scholarly journals Targeted delivery of antisense oligonucleotides by molecular conjugates

1992 ◽  
Vol 18 (6) ◽  
pp. 559-569 ◽  
Author(s):  
Bruce A. Bunnell ◽  
Frederick K. Askari ◽  
James M. Wilson
Keyword(s):  
Targeted Delivery ◽  
Antisense Oligonucleotides

N-Acetyl Galactosamine Targeting: Paving the Way for Clinical Application of Nucleotide Medicines in Cardiovascular Diseases

2021 ◽  
Author(s):  
Erik A.L. Biessen ◽  
Theo J.C. Van Berkel
Keyword(s):  
Immune Responses ◽  
Targeted Delivery ◽  
Antisense Oligonucleotides ◽  
Sugar Metabolism ◽  
Innate Immune ◽  
Future Research ◽  
Clinical Use ◽  
Innate Immune Responses ◽  
Chemically Modified ◽  
Intrinsic Capacity

While the promise of oligonucleotide therapeutics, such as (chemically modified) ASO (antisense oligonucleotides) and short interfering RNAs, is undisputed from their introduction onwards, their unfavorable pharmacokinetics and intrinsic capacity to mobilize innate immune responses, were limiting widespread clinical use. However, these major setbacks have been tackled by breakthroughs in chemistry, stability and delivery. When aiming an intervention hepatic targets, such as lipid and sugar metabolism, coagulation, not to mention cancer and virus infection, introduction of N-acetylgalactosamine aided targeting technology has advanced the field profoundly and by now a dozen of N-acetylgalactosamine therapeutics for these indications have been approved for clinical use or have progressed to clinical trial stage 2 to 3 testing. This technology, in combination with major advances in oligonucleotide stability allows safe and durable intervention in targets that were previously deemed undruggable, such as Lp(a) and PCSK9, at high efficacy and specificity, often with as little as 2 doses per year. Their successful use even the most visionary would not have predicted 2 decades ago. Here, we will review the evolution of N-acetylgalactosamine technology. We shall outline their fundamental design principles and merits, and their application for the delivery of oligonucleotide therapeutics to the liver. Finally, we will discuss the perspectives of N-acetylgalactosamine technology and propose directions for future research in receptor targeted delivery of these gene medicines.

scholarly journals Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

2014 ◽  
Vol 42 (13) ◽  
pp. 8796-8807 ◽  
Author(s):  
Thazha P. Prakash ◽  
Mark J. Graham ◽  
Jinghua Yu ◽  
Rick Carty ◽  
Audrey Low ◽  
...  
Keyword(s):  
Targeted Delivery ◽  
Antisense Oligonucleotides

Targeted delivery of antisense oligonucleotides in cancer

2001 ◽  
Vol 74 (1-3) ◽  
pp. 69-75 ◽  
Author(s):  
F. Pastorino ◽  
D. Stuart ◽  
M. Ponzoni ◽  
T.M. Allen
Keyword(s):  
Targeted Delivery ◽  
Antisense Oligonucleotides

scholarly journals 967. Targeted Delivery of Antisense Oligonucleotides to Human T-Leukemia Cells Mediated by Novel Liposomal Formulations: Evaluation of In Vitro and In Vivo Anti-Tumoral Activity

2006 ◽  
Vol 13 ◽  
pp. S372
Author(s):  
Cristina Fonseca ◽  
Liliana Mendonça ◽  
João Nuno Moreira ◽  
António Cabrita ◽  
Artur Paiva ◽  
...  
Keyword(s):  
Targeted Delivery ◽  
Antisense Oligonucleotides ◽  
Leukemia Cells ◽  
Liposomal Formulations

Glucagon Like Peptide 1 Receptor Agonists for Targeted Delivery of Antisense Oligonucleotides to Pancreatic Beta Cell

2021 ◽  
Vol 143 (9) ◽  
pp. 3416-3429
Author(s):  
Laurent Knerr ◽  
Thazha P. Prakash ◽  
Richard Lee ◽  
William J. Drury III ◽  
Mehran Nikan ◽  
...  
Keyword(s):  
Beta Cell ◽  
Targeted Delivery ◽  
Antisense Oligonucleotides ◽  
Pancreatic Beta Cell ◽  
Receptor Agonists ◽  
Glucagon Like Peptide ◽  
Glucagon Like Peptide 1

Targeted Delivery of Antisense Oligonucleotides to Cells by Immunoliposomes

1991 ◽  
Vol 1 (4) ◽  
pp. 379-379
Author(s):  
H. LOUGHREY ◽  
P. MACHY ◽  
O. ZELPHATI ◽  
L. LESERMAN
Keyword(s):  
Targeted Delivery ◽  
Antisense Oligonucleotides

Targeted delivery system for antisense oligonucleotides: a novel experimental strategy for neuroblastoma treatment

2003 ◽  
Vol 197 (1-2) ◽  
pp. 231-235 ◽  
Author(s):  
Chiara Brignole ◽  
Gabriella Pagnan ◽  
Danilo Marimpietri ◽  
Emilio Cosimo ◽  
Theresa M. Allen ◽  
...  
Keyword(s):  
Delivery System ◽  
Targeted Delivery ◽  
Antisense Oligonucleotides ◽  
Experimental Strategy ◽  
Targeted Delivery System
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