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Element 1 atisense morpholinos

标题

SMN2 ELEMENT 1 ANTISENSE COMPOSITIONS AND METHODS AND USES THEREOF

详细技术说明

None

*Abstract

Element 1 Antisense MorpholinosSpinal Muscular Atrophy (SMA) is a lethal genetic disease in infants that causes degeneration of SMN protein in the spinal cord. In humans, SMN protein is produced by a gene called Survival Motor Neuron 1 (SMN1). Loss of the SMN1 gene in newborns leads to the development of SMA and ultimately death. An identical gene to SMN1 known as Survival Motor Neuron 2(SMN2) is expressed in humans but can only make a fraction of SMN protein. SMN2 lacks a final coding exon (exon 7) that permits SMN1 the ability to create an abundant amount of SMN protein. However, infants with SMA still retain a copy of SMN2 thereby making it an ideal target to modulate its splicing patterns and produce more SMN protein.The current invention developed by the researchers at the University of Missouri utilizes Morpholino chemistry to create a SMN2 specific antisense oligonucleotide (ASO). This antisense oligonucleotide anneals to a repressor region of SMN2 exon 7 called Element 1 (E1). Uninhibited E1 activity leads to the reduced production of SMN protein by the SMN2 gene. The antisense oligonucleotide blocks E1 repressive activity and allows the SMN2 gene to express large amounts of SMN protein. This gene therapy can help replenish the defect of SMN protein seen in infants with SMA.POTENTIAL AREAS OF APPLICATION- Pharmaceutical companies developing SMA-specific therapiesMAIN ADVANTAGES OF INVENTION- Unique mechanism for increased SMN protein productionSTATE OF DEVELOPMENT:Complete. However, additional data is being gathered.LICENSING POTENTIAL: University seeks licensee with the potential to continue research and commercializePATENT STATUS: New - In ProgressINVENTORS: Christian Lorson and Erkan Osman TECHNOLOGY MANAGEMENT CONTACTSHarriet F. Francis, MS; J.D.; francish@missouri.edu; 573-884-037Nancy Parker, PhD; parkern@missouri.edu; 573- 884-3553

*IP Issue Date

Feb 6, 2018

*IP Publication Date

Mar 10, 2016

*Principal Investigation

Name: Chris Lorson, Associate Professor, Veterniary Pathobiology

Department:

Name: Erkan Osman

Department:

申请日期

Oct 9, 2015

申请号码

9,885,040

其他

国家/地区

美国