[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 …
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 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 …
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 vision outcomes of subretinal gene therapy targeting cone photoreceptors in achromatopsia: a nonrandomized controlled trial
MD Fischer, S Michalakis, B Wilhelm… - JAMA …, 2020 - jamanetwork.com
Importance Achromatopsia linked to variations in theCNGA3gene is associated with day
blindness, poor visual acuity, photophobia, and involuntary eye movements owing to lack of …
blindness, poor visual acuity, photophobia, and involuntary eye movements owing to lack of …
Successful delivery of rAAV8. CNGA3 in a patient with CNGA3 achromatopsia
MD Fischer, B Wilhelm, S Michalakis… - … & Visual Science, 2016 - iovs.arvojournals.org
Purpose: The aim of this clinical interventional study (NCT02610582) was to test safety
aspects of AAV8 based supplementation gene therapy in patients with CNGA3 …
aspects of AAV8 based supplementation gene therapy in patients with CNGA3 …
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 …
[HTML][HTML] Characterization of retinal structure in ATF6-associated achromatopsia
RR Mastey, M Georgiou, CS Langlo… - … & visual science, 2019 - tvst.arvojournals.org
Purpose: Mutations in six genes have been associated with achromatopsia (ACHM):
CNGA3, CNGB3, PDE6H, PDE6C, GNAT2, and ATF6. ATF6 is the most recent gene to be …
CNGA3, CNGB3, PDE6H, PDE6C, GNAT2, and ATF6. ATF6 is the most recent gene to be …
Three-year results of phase I retinal gene therapy trial for CNGA3-mutated achromatopsia: results of a non randomised controlled trial
FF Reichel, S Michalakis, B Wilhelm, D Zobor… - British Journal of …, 2022 - bjo.bmj.com
Aims To determine long-term safety and efficacy outcomes of a subretinal gene therapy for
CNGA3-associated achromatopsia. We present data from an open-label, nonrandomised …
CNGA3-associated achromatopsia. We present data from an open-label, nonrandomised …
[HTML][HTML] The clinical phenotype of CNGA3-related achromatopsia: pretreatment characterization in preparation of a gene replacement therapy trial
D Zobor, A Werner, F Stanzial… - … & Visual Science, 2017 - tvst.arvojournals.org
Purpose: The purpose of this study was to clinically characterize patients with CNGA3-linked
achromatopsia (CNGA3-ACHM) in preparation of a gene therapy trial. Methods: Thirty-six …
achromatopsia (CNGA3-ACHM) in preparation of a gene therapy trial. Methods: Thirty-six …