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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| creator | BURNS, Sean Michael HESSE, Sarah Beth MURRAY, Bradley Andrew |
| description | 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. |
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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.</description><language>eng ; fre</language><subject>HUMAN NECESSITIES ; HYGIENE ; MEDICAL OR VETERINARY SCIENCE ; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><creationdate>2017</creationdate><oa>free_for_read</oa><woscitedreferencessubscribed>false</woscitedreferencessubscribed></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktohtml>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20171026&DB=EPODOC&CC=WO&NR=2017185054A1$$EHTML$$P50$$Gepo$$Hfree_for_read</linktohtml><link.rule.ids>230,308,776,881,25542,76516</link.rule.ids><linktorsrc>$$Uhttps://worldwide.espacenet.com/publicationDetails/biblio?FT=D&date=20171026&DB=EPODOC&CC=WO&NR=2017185054A1$$EView_record_in_European_Patent_Office$$FView_record_in_$$GEuropean_Patent_Office$$Hfree_for_read</linktorsrc></links><search><creatorcontrib>BURNS, Sean Michael</creatorcontrib><creatorcontrib>HESSE, Sarah Beth</creatorcontrib><creatorcontrib>MURRAY, Bradley Andrew</creatorcontrib><title>COMPOSITIONS AND METHODS FOR TREATMENT OF DISEASES ASSOCIATED WITH TRINUCLEOTIDE REPEATS IN TRANSCRIPTION FACTOR FOUR</title><description>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.</description><subject>HUMAN NECESSITIES</subject><subject>HYGIENE</subject><subject>MEDICAL OR VETERINARY SCIENCE</subject><subject>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</subject><fulltext>true</fulltext><rsrctype>patent</rsrctype><creationdate>2017</creationdate><recordtype>patent</recordtype><sourceid>EVB</sourceid><recordid>eNqNjDEKwkAQRdNYiHqHAWshUYO2y-4sGTA7YWfEMgRZK9FA9P6u4AGsfvHfe_PibbntWEiJg4AJDlrUhp2A5wga0WiLQYE9OBI0gpkSYUtG0cGFtMkUhbM9ISs5hIhdlgQo5MMEsZG6bx28sZqbns9xWcxuw31Kq98uirVHtc0mjc8-TeNwTY_06i-8LatDdazLem-q3X_UByUEOqo</recordid><startdate>20171026</startdate><enddate>20171026</enddate><creator>BURNS, Sean Michael</creator><creator>HESSE, Sarah Beth</creator><creator>MURRAY, Bradley Andrew</creator><scope>EVB</scope></search><sort><creationdate>20171026</creationdate><title>COMPOSITIONS AND METHODS FOR TREATMENT OF DISEASES ASSOCIATED WITH TRINUCLEOTIDE REPEATS IN TRANSCRIPTION FACTOR FOUR</title><author>BURNS, Sean Michael ; HESSE, Sarah Beth ; MURRAY, Bradley Andrew</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-epo_espacenet_WO2017185054A13</frbrgroupid><rsrctype>patents</rsrctype><prefilter>patents</prefilter><language>eng ; fre</language><creationdate>2017</creationdate><topic>HUMAN NECESSITIES</topic><topic>HYGIENE</topic><topic>MEDICAL OR VETERINARY SCIENCE</topic><topic>PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES</topic><toplevel>online_resources</toplevel><creatorcontrib>BURNS, Sean Michael</creatorcontrib><creatorcontrib>HESSE, Sarah Beth</creatorcontrib><creatorcontrib>MURRAY, Bradley Andrew</creatorcontrib><collection>esp@cenet</collection></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext_linktorsrc</fulltext></delivery><addata><au>BURNS, Sean Michael</au><au>HESSE, Sarah Beth</au><au>MURRAY, Bradley Andrew</au><format>patent</format><genre>patent</genre><ristype>GEN</ristype><title>COMPOSITIONS AND METHODS FOR TREATMENT OF DISEASES ASSOCIATED WITH TRINUCLEOTIDE REPEATS IN TRANSCRIPTION FACTOR FOUR</title><date>2017-10-26</date><risdate>2017</risdate><abstract>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.</abstract><oa>free_for_read</oa></addata></record> |
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| title | COMPOSITIONS AND METHODS FOR TREATMENT OF DISEASES ASSOCIATED WITH TRINUCLEOTIDE REPEATS IN TRANSCRIPTION FACTOR FOUR |
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