BEGIN:VCALENDAR
VERSION:2.0
PRODID:-//Center for RNA Biomedicine - ECPv6.15.17//NONSGML v1.0//EN
CALSCALE:GREGORIAN
METHOD:PUBLISH
X-WR-CALNAME:Center for RNA Biomedicine
X-ORIGINAL-URL:https://rna.umich.edu
X-WR-CALDESC:Events for Center for RNA Biomedicine
REFRESH-INTERVAL;VALUE=DURATION:PT1H
X-Robots-Tag:noindex
X-PUBLISHED-TTL:PT1H
BEGIN:VTIMEZONE
TZID:America/Detroit
BEGIN:DAYLIGHT
TZOFFSETFROM:-0500
TZOFFSETTO:-0400
TZNAME:EDT
DTSTART:20190310T070000
END:DAYLIGHT
BEGIN:STANDARD
TZOFFSETFROM:-0400
TZOFFSETTO:-0500
TZNAME:EST
DTSTART:20191103T060000
END:STANDARD
BEGIN:DAYLIGHT
TZOFFSETFROM:-0500
TZOFFSETTO:-0400
TZNAME:EDT
DTSTART:20200308T070000
END:DAYLIGHT
BEGIN:STANDARD
TZOFFSETFROM:-0400
TZOFFSETTO:-0500
TZNAME:EST
DTSTART:20201101T060000
END:STANDARD
BEGIN:DAYLIGHT
TZOFFSETFROM:-0500
TZOFFSETTO:-0400
TZNAME:EDT
DTSTART:20210314T070000
END:DAYLIGHT
BEGIN:STANDARD
TZOFFSETFROM:-0400
TZOFFSETTO:-0500
TZNAME:EST
DTSTART:20211107T060000
END:STANDARD
END:VTIMEZONE
BEGIN:VEVENT
DTSTART;TZID=America/Detroit:20201116T160000
DTEND;TZID=America/Detroit:20201116T170000
DTSTAMP:20260420T093340
CREATED:20200819T182858Z
LAST-MODIFIED:20201105T193634Z
UID:6391-1605542400-1605546000@rna.umich.edu
SUMMARY:RNA Innovation Seminar: Michelle Hastings\, Rosalind Franklin University
DESCRIPTION:“Splice-switching Antisense Oligonucleotides for the Treatment of Disease”\n\nMichelle Hastings\, Ph.D.\nProfessor\, Cell Biology and Anatomy; Director\, Center for Genetic Diseases\nRosalind Franklin University of Medicine and Science\n \nZOOM \nREGISTRATION REQUIRED: https://umich.zoom.us/webinar/register/WN_VWX5SY6lSiaNyh5Weh8cHw \nSeminar flyer in PDF \n  \nABSTRACT: Antisense oligonucleotides (ASOs) have proven to be an effective therapeutic platform for the treatment of disease. These short\, single-stranded\, modified nucleotides function by base-pairing with the complementary sequence of an RNA and modulating gene expression in a manner that is dependent on the ASO design and targeting site. We have used ASOs to normalize aberrant gene expression associated with a number of diseases of the nervous system including Alzheimer’s and Parkinson’s disease and Usher syndrome. One of our approaches is under development for the treatment of CLN3 Batten disease\, a fatal\, pediatric lysosomal storage disease caused by mutations in a gene encoding the lysosomal membrane protein CLN3. The most common mutation associated with CLN3 Batten is a deletion of exons 7 and 8 (CLN3Δex78)\, which disrupts the mRNA open reading frame by creating a premature termination codon that results in the production of a truncated protein. We devised a therapeutic strategy for treating CLN3 Batten Disease using an ASO that basepairs to CLN3 pre-mRNA and alters splicing to correct the open reading frame of the mutated transcript. Treatment of CLN3Δex78 neonatal mice by intracerebroventricular injection of the ASO resulted in the desired splicing effect throughout the central nervous system\, improved motor deficits associated with the disease in mice\, reduced histopathological features of the disease in the brain and extended life in a severe mouse model of the disease. Our results demonstrate that ASO-mediated reading frame correction is a promising therapeutic approach for CLN3 Batten disease. \nKEYWORDS: pre-mRNA splicing\, Antisense oligonucleotides\, Usher syndrome\, Batten Disease\, lysosomal storage diseases
URL:https://rna.umich.edu/events/seminar-michelle-hastings-rosalind-franklin/
END:VEVENT
END:VCALENDAR