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פותח על ידי קלירמאש פתרונות בע"מ -
From sheep breeding to ophthalmological bio-medical research: Day-blind sheep as an animal model for restoration of visual function using gene therapy
Year:
2020
Source of publication :
וולקני וויס
Authors :
גוטויין, אלישע
;
.
Volume :
8
Co-Authors:
Facilitators :
From page:
5
To page:
7
(
Total pages:
3
)
Abstract:

Achromatopsia is a hereditary form of day blindness, caused by cone photoreceptor dysfunction. A sheep model of this disease was characterized by us, and has been utilized successfully to test the safety and efficacy of achromatopsia gene therapy. The rescue effect has, so far, been maintained for over 8 years. Following FDA approval, clinical trials of CNGA3 achromatopsia gene therapy in patients were initiated last year.

Note:
Related Files :
Achromatopsia
biomedicine
Day blindness
Eye diseases
gene therapy
major genes
Prolificacy
עוד תגיות
תוכן קשור
More details
DOI :
Article number:
0
Affiliations:
Database:
Publication Type:
מאמר
;
.
Language:
אנגלית
Editors' remarks:
ID:
55561
Last updated date:
02/03/2022 17:27
Creation date:
13/07/2021 08:30
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From sheep breeding to ophthalmological bio-medical research: Day-blind sheep as an animal model for restoration of visual function using gene therapy
8
From sheep breeding to ophthalmological bio-medical research: Day-blind sheep as an animal model for restoration of visual function using gene therapy

Achromatopsia is a hereditary form of day blindness, caused by cone photoreceptor dysfunction. A sheep model of this disease was characterized by us, and has been utilized successfully to test the safety and efficacy of achromatopsia gene therapy. The rescue effect has, so far, been maintained for over 8 years. Following FDA approval, clinical trials of CNGA3 achromatopsia gene therapy in patients were initiated last year.

Scientific Publication
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