Safety and biodistribution evaluation in cynomolgus macaques of rAAV2tYF-PR1. 7-hCNGB3, a recombinant AAV vector for treatment of achromatopsia
G Ye, E Budzynski, P Sonnentag, TM Nork… - Human gene therapy …, 2016 - liebertpub.com
Applied Genetic Technologies Corporation (AGTC) is developing rAAV2tYF-PR1. 7-
hCNGB3, a recombinant adeno-associated viral (rAAV) vector expressing the human …
hCNGB3, a recombinant adeno-associated viral (rAAV) vector expressing the human …
Safety and biodistribution evaluation in CNGB3-deficient mice of rAAV2tYF-PR1. 7-hCNGB3, a recombinant AAV vector for treatment of achromatopsia
G Ye, E Budzynski, P Sonnentag, TM Nork… - Human Gene …, 2016 - liebertpub.com
Applied Genetic Technologies Corporation (AGTC) is developing rAAV2tYF-PR1. 7-
hCNGB3, a recombinant adeno-associated virus (rAAV) vector expressing the human …
hCNGB3, a recombinant adeno-associated virus (rAAV) vector expressing the human …
Safety and efficacy evaluation of rAAV2tYF-PR1. 7-hCNGA3 vector delivered by subretinal injection in CNGA3 mutant achromatopsia sheep
E Gootwine, R Ofri, E Banin, A Obolensky… - Human gene therapy …, 2017 - liebertpub.com
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-
associated virus (rAAV) vector expressing the human CNGA3 gene designated AGTC-402 …
associated virus (rAAV) vector expressing the human CNGA3 gene designated AGTC-402 …
Safety and toxicology of ocular gene therapy with recombinant AAV vector rAAV. hCNGA3 in nonhuman primates
P Tobias, SI Philipp, M Stylianos, B Martin… - Human Gene …, 2019 - liebertpub.com
The purpose of this study was to examine the toxicity and side effects of a recombinant
adeno-associated virus 8 (AAV8) vector, aimed to treat cyclic nucleotide gated channel …
adeno-associated virus 8 (AAV8) vector, aimed to treat cyclic nucleotide gated channel …
Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-Mutant Dogs
G Ye, AM Komaromy, C Zeiss, R Calcedo… - Human Gene …, 2017 - liebertpub.com
Achromatopsia is an inherited retinal disorder of cone photoreceptors characterized by
markedly reduced visual acuity, extreme light sensitivity, and absence of color …
markedly reduced visual acuity, extreme light sensitivity, and absence of color …
[HTML][HTML] First-in-human gene therapy trial of AAV8-hCARp. hCNGB3 in adults and children with CNGB3-associated achromatopsia
M Michaelides, N Hirji, SC Wong, CG Besirli… - American Journal of …, 2023 - Elsevier
Purpose To assess the safety and efficacy of AAV8-hCARp. hCNGB3 in participants with
CNGB3-associated achromatopsia (ACHM). Design Prospective, phase 1/2 …
CNGB3-associated achromatopsia (ACHM). Design Prospective, phase 1/2 …
Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia …
NA Kahle, T Peters, D Zobor, L Kuehlewein… - Human Gene …, 2018 - liebertpub.com
Achromatopsia is an autosomal recessively inherited congenital defect characterized by a
lack of cone photoreceptor function, leading to severely impaired vision. In this clinical study …
lack of cone photoreceptor function, leading to severely impaired vision. In this clinical study …
AAV8 can induce innate and adaptive immune response in the primate eye
FF Reichel, DL Dauletbekov, R Klein, T Peters… - Molecular Therapy, 2017 - cell.com
Ocular gene therapy has evolved rapidly into the clinical realm due to promising pre-clinical
proof-of-concept studies, recognition of the high unmet medical need of blinding disorders …
proof-of-concept studies, recognition of the high unmet medical need of blinding disorders …
Novel AAV capsids for intravitreal gene therapy of photoreceptor disorders
M Pavlou, C Schön, LM Occelli, A Rossi… - EMBO molecular …, 2021 - embopress.org
Gene therapy using recombinant adeno‐associated virus (rAAV) vectors to treat blinding
retinal dystrophies has become clinical reality. Therapeutically impactful targeting of …
retinal dystrophies has become clinical reality. Therapeutically impactful targeting of …
Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 …
Achromatopsia causes severely reduced visual acuity, photoaversion, and inability to
discern colors due to cone photoreceptor dysfunction. In 2010, we reported on day …
discern colors due to cone photoreceptor dysfunction. In 2010, we reported on day …