therapy, and to make them more efficient.

The proposed gene therapy is based on the success of small human trials of killer T - cells (Chimeric Antigen Receptor [CAR] T - cell therapy).

Since the initial Michigan trial, the liposomal transport system — developed by researchers at Vical, a San Diego - based biopharmaceutical company interested in gene therapy — has been altered in ways that are expected to make it safer, more effective and easier to administer.

Doudna believes a clinical trial of CRISPR - based gene therapy could begin in less than a decade.