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GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease. However, before siRNAs can become drugs, they must overcome a billion years of evolutionary defenses designed to keep in... Full description

Journal Title: Nucleic acid therapeutics 2018, Vol.28 (3), p.19-118
Main Author: Springer, Aaron D.
Other Authors: Dowdy, Steven F.
Format: Electronic Article Electronic Article
Language: English
Subjects:
RNA
Quelle: Alma/SFX Local Collection
Publisher: United States: Mary Ann Liebert, Inc
ID: ISSN: 2159-3337
Link: https://www.ncbi.nlm.nih.gov/pubmed/29792572
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title: GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics
format: Article
creator:
  • Springer, Aaron D.
  • Dowdy, Steven F.
subjects:
  • Acids
  • Antisense oligonucleotides
  • Antisense RNA
  • Cancer
  • Clinical trials
  • Conjugates
  • Cytoplasm
  • Endocytosis
  • Gene expression
  • Hepatitis
  • Hepatocytes
  • Investments
  • Ligands
  • Liver
  • Medical research
  • Movement disorders
  • N-Acetylgalactosamine
  • Nanoparticles
  • Neurodegenerative diseases
  • Outbreaks
  • Pandemics
  • Parkinson's disease
  • Reviews
  • Ribonucleic acid
  • RNA
  • RNA-mediated interference
  • siRNA
  • Studies
  • Two phase
ispartof: Nucleic acid therapeutics, 2018, Vol.28 (3), p.19-118
description: Short-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease. However, before siRNAs can become drugs, they must overcome a billion years of evolutionary defenses designed to keep invading RNAs on the outside cells from getting to the inside of cells. Not surprisingly, significant effort has been placed in developing a wide array of delivery technologies. Foremost of these has been the development of N -acetylgalactosamine (GalNAc) siRNA conjugates for delivery to liver. Tris-GalNAc binds to the Asialoglycoprotein receptor that is highly expressed on hepatocytes resulting in rapid endocytosis. While the exact mechanism of escape across the endosomal lipid bilayer membrane remains unknown, sufficient amounts of siRNAs enter the cytoplasm to induce robust, target selective RNAi responses in vivo . Multiple GalNAc-siRNA conjugate clinical trials, including two phase III trials, are currently underway by three biotech companies to treat a wide variety of diseases. GalNAc-siRNA conjugates are a simple solution to the siRNA delivery problem for liver hepatocytes and have shown the RNAi (and antisense oligonucleotide) field the path forward for targeting other tissue types.
language: eng
source: Alma/SFX Local Collection
identifier: ISSN: 2159-3337
fulltext: fulltext
issn:
  • 2159-3337
  • 2159-3345
  • 1557-8526
url: Link


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descriptionShort-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease. However, before siRNAs can become drugs, they must overcome a billion years of evolutionary defenses designed to keep invading RNAs on the outside cells from getting to the inside of cells. Not surprisingly, significant effort has been placed in developing a wide array of delivery technologies. Foremost of these has been the development of N -acetylgalactosamine (GalNAc) siRNA conjugates for delivery to liver. Tris-GalNAc binds to the Asialoglycoprotein receptor that is highly expressed on hepatocytes resulting in rapid endocytosis. While the exact mechanism of escape across the endosomal lipid bilayer membrane remains unknown, sufficient amounts of siRNAs enter the cytoplasm to induce robust, target selective RNAi responses in vivo . Multiple GalNAc-siRNA conjugate clinical trials, including two phase III trials, are currently underway by three biotech companies to treat a wide variety of diseases. GalNAc-siRNA conjugates are a simple solution to the siRNA delivery problem for liver hepatocytes and have shown the RNAi (and antisense oligonucleotide) field the path forward for targeting other tissue types.
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subjectAcids ; Antisense oligonucleotides ; Antisense RNA ; Cancer ; Clinical trials ; Conjugates ; Cytoplasm ; Endocytosis ; Gene expression ; Hepatitis ; Hepatocytes ; Investments ; Ligands ; Liver ; Medical research ; Movement disorders ; N-Acetylgalactosamine ; Nanoparticles ; Neurodegenerative diseases ; Outbreaks ; Pandemics ; Parkinson's disease ; Reviews ; Ribonucleic acid ; RNA ; RNA-mediated interference ; siRNA ; Studies ; Two phase
ispartofNucleic acid therapeutics, 2018, Vol.28 (3), p.19-118
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abstractShort-interfering RNA (siRNA)-induced RNAi responses have great potential to treat a wide variety of human diseases from cancer to pandemic viral outbreaks to Parkinson's Disease. However, before siRNAs can become drugs, they must overcome a billion years of evolutionary defenses designed to keep invading RNAs on the outside cells from getting to the inside of cells. Not surprisingly, significant effort has been placed in developing a wide array of delivery technologies. Foremost of these has been the development of N -acetylgalactosamine (GalNAc) siRNA conjugates for delivery to liver. Tris-GalNAc binds to the Asialoglycoprotein receptor that is highly expressed on hepatocytes resulting in rapid endocytosis. While the exact mechanism of escape across the endosomal lipid bilayer membrane remains unknown, sufficient amounts of siRNAs enter the cytoplasm to induce robust, target selective RNAi responses in vivo . Multiple GalNAc-siRNA conjugate clinical trials, including two phase III trials, are currently underway by three biotech companies to treat a wide variety of diseases. GalNAc-siRNA conjugates are a simple solution to the siRNA delivery problem for liver hepatocytes and have shown the RNAi (and antisense oligonucleotide) field the path forward for targeting other tissue types.
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