Gene dosage manipulation alleviates manifestations of hereditary PAX6 haploinsufficiency in mice-Reference-Cited by-同舟云学术

Gene dosage manipulation alleviates manifestations of hereditary PAX6 haploinsufficiency in mice

Published:2020-12-09 Issue:573 Volume:12 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Rabiee Behnam¹^ORCID,Anwar Khandaker N.¹^ORCID,Shen Xiang¹,Putra Ilham¹,Liu Mingna²,Jung Rebecca¹^ORCID,Afsharkhamseh Neda¹^ORCID,Rosenblatt Mark I.¹^ORCID,Fishman Gerald A.¹³,Liu Xiaorong²^ORCID,Ghassemi Mahmood¹,Djalilian Ali R.¹^ORCID

Affiliation:

1. Department of Ophthalmology and Visual Sciences, University of Illinois at Chicago, Chicago, IL 60612, USA.

2. Departments of Biology and Psychology, University of Virginia, Charlottesville, VA 22903, USA.

3. Pangere Center for Inherited Retinal Diseases, The Chicago Lighthouse, Chicago, IL 60608, USA.

Abstract

Pharmacological induction of wild-type PAX6 allele improved ocular abnormalities induced by PAX6 haploinsufficiency in mice.

Funder

National Institutes of Health

National Eye Institute

Research to Prevent Blindness

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