Methods and agents for increasing rbm3 expression

Simple SummaryContent extracted from patent full text and abstract with AI.

This invention describes methods and agents, such as antisense oligonucleotides and CRISPR/Cas systems, to increase the expression of the RBM3 protein by targeting its exon 3a or related splice sites. The increased RBM3 expression can be used for neuroprotective purposes and for treating diseases influenced by RBM3 levels.

Use CasesContent extracted from patent full text and abstract with AI.

• Developing new therapies to protect neurons in neurodegenerative diseases such as Alzheimer's or Parkinson's disease
• Enhancing neuron survival after brain injury or stroke
• Providing treatments for conditions where increased RBM3 can improve outcomes, such as traumatic brain injury or ischemia
• Research tools for studying RBM3 function and its role in cellular processes

BenefitsContent extracted from patent full text and abstract with AI.

• Offers targeted approaches to upregulate a neuroprotective protein (RBM3)
• Potential to slow or prevent neurodegeneration in serious diseases
• Enables precision intervention at the genetic or RNA level for specific therapeutic outcomes
• May improve recovery outcomes after neuronal injury
• Provides a foundation for safer, more effective neuroprotective treatments

Inventors & Applicants

Patent Abstract

The present invention provides agents (e.g., antisense oligonucleotides (ASO), CRISPR/Cas based base editing systems) that are capable of increasing the expression of RNA binding motif protein 3 (RBM3) by targeting the toxic exon of RBM3, namely exon 3a, or a splice site thereof. Also disclosed are methods for increasing expression of RBM3 in a cell, methods for treating or preventing a disease affected by expression of RMB3 in a subject, or methods for providing a neuroprotective treatment to a subject.