Sentences with phrase «gene therapy clinical trials»

He notes that AAV is already being used in gene therapy clinical trials for Parkinson's disease, hemophilia, and vision disorders.

Vectors designed and manufactured at St. Jude have already helped transform the lives of patients participating in gene therapy clinical trials for immune and blood disorders.

Several gene therapy clinical trials using gene - modified hematopoietic stem cells have shown therapeutic efficacy in multiple disease areas.

As a new generation of gene therapy clinical trials shows promise to cure or halt the progression of several rare diseases, the time has come to explore ways to pay for the cutting edge treatments, a pediatric hematologist - oncologist from Dana - Farber / Boston Children's Cancer and Blood Disorders Center argues in a commentary published by the journal Science.

There are now more than 100 gene therapy clinical trials aimed toward cancer, genetic diseases (such as ADA deficiency, cystic fibrosis and hemophilia A), infectious diseases (including AIDS) and autoimmune diseases (such as rheumatoid arthritis).

11/10/2008 UC San Diego Medical Center Offers Gene Therapy Clinical Trial for Advanced Heart Failure UC San Diego Medical Center is currently enrolling patients in a Phase 2 clinical trial of an investigational drug for the treatment of advanced heart failure.

Lysogene's MPS IIIA gene therapy clinical trial strategy includes a partnership with the MPS patient networks to ensure families are well informed about our development program and are able to make informed choices concerning the best treatment strategy for their children.

The data supports the development of gene therapy clinical trials for myotubular myopathy, the researchers concluded.

Many point to the Jesse Gelsinger case, in which a teenager died during a gene therapy clinical trial.

Colleen Dansereau, RN, MSN, CPN Program Manager, Gene Therapy Program As manager of the Gene Therapy Program, Colleen administratively manages the CAR T - cell therapy program as well as all administrative responsibility for all of the gene therapy clinical trials that take place at Dana - Farber / Boston Children's, for conditions including sickle cell disease, Wiskott - Aldrich Syndrome (WAS), and chronic granulomatous disease (CGD).

Reid and his colleagues undertook this study following the death of Jesse Gelsinger, a participant in a gene therapy clinical trial at University of Pennsylvania for ornithine transcarbanoylase (OTC) deficiency, a metabolic liver disorder.