Diseases, Conditions, Syndromes

Curing 'bubble baby' disease—from the lab to the clinic

For the last several decades, scientists worldwide have been seeking to harness the power of stem cells to develop therapies for human diseases and conditions. At UCLA's Broad Stem Cell Research Center, the potential to bring ...

Oncology & Cancer

Science commentary explores ways to pay for success in gene therapy

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 ...

Ophthalmology

Gene therapy shows long-term benefit for treating rare blindness

Pioneering gene therapy has restored some vision to patients with a rare form of genetic blindness for as long as four years, raising hopes it could be used to cure common causes of vision loss, new University of Oxford research ...

Ophthalmology

Team develops novel gene therapy for achromatopsia

Researchers from the University of Pennsylvania and Michigan State University presented new preclinical data this week that evaluates the efficacy of a gene therapy treatment for achromatopsia, a rare inherited retinal disease ...

Medical research

Gene therapy trial for 'Bubble boy' disease promising

(Medical Xpress)—Researchers reported promising outcomes data for the first group of boys with X-linked severe combined immunodeficiency syndrome (SCID-X1), a fatal genetic immunodeficiency also known as "bubble boy" disease, ...

page 8 from 10