Ku Cristy A

Ku Cristy A, Igelman Austin D, Huang Samuel J, Bailey Steven T, Lauer Andreas K, Duncan Jacque L et al.

Voretigene neparvovec-rzyl caused perimacular atrophy in eyes previously treated with a different viral vector, despite visual gains. This highlights potential risks when using different gene therapy vectors in each eye.

Ku Cristy A, Igelman Austin D, Huang Samuel J, Vasconcelos Huber, da Palma Mariana Matioli, Bailey Steven T et al.

Gene therapy for RPE65 retinopathy significantly improved rod sensitivity across the retina, offering a sensitive method for monitoring treatment efficacy in future inherited retinal dystrophy trials.

Hull Sarah, Arno Gavin, Ku Cristy A, Ge Zhongqi, Waseem Naushin, Chandra Aman et al.

This study expanded Knobloch syndrome findings, revealing new features like pigment dispersion and glaucoma, cone-rod dysfunction, and normal neuroradiology in some, aiding early diagnosis and management.