U1 Adaptor: Demonstrated In Vivo Potency and Low Toxicity Provide Clear Advantages for a Next-Generation Gene Silencing Technology in Life Science Research

Samuel Gunderson (Inventor), Rafal Goraczniak (Inventor)

Research output: Innovation

Abstract


Invention Summary:

Rutgers scientists have developed compositions of single-stranded oligonucleotides that inhibit RNA biosynthesis by a novel mechanism of action. This enables the development of highly efficient gene silencing agents that have shown unprecedented inhibitory levels both in vitro and in vivo. The U1 adaptor consists of two parts, a target-gene binding domain and a U1 domain that attracts and inhibits the cellular polyA-tail addition apparatus. By combining both capabilities in the same molecule, the U1 adaptor can specifically inhibit the pre-mRNA maturation step of polyA-tail addition of a targeted sequence. Further, the domains of the oligonucleotides are independent so transcript binding and polyA-tail addition inhibition can be independently optimized and adapted to a wide array of genes.

Market Application:

Novel life science research tools for in vitro and in vivo gene function studies and gene target validation for therapeutics discovery.

Advantages:

The ability to selectively target gene silencing technologies to a wide array of desired genes is a broadly applicable and valuable research tool. To date, two main types of oligonucleotide-based methods have been employed in determining the effects of gene silencing: (1) antisense oligonucleotides (ASO) and (2) small interfering RNA (siRNA). In spite of the general success of siRNA in cell culture, its translation into in vivo models has proven difficult - primarily because of the inability to deliver silencing activity to specific organs or cell types. ASOs on the other hand have struggled with low potency.

The gene silencing approach made possible by the U1 Adaptor is the answer to many of the short-falls of the other technologies. It works via a distinct mechanism, involving blocking polyA tail addition to the 3’ end of gene-specific mRNA, and has several advantages over these other methods. One of the greatest advantages is its highly flexible chemistry that allows a broad variety of nucleotide modifications to promote stability and cell-specific delivery in vivo with virtually no ‘off-target’ effects.

Intellectual Property & Development Status:

Patent pending. Claims of compositions and methods for modulating gene expression. Technology is exclusively licensed to company, through which sub-licenses are available; company is also seeking funding and collaborations.

Original languageEnglish (US)
StatePublished - Feb 2015

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Biological Science Disciplines
Gene Silencing
Technology
Research
Oligonucleotides
Genes
Small Interfering RNA
Intellectual Property
Antisense Oligonucleotides
RNA Precursors
Licensure
Nucleotides
Cell Culture Techniques
RNA
Gene Expression
Messenger RNA

Keywords

  • Oligonucleotide
  • Prophylactics
  • RNAi
  • Research Tool
  • Therapeutics

Cite this

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title = "U1 Adaptor: Demonstrated In Vivo Potency and Low Toxicity Provide Clear Advantages for a Next-Generation Gene Silencing Technology in Life Science Research",
abstract = "Invention Summary: Rutgers scientists have developed compositions of single-stranded oligonucleotides that inhibit RNA biosynthesis by a novel mechanism of action. This enables the development of highly efficient gene silencing agents that have shown unprecedented inhibitory levels both in vitro and in vivo. The U1 adaptor consists of two parts, a target-gene binding domain and a U1 domain that attracts and inhibits the cellular polyA-tail addition apparatus. By combining both capabilities in the same molecule, the U1 adaptor can specifically inhibit the pre-mRNA maturation step of polyA-tail addition of a targeted sequence. Further, the domains of the oligonucleotides are independent so transcript binding and polyA-tail addition inhibition can be independently optimized and adapted to a wide array of genes. Market Application: Novel life science research tools for in vitro and in vivo gene function studies and gene target validation for therapeutics discovery. Advantages: The ability to selectively target gene silencing technologies to a wide array of desired genes is a broadly applicable and valuable research tool. To date, two main types of oligonucleotide-based methods have been employed in determining the effects of gene silencing: (1) antisense oligonucleotides (ASO) and (2) small interfering RNA (siRNA). In spite of the general success of siRNA in cell culture, its translation into in vivo models has proven difficult - primarily because of the inability to deliver silencing activity to specific organs or cell types. ASOs on the other hand have struggled with low potency. The gene silencing approach made possible by the U1 Adaptor is the answer to many of the short-falls of the other technologies. It works via a distinct mechanism, involving blocking polyA tail addition to the 3’ end of gene-specific mRNA, and has several advantages over these other methods. One of the greatest advantages is its highly flexible chemistry that allows a broad variety of nucleotide modifications to promote stability and cell-specific delivery in vivo with virtually no ‘off-target’ effects. Intellectual Property & Development Status: Patent pending. Claims of compositions and methods for modulating gene expression. Technology is exclusively licensed to company, through which sub-licenses are available; company is also seeking funding and collaborations.",
keywords = "Oligonucleotide, Prophylactics, RNAi, Research Tool, Therapeutics",
author = "Samuel Gunderson and Rafal Goraczniak",
year = "2015",
month = "2",
language = "English (US)",
type = "Patent",

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AU - Gunderson, Samuel

AU - Goraczniak, Rafal

PY - 2015/2

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N2 - Invention Summary: Rutgers scientists have developed compositions of single-stranded oligonucleotides that inhibit RNA biosynthesis by a novel mechanism of action. This enables the development of highly efficient gene silencing agents that have shown unprecedented inhibitory levels both in vitro and in vivo. The U1 adaptor consists of two parts, a target-gene binding domain and a U1 domain that attracts and inhibits the cellular polyA-tail addition apparatus. By combining both capabilities in the same molecule, the U1 adaptor can specifically inhibit the pre-mRNA maturation step of polyA-tail addition of a targeted sequence. Further, the domains of the oligonucleotides are independent so transcript binding and polyA-tail addition inhibition can be independently optimized and adapted to a wide array of genes. Market Application: Novel life science research tools for in vitro and in vivo gene function studies and gene target validation for therapeutics discovery. Advantages: The ability to selectively target gene silencing technologies to a wide array of desired genes is a broadly applicable and valuable research tool. To date, two main types of oligonucleotide-based methods have been employed in determining the effects of gene silencing: (1) antisense oligonucleotides (ASO) and (2) small interfering RNA (siRNA). In spite of the general success of siRNA in cell culture, its translation into in vivo models has proven difficult - primarily because of the inability to deliver silencing activity to specific organs or cell types. ASOs on the other hand have struggled with low potency. The gene silencing approach made possible by the U1 Adaptor is the answer to many of the short-falls of the other technologies. It works via a distinct mechanism, involving blocking polyA tail addition to the 3’ end of gene-specific mRNA, and has several advantages over these other methods. One of the greatest advantages is its highly flexible chemistry that allows a broad variety of nucleotide modifications to promote stability and cell-specific delivery in vivo with virtually no ‘off-target’ effects. Intellectual Property & Development Status: Patent pending. Claims of compositions and methods for modulating gene expression. Technology is exclusively licensed to company, through which sub-licenses are available; company is also seeking funding and collaborations.

AB - Invention Summary: Rutgers scientists have developed compositions of single-stranded oligonucleotides that inhibit RNA biosynthesis by a novel mechanism of action. This enables the development of highly efficient gene silencing agents that have shown unprecedented inhibitory levels both in vitro and in vivo. The U1 adaptor consists of two parts, a target-gene binding domain and a U1 domain that attracts and inhibits the cellular polyA-tail addition apparatus. By combining both capabilities in the same molecule, the U1 adaptor can specifically inhibit the pre-mRNA maturation step of polyA-tail addition of a targeted sequence. Further, the domains of the oligonucleotides are independent so transcript binding and polyA-tail addition inhibition can be independently optimized and adapted to a wide array of genes. Market Application: Novel life science research tools for in vitro and in vivo gene function studies and gene target validation for therapeutics discovery. Advantages: The ability to selectively target gene silencing technologies to a wide array of desired genes is a broadly applicable and valuable research tool. To date, two main types of oligonucleotide-based methods have been employed in determining the effects of gene silencing: (1) antisense oligonucleotides (ASO) and (2) small interfering RNA (siRNA). In spite of the general success of siRNA in cell culture, its translation into in vivo models has proven difficult - primarily because of the inability to deliver silencing activity to specific organs or cell types. ASOs on the other hand have struggled with low potency. The gene silencing approach made possible by the U1 Adaptor is the answer to many of the short-falls of the other technologies. It works via a distinct mechanism, involving blocking polyA tail addition to the 3’ end of gene-specific mRNA, and has several advantages over these other methods. One of the greatest advantages is its highly flexible chemistry that allows a broad variety of nucleotide modifications to promote stability and cell-specific delivery in vivo with virtually no ‘off-target’ effects. Intellectual Property & Development Status: Patent pending. Claims of compositions and methods for modulating gene expression. Technology is exclusively licensed to company, through which sub-licenses are available; company is also seeking funding and collaborations.

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KW - Prophylactics

KW - RNAi

KW - Research Tool

KW - Therapeutics

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M3 - Innovation

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