Laura Cyckowski

TL;DR: The researchers report that the lack of immune response and the robust safety profile in this readministration gene therapy study may be due in part to the immune-privileged nature of the eye, and the low dose and very pure preparation of the AAV vector.

TL;DR: To the knowledge, AAV2-hRPE65v2 is the first successful gene therapy administered to the contralateral eye and the results highlight the use of several outcome measures and help to delineate the variables that contribute to maximal benefit from gene augmentation therapy in this disease.

TL;DR: Compared hippocampal morphology in 14 "treatment-seeking" adolescents with a history of prior heavy cannabis use with an average of 6.7 month of supervised abstinence lends support to a theory that cannabis use may impart long-term structural and functional damage.

TL;DR: Functional MRI data suggest that despite severe and long-term visual impairment, treated LCA2 patients have intact and responsive visual pathways, and suggest that gene therapy resulted in not only sustained and improved visual ability, but also enhanced contrast sensitivity.

TL;DR: It is demonstrated that reversing blindness with gene therapy promoted long-term structural plasticity in the visual pathways emanating from the treated retina of LCA patients, and improvements and normalization along the visual fibers corresponding to the site of retinal injection of the gene therapy vector carrying the therapeutic gene in the treated eye compared to the visual pathway for the untreated eye of L CA patients.