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Study: Specific class of drugs can be repurposed to treat genetic diseases


Study: Specific class of drugs can be repurposed to treat genetic diseases
Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases.
A team from the UIC Department of Ophthalmology and Visual Sciences published the article, Gene dosage manipulation alleviates manifestations of hereditary PAX6 haploinsufficiency in mice in the journal
Science Translational Medicine.
Nearly all the genes in human DNA have two copies, one inherited from the mother and one from the father. There are some genetic diseases where only one copy is normal and the other one is non-functional due to a mistake in the DNA. The idea behind this study was to see if the normal copy can be enhanced to make up for the non-functional copy, said Ali Djalilian, UIC professor of ophthalmology and corresponding author of the paper. ....

United States , Mark Rosenblatt , Ali Djalilian , Emily Henderson , University Of Illinois Chicago , College Of Medicine , Department Of Ophthalmology , Corneal Regenerative Medicine Laboratory , Illinois Chicago , Visual Sciences , Science Translational , Study Corresponding Author , ஒன்றுபட்டது மாநிலங்களில் , குறி ரோசன்ப்ளாட் , எமிலி ஹென்டர்சன் , பல்கலைக்கழகம் ஆஃப் இல்லினாய்ஸ் சிகாகோ , கல்லூரி ஆஃப் மருந்து , துறை ஆஃப் கண் மருத்துவம் , கார்னியல் மீளுருவாக்கம் மருந்து ஆய்வகம் , இல்லினாய்ஸ் சிகாகோ , காட்சி அறிவியல் , அறிவியல் மொழிபெயர்ப்பு ,

Study in mice shows genes may be altered through drug repurposing


Study in mice shows genes may be altered through drug repurposing
Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases.
A team from the UIC Department of Ophthalmology and Visual Sciences published the article, Gene dosage manipulation alleviates manifestations of hereditary PAX6 haploinsufficiency in mice in the journal Science Translational Medicine.
Nearly all the genes in human DNA have two copies, one inherited from the mother and one from the father. There are some genetic diseases where only one copy is normal and the other one is non-functional due to a mistake in the DNA. The idea behind this study was to see if the normal copy can be enhanced to make up for the non-functional copy, said Ali Djalilian, UIC professor of ophthalmology and corresponding author of the paper. ....

United States , Gerald Fishman , Mark Rosenblatt , Khandaker Anwar , Behnam Rabiee , Rebecca Jung , Neda Afsharkhamseh , Mingna Liu , Mahmood Ghassemi , Ali Djalilian , Xiaorong Liu , Xiang Shen , University Of Illinois Chicago , Chicago Lighthouse Pangere Center , Research To Prevent Blindness , National Eye Institute , University Of Virginia , Department Of Ophthalmology , College Of Medicine , Corneal Regenerative Medicine Laboratory , Illinois Chicago , Visual Sciences , Science Translational , Ilham Putra , Inherited Retinal , Prevent Blindness ,