Sentences with phrase «gene therapy studies»

Such long - term expression is important for gene therapy studies in humans.

In most gene therapy studies, a «normal» gene is inserted into the genome to replace an «abnormal,» disease - causing gene.

This level of bone marrow stem cell correction has not been achieved in prior gene therapy studies in XSCID infants.

That's why a recent gene therapy study from a research group at the University of Pennsylvania has many people excited.

Field reports suggest that not all K13 mutations are capable of causing resistance, and the genetic system developed by Dr. Fidock to study K13, based on DNA repair approaches that are being used in human gene therapy studies, will be critical in identifying real hot spots of resistance.

Then he joined a gene therapy study conducted by molecular geneticist and physician Jean Bennett of the University of Pennsylvania School of Medicine and her husband, Albert Maguire, a retinal surgeon at the Children's Hospital of Philadelphia.

Wilson famously led a gene therapy study with an adenovirus vector that killed a patient, Jesse Gelsinger, in 1999 — a major setback for the entire field.

The Milto family raised money for a gene therapy study for Batten disease that included their sons Nathan (right) and PJ.