Safety and Efficacy of AAV2tYF-PR1.7-CNGA3 in CNGA3-deficient Sheep

Mark S. Shearman; Elisha Gootwine; Eyal Banin; Ron Ofri; Raaya Ezra-Elia; Guo-jie Ye; Paulette Robinson; David Knop; Jeffrey D. Chulay

Safety and Efficacy of AAV2tYF-PR1.7-CNGA3 in CNGA3-deficient Sheep

Mark S. Shearman
, Elisha Gootwine
, Eyal Banin
, Ron Ofri
, Raaya Ezra-Elia
, Guo-jie Ye
, Paulette Robinson
, David Knop
, Jeffrey D. Chulay

The Koret School of Veterinary Medicine

Research output: Contribution to journal › Meeting Abstract › peer-review

Abstract

This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.
Purpose : Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity and absence of color discrimination. AGTC is developing AAV2tYF-PR1.7-CNGA3, a recombinant adeno-associated virus (rAAV) vector expressing CNGA3, for treatment of CNGA3-related achromatopsia. Here we report results of a toxicology and efficacy study of this vector administered by subretinal injection in CNGA3-deficient sheep.

Original language	English
Journal	Investigative Ophthalmology and Visual Science
Volume	58
Issue number	8
State	Published - Jun 2017

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title = "Safety and Efficacy of AAV2tYF-PR1.7-CNGA3 in CNGA3-deficient Sheep",

abstract = "This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017. Purpose : Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity and absence of color discrimination. AGTC is developing AAV2tYF-PR1.7-CNGA3, a recombinant adeno-associated virus (rAAV) vector expressing CNGA3, for treatment of CNGA3-related achromatopsia. Here we report results of a toxicology and efficacy study of this vector administered by subretinal injection in CNGA3-deficient sheep.",

author = "Shearman, \{Mark S.\} and Elisha Gootwine and Eyal Banin and Ron Ofri and Raaya Ezra-Elia and Guo-jie Ye and Paulette Robinson and David Knop and Chulay, \{Jeffrey D.\}",

year = "2017",

month = jun,

language = "אנגלית",

volume = "58",

journal = "Investigative Ophthalmology and Visual Science",

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T1 - Safety and Efficacy of AAV2tYF-PR1.7-CNGA3 in CNGA3-deficient Sheep

AU - Shearman, Mark S.

AU - Gootwine, Elisha

AU - Banin, Eyal

AU - Ofri, Ron

AU - Ezra-Elia, Raaya

AU - Ye, Guo-jie

AU - Robinson, Paulette

AU - Knop, David

AU - Chulay, Jeffrey D.

PY - 2017/6

Y1 - 2017/6

N2 - This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017. Purpose : Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity and absence of color discrimination. AGTC is developing AAV2tYF-PR1.7-CNGA3, a recombinant adeno-associated virus (rAAV) vector expressing CNGA3, for treatment of CNGA3-related achromatopsia. Here we report results of a toxicology and efficacy study of this vector administered by subretinal injection in CNGA3-deficient sheep.

AB - This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017. Purpose : Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity and absence of color discrimination. AGTC is developing AAV2tYF-PR1.7-CNGA3, a recombinant adeno-associated virus (rAAV) vector expressing CNGA3, for treatment of CNGA3-related achromatopsia. Here we report results of a toxicology and efficacy study of this vector administered by subretinal injection in CNGA3-deficient sheep.

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SN - 0146-0404

VL - 58

JO - Investigative Ophthalmology and Visual Science

JF - Investigative Ophthalmology and Visual Science

IS - 8

ER -

Safety and Efficacy of AAV2tYF-PR1.7-CNGA3 in CNGA3-deficient Sheep

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