Nucleic acid aptamers: Clinical applications and promising new horizons

X. Ni, M. Castanares, Amarnath Mukherjee, S. E. Lupold

Research output: Contribution to journalReview article

169 Citations (Scopus)

Abstract

Aptamers are a special class of nucleic acid molecules that are beginning to be investigated for clinical use. These small RNA/DNA molecules can form secondary and tertiary structures capable of specifically binding proteins or other cellular targets; they are essentially a chemical equivalent of antibodies. Aptamers have the advantage of being highly specific, relatively small in size, and non-immunogenic. Since the discovery of aptamers in the early 1990s, great efforts have been made to make them clinically relevant for diseases like cancer, HIV, and macular degeneration. In the last two decades, many aptamers have been clinically developed as inhibitors for targets such as vascular endothelial growth factor (VEGF) and thrombin. The first aptamer based therapeutic was FDA approved in 2004 for the treatment of age-related macular degeneration and several other aptamers are currently being evaluated in clinical trials. With advances in targeted-therapy, imaging, and nanotechnology, aptamers are readily considered as potential targeting ligands because of their chemical synthesis and ease of modification for conjugation. Preclinical studies using aptamer-siRNA chimeras and aptamer targeted nanoparticle therapeutics have been very successful in mouse models of cancer and HIV. In summary aptamers are in several stages of development, from pre-clinical studies to clinical trials and even as FDA approved therapeutics. In this review, we will discuss the current state of aptamers in clinical trials as well as some promising aptamers in pre-clinical development.

Original languageEnglish (US)
Pages (from-to)4206-4214
Number of pages9
JournalCurrent Medicinal Chemistry
Volume18
Issue number27
DOIs
StatePublished - Sep 2011
Externally publishedYes

Fingerprint

Nucleic Acids
Molecules
Macular Degeneration
Clinical Trials
Nanotechnology
Thrombin
Vascular Endothelial Growth Factor A
Small Interfering RNA
Carrier Proteins
HIV
RNA
Nanoparticles
Ligands
Imaging techniques
Antibodies
DNA
Therapeutics
Neoplasms

Keywords

  • Aptamers
  • Clinical trial
  • Imaging
  • Nanoparticle
  • Oligonucleotides
  • SELEX
  • SiRNA
  • Target therapy

ASJC Scopus subject areas

  • Molecular Medicine
  • Pharmacology

Cite this

Nucleic acid aptamers : Clinical applications and promising new horizons. / Ni, X.; Castanares, M.; Mukherjee, Amarnath; Lupold, S. E.

In: Current Medicinal Chemistry, Vol. 18, No. 27, 09.2011, p. 4206-4214.

Research output: Contribution to journalReview article

Ni, X. ; Castanares, M. ; Mukherjee, Amarnath ; Lupold, S. E. / Nucleic acid aptamers : Clinical applications and promising new horizons. In: Current Medicinal Chemistry. 2011 ; Vol. 18, No. 27. pp. 4206-4214.
@article{30754c9ebc0c4d128a00077adf0f7d8e,
title = "Nucleic acid aptamers: Clinical applications and promising new horizons",
abstract = "Aptamers are a special class of nucleic acid molecules that are beginning to be investigated for clinical use. These small RNA/DNA molecules can form secondary and tertiary structures capable of specifically binding proteins or other cellular targets; they are essentially a chemical equivalent of antibodies. Aptamers have the advantage of being highly specific, relatively small in size, and non-immunogenic. Since the discovery of aptamers in the early 1990s, great efforts have been made to make them clinically relevant for diseases like cancer, HIV, and macular degeneration. In the last two decades, many aptamers have been clinically developed as inhibitors for targets such as vascular endothelial growth factor (VEGF) and thrombin. The first aptamer based therapeutic was FDA approved in 2004 for the treatment of age-related macular degeneration and several other aptamers are currently being evaluated in clinical trials. With advances in targeted-therapy, imaging, and nanotechnology, aptamers are readily considered as potential targeting ligands because of their chemical synthesis and ease of modification for conjugation. Preclinical studies using aptamer-siRNA chimeras and aptamer targeted nanoparticle therapeutics have been very successful in mouse models of cancer and HIV. In summary aptamers are in several stages of development, from pre-clinical studies to clinical trials and even as FDA approved therapeutics. In this review, we will discuss the current state of aptamers in clinical trials as well as some promising aptamers in pre-clinical development.",
keywords = "Aptamers, Clinical trial, Imaging, Nanoparticle, Oligonucleotides, SELEX, SiRNA, Target therapy",
author = "X. Ni and M. Castanares and Amarnath Mukherjee and Lupold, {S. E.}",
year = "2011",
month = "9",
doi = "10.2174/092986711797189600",
language = "English (US)",
volume = "18",
pages = "4206--4214",
journal = "Current Medicinal Chemistry",
issn = "0929-8673",
publisher = "Bentham Science Publishers B.V.",
number = "27",

}

TY - JOUR

T1 - Nucleic acid aptamers

T2 - Clinical applications and promising new horizons

AU - Ni, X.

AU - Castanares, M.

AU - Mukherjee, Amarnath

AU - Lupold, S. E.

PY - 2011/9

Y1 - 2011/9

N2 - Aptamers are a special class of nucleic acid molecules that are beginning to be investigated for clinical use. These small RNA/DNA molecules can form secondary and tertiary structures capable of specifically binding proteins or other cellular targets; they are essentially a chemical equivalent of antibodies. Aptamers have the advantage of being highly specific, relatively small in size, and non-immunogenic. Since the discovery of aptamers in the early 1990s, great efforts have been made to make them clinically relevant for diseases like cancer, HIV, and macular degeneration. In the last two decades, many aptamers have been clinically developed as inhibitors for targets such as vascular endothelial growth factor (VEGF) and thrombin. The first aptamer based therapeutic was FDA approved in 2004 for the treatment of age-related macular degeneration and several other aptamers are currently being evaluated in clinical trials. With advances in targeted-therapy, imaging, and nanotechnology, aptamers are readily considered as potential targeting ligands because of their chemical synthesis and ease of modification for conjugation. Preclinical studies using aptamer-siRNA chimeras and aptamer targeted nanoparticle therapeutics have been very successful in mouse models of cancer and HIV. In summary aptamers are in several stages of development, from pre-clinical studies to clinical trials and even as FDA approved therapeutics. In this review, we will discuss the current state of aptamers in clinical trials as well as some promising aptamers in pre-clinical development.

AB - Aptamers are a special class of nucleic acid molecules that are beginning to be investigated for clinical use. These small RNA/DNA molecules can form secondary and tertiary structures capable of specifically binding proteins or other cellular targets; they are essentially a chemical equivalent of antibodies. Aptamers have the advantage of being highly specific, relatively small in size, and non-immunogenic. Since the discovery of aptamers in the early 1990s, great efforts have been made to make them clinically relevant for diseases like cancer, HIV, and macular degeneration. In the last two decades, many aptamers have been clinically developed as inhibitors for targets such as vascular endothelial growth factor (VEGF) and thrombin. The first aptamer based therapeutic was FDA approved in 2004 for the treatment of age-related macular degeneration and several other aptamers are currently being evaluated in clinical trials. With advances in targeted-therapy, imaging, and nanotechnology, aptamers are readily considered as potential targeting ligands because of their chemical synthesis and ease of modification for conjugation. Preclinical studies using aptamer-siRNA chimeras and aptamer targeted nanoparticle therapeutics have been very successful in mouse models of cancer and HIV. In summary aptamers are in several stages of development, from pre-clinical studies to clinical trials and even as FDA approved therapeutics. In this review, we will discuss the current state of aptamers in clinical trials as well as some promising aptamers in pre-clinical development.

KW - Aptamers

KW - Clinical trial

KW - Imaging

KW - Nanoparticle

KW - Oligonucleotides

KW - SELEX

KW - SiRNA

KW - Target therapy

UR - http://www.scopus.com/inward/record.url?scp=80052228547&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=80052228547&partnerID=8YFLogxK

U2 - 10.2174/092986711797189600

DO - 10.2174/092986711797189600

M3 - Review article

C2 - 21838685

AN - SCOPUS:80052228547

VL - 18

SP - 4206

EP - 4214

JO - Current Medicinal Chemistry

JF - Current Medicinal Chemistry

SN - 0929-8673

IS - 27

ER -