Sentences with phrase «gene therapy clinical trials shows»

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.

Not exact matches

«My team at Nationwide Children's has worked with commitment and dedication to develop a therapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide Chiltherapy that may subsequently be shown through future clinical trials to potentially alter the course of this unforgiving condition and provide a therapeutic option for the families and infants with SMA1,» says Jerry Mendell, MD, principal investigator in the Center for Gene Therapy at Nationwide ChilTherapy at Nationwide Children's.

It showed effectiveness against liver cancer in a phase II clinical trial and will move into a phase III trial later this year, David Kirn, an oncologist and the company's president and chief executive officer, said at a recent meeting of the American Society for Gene & Cell Therapy in Washington, D.C..

Despite expectations of a rapid breakthrough, no cystic fibrosis gene therapy trial so far has been able to show long - term clinical improvement.

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.

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

Within the past 10 years or so, lentiviruses have shown promise in clinical trials (1 — 3), and adenoassociated viruses (AAVs) have been used in the first approved gene therapies in the Western world (4).