AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

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  AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

  By Jean Bennett, Manzar Ashtari, Jennifer Wellman, Kathleen A. Marshall, Laura L. Cyckowski, Daniel C. Chung, Sarah McCague, Eric A. Pierce, Yifeng Chen, Jeannette L. Bennicelli, Xiaosong Zhu, Gui-shuang Ying, Junwei Sun, J. Fraser Wright, Alberto Auricchio, Francesca Simonelli, Kenneth S. Shindler, Federico Mingozzi, Katherine A. High, Albert M. Maguire

  Science Translational Medicine08 Feb 2012 : 120ra15

  Repeat administration of gene therapy to the contralateral retina of three congenitally blind patients was safe and resulted in improved vision.

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  AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

  By Jean Bennett, Manzar Ashtari, Jennifer Wellman, Kathleen A. Marshall, Laura L. Cyckowski, Daniel C. Chung, Sarah McCague, Eric A. Pierce, Yifeng Chen, Jeannette L. Bennicelli, Xiaosong Zhu, Gui-shuang Ying, Junwei Sun, J. Fraser Wright, Alberto Auricchio, Francesca Simonelli, Kenneth S. Shindler, Federico Mingozzi, Katherine A. High, Albert M. Maguire

  Science Translational Medicine08 Feb 2012 : 120ra15

  Repeat administration of gene therapy to the contralateral retina of three congenitally blind patients was safe and resulted in improved vision.

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