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

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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Bibliographische Detailangaben
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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Zusammenfassung: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 subunit of collagen VIII are also disclosed for treatment of mutations therein that may contribute to FECD. L'invention concerne des compositions et des méthodes d'excision de répétitions trinucléotidiques (TNR) contenues dans l'intron 3 de TCF4, tels que celles qui sont observées chez des sujets présentant une dystrophie de l'endothélium cornéen de Fuchs (FECD), une PSC et la schizophrénie. L'invention concerne également des compositions comprenant des séquences de guidage ciblant la sous-unité alpha 2 du collagène VIII pour le traitement de mutations en leur sein qui peuvent contribuer à la FECD.