BEGIN:VCALENDAR
VERSION:2.0
METHOD:PUBLISH
CALSCALE:GREGORIAN
PRODID:-//WordPress - MECv7.32.0//EN
X-ORIGINAL-URL:https://oligotherapeutics.org/
X-WR-CALNAME:Oligonucleotide Therapeutics Society
X-WR-CALDESC:Oligonucleotide Therapeutics Society
X-WR-TIMEZONE:UTC
BEGIN:VTIMEZONE
TZID:UTC
X-LIC-LOCATION:UTC
BEGIN:STANDARD
TZOFFSETFROM:+0000
TZOFFSETTO:+0000
TZNAME:UTC
DTSTART:20260514T093904
END:STANDARD
END:VTIMEZONE
REFRESH-INTERVAL;VALUE=DURATION:PT1H
X-PUBLISHED-TTL:PT1H
X-MS-OLK-FORCEINSPECTOROPEN:TRUE
BEGIN:VEVENT
CLASS:PUBLIC
UID:MEC-94ba20fc729f746b6318b7b6bf026045@oligotherapeutics.org
DTSTART;TZID=UTC:20240822T170000
DTEND;TZID=UTC:20240822T190000
DTSTAMP:20240618T024642Z
CREATED:20240618
LAST-MODIFIED:20240618
PRIORITY:5
SEQUENCE:0
TRANSP:OPAQUE
SUMMARY:2023 OTS Paper of the Year Spotlight: A Framework for Individualized Splice-Switching Oligonucleotide Therapy
DESCRIPTION:Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of individuals with genetic diseases, but the systematic identification of such individuals remains a challenge. We performed whole-genome sequencing on 235 individuals with ataxia-telangiectasia, a severe genetic disorder, and developed a predictive taxonomy to assess their suitability for ASO intervention, finding that 9% and 6% of the individuals had variants ‘probably’ or ‘possibly’ amenable to ASO splice modulation, respectively, with most located in deep intronic regions. We developed ASOs that successfully corrected mis-splicing and restored ATM cellular signaling in patient fibroblasts for two recurrent variants. In a pilot clinical study, one of these ASOs was used to treat a child with ataxia-telangiectasia, showing good tolerability without serious adverse events for over three years. Our study provides a framework for identifying individuals with genetic diseases who might benefit from splice-switching ASOs.\n
URL:http://oligotherapeutics.org/webinar-title-2023-ots-paper-of-the-year-spotlight-a-framework-for-individualized-splice-switching-oligonucleotide-therapy/
CATEGORIES:Webinar
ATTACH;FMTTYPE=image/webp:https://oligotherapeutics.org/wp-content/uploads/2024/06/08-22-20241.webp
END:VEVENT
END:VCALENDAR