Researchers suggest an application to FDA may be made within the next 12 months, requesting approval to carry out a human clinical trial with the gene therapy.
Can you stop LCA, a form of blindness caused by a change in a single base pair, simply by injecting a viral vector with the ... and doctors planning human gene therapy trials for LCA.
New data publishing 11/11 in Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial ... genetic mutations with a single vector. This clinical-grade vector ...