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Novartis Foundation Symposium|January 31, 2004
Range of retinal diseases potentially treatable by AAV-vectored gene therapyWilliam W Hauswirth, Quihong Li, Brian Raisler, et al.Science (New York, N.Y.)|December 13, 2003
A muscleblind knockout model for myotonic dystrophyRahul N Kanadia, Karen A Johnstone, Ami Mankodi, et al.Human Gene Therapy|September 24, 2019
Ocular Inflammatory Response to Intravitreal Injection of Adeno-Associated Virus Vector: Relative Contribution of Genome and CapsidAdrian M Timmers, Judith A Newmark, Heikki T Turunen, et al.Nature Medicine|May 23, 2007
Restoration of cone vision in a mouse model of achromatopsiaJohn J Alexander, Yumiko Umino, Drew Everhart, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 20, 2005
Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisisSeok H Min, Laurie L Molday, Mathias W Seeliger, et al.Human Gene Therapy|May 17, 2020
Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene TherapyChunjuan Song, Valérie L Dufour, Artur V Cideciyan, et al.Pageof 2