COMPOSITIONS AND METHODS FOR TREATMENT OF DISEASES ASSOCIATED WITH TRINUCLEOTIDE REPEATS IN TRANSCRIPTION FACTOR FOUR

This application relates to compositions and methods for excising trinucleotide repeats (TNRs) contained within intron 3 of TCF4, such as is seen in subjects having Fuchs endothelial corneal dystrophy (FECD), PSC, and Schizophrenia. Compositions comprising guide sequences targeting the alpha 2 subun...

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Hauptverfasser:	HESSE, SARAH BETH, MURRAY, BRADLEY ANDREW, BURNS, SEAN MICHAEL
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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