Online citations, reference lists, and bibliographies.
← Back to Search

The Cellular Delivery Of Antisense Oligonucleotides And Ribozymes.

M. Hughes, M. Hussain, Q. Nawaz, P. Sayyed, S. Akhtar
Published 2001 · Biology, Medicine

Save to my Library
Download PDF
Analyze on Scholarcy
Share
The design and development of antisense oligonucleotides and ribozymes for the treatment of diseases arising from genetic abnormalities has become a real possibility over the past few years. Improvements in oligonucleotide chemistry have led to the synthesis of nucleic acids that are relatively stable in the biological milieu. However, advances in cellular targeting and intracellular delivery will probably lead to more widespread clinical applications. This review looks at recent advances in the in vitro and in vivo delivery of antisense oligodeoxynucleotides and ribozymes.
This paper references
10.3109/10717549609029458
Dendrimer Delivery of Oligonucleotides
S. Poxon (1996)
Pharmacokinetics and organ clearance of a 3'-biotinylated, internally [32P]-labeled phosphodiester oligodeoxynucleotide coupled to a neutral avidin/monoclonal antibody conjugate.
Y. Kang (1995)
10.1055/S-2007-1007101
Gene repair using chimeric RNA/DNA oligonucleotides.
B. Kren (1999)
10.1006/BMME.1996.0080
Toxicity of cationic lipid-ribozyme complexes in human prostate tumor cells can mimic ribozyme activity.
S. J. Freedland (1996)
10.1093/NAR/26.22.5079
Rapid determination and quantitation of the accessibility to native RNAs by antisense oligodeoxynucleotides in murine cell extracts.
M. Scherr (1998)
10.1126/SCIENCE.2468181
On finding all suboptimal foldings of an RNA molecule.
M. Zuker (1989)
10.1016/0014-5793(93)81002-H
Penetration of oligonucleotides into mouse organism through mucosa and skin
V. Vlassov (1993)
10.1126/SCIENCE.285.5433.1569
In vivo protein transduction: delivery of a biologically active protein into the mouse.
S. Schwarze (1999)
10.1089/OLI.1.1997.7.319
Cellular uptake properties of a 2'-amino/2'-O-methyl-modified chimeric hammerhead ribozyme targeted to the epidermal growth factor receptor mRNA.
P. Fell (1997)
10.1093/NAR/24.11.2176
Regulation of in vitro gene expression using antisense oligonucleotides or antisense expression plasmids transfected using starburst PAMAM dendrimers.
A. Bielinska (1996)
10.1073/PNAS.93.26.15481
Sequence-specific antitumor activity of a phosphorothioate oligodeoxyribonucleotide targeted to human C-raf kinase supports an antisense mechanism of action in vivo.
B. Monia (1996)
10.1038/nm0695-528
A single-injection protein kinase A-directed antisense treatment to inhibit tumour growth
M. Nesterova (1995)
10.1016/S0006-2952(00)00310-5
Antisense inhibition of P-glycoprotein expression using peptide-oligonucleotide conjugates.
A. Astriab-Fisher (2000)
10.1021/BC00029A005
Complete inactivation of target mRNA by biotinylated antisense oligodeoxynucleotide-avidin conjugates.
R. Boado (1994)
10.1016/0378-5173(96)04474-2
Biodegradable polymer matrices for the sustained exogenous delivery of a biologically active c-myc hammerhead ribozyme☆
A. Hudson (1996)
10.1073/PNAS.91.24.11762
Inhibition of protein kinase C-alpha expression in mice after systemic administration of phosphorothioate antisense oligodeoxynucleotides.
N. Dean (1994)
10.3109/10611869808995877
Antisense technology: selection and delivery of optimally acting antisense oligonucleotides.
S. Akhtar (1998)
10.1007/s002109900091
CB1 receptor density and CB1 receptor-mediated functional effects in rat hippocampus are decreased by an intracerebroventricularly administered antisense oligodeoxynucleotide
M. Kathmann (1999)
10.1016/S0968-0004(96)80019-3
Ribozymes: structure and mechanism in RNA catalysis.
W. G. Scott (1996)
c-Myc antisense limits rat liver regeneration and indicates role for c-Myc in regulating cytochrome P-450 3A activity.
V. Arora (2000)
10.1016/0006-2952(95)00160-2
Absorption, tissue distribution and in vivo stability in rats of a hybrid antisense oligonucleotide following oral administration.
S. Agrawal (1995)
10.1016/0165-022X(88)90065-6
alpha-Oligodeoxynucleotide stability in serum, subcellular extracts and culture media.
T. Bacon (1988)
Pharmacokinetics and blood-brain barrier transport of [3H]-biotinylated phosphorothioate oligodeoxynucleotide conjugated to a vector-mediated drug delivery system.
D. Wu (1996)
10.1016/S1074-5521(99)80046-5
Cellular delivery of peptide nucleic acids and inhibition of human telomerase.
S. Hamilton (1999)
10.1016/S0169-409X(00)00080-6
The delivery of antisense therapeutics.
S. Akhtar (2000)
Down-regulation of HER2/neu expression induces apoptosis in human cancer cells that overexpress HER2/neu.
H. Roh (2000)
10.3109/10611869909085520
Studies on uptake, sub-cellular trafficking and efflux of antisense oligodeoxynucleotides in glioma cells using self-assembling cationic lipoplexes as delivery systems.
A. Islam (2000)
10.3109/10611869808995882
Development of a sustained-release biodegradable polymer delivery system for site-specific delivery of oligonucleotides: characterization of P(LA-GA) copolymer microspheres in vitro.
K. Lewis (1998)
10.1016/0030-4220(71)90265-9
Histologic comparison of polylactic and polyglycolic acid sutures.
D. E. Cutright (1971)
10.1089/OLI.1.1999.9.333
Antisense technologies have a future fighting neurodegenerative diseases.
S. Seidman (1999)
10.1038/NM0498-467
In vivo imaging of oligonucleotides with positron emission tomography
B. Tavitian (1998)
10.1093/NAR/27.16.3334
Novel cationic amphiphiles as delivery agents for antisense oligonucleotides.
R. DeLong (1999)
10.1073/PNAS.91.22.10460
Antisense oligonucleotides adsorbed to polyalkylcyanoacrylate nanoparticles specifically inhibit mutated Ha-ras-mediated cell proliferation and tumorigenicity in nude mice.
G. Schwab (1994)
10.1016/S0378-5173(97)00363-3
Oligonucleotide delivery : Uptake of rat transferrin receptor antibody (OX-26) conjugates into an in vitro immortalised cell line model of the blood-brain barrier
N. Normand-Sdiqui (1998)
10.1016/0169-409X(95)00092-L
Biosynthetic polyhydroxyalkanoates and their potential in drug delivery
C. Pouton (1996)
10.1016/S0165-6147(96)01002-4
In vivo studies with antisense oligonucleotides.
S. Akhtar (1997)
Fate and behavior of liposomes in vivo: a review of controlling factors.
J. H. Senior (1987)
10.1089/OLI.1.1999.9.451
Enhanced anti-tumor effects with microencapsulated c-myc antisense oligonucleotide.
S. Putney (1999)
10.3109/10611860008997909
Site-Specific Administration of Antisense Oligonucleotides using Biodegradable Polymer Microspheres Provides Sustained Delivery and Improved Subcellular Biodistribution in the Neostriatum of the Rat Brain
A. Khan (2000)
10.1016/S0076-6879(00)13021-6
In vitro transport and delivery of antisense oligonucleotides.
J. Hughes (2000)
10.1016/0024-3205(91)90480-Y
Stability of antisense DNA oligodeoxynucleotide analogs in cellular extracts and sera.
S. Akhtar (1991)
10.1093/NAR/22.8.1368
Arrays of complementary oligonucleotides for analysing the hybridisation behaviour of nucleic acids.
E. Southern (1994)
10.1016/0006-8993(95)00363-U
Transport of [125I]transferrin through the rat blood-brain barrier
S. Skarlatos (1995)
10.1080/096876899294869
Lipid-based systems for the intracellular delivery of genetic drugs.
N. Maurer (1999)
10.1016/S0076-6879(00)13024-1
Cell-specific optimization of phosphorothioate antisense oligodeoxynucleotide delivery by cationic lipids.
S. Williams (2000)
10.1042/BJ3400783
Targeted delivery of oligodeoxynucleotides to parenchymal liver cells in vivo.
E. A. Biessen (1999)
10.1016/S0378-5173(99)00062-9
Cellular delivery of hammerhead ribozymes conjugated to a transferrin receptor antibody.
A. J. Hudson (1999)
10.1016/0962-8924(92)90100-2
Cellular uptake and intracellular fate of antisense oligonucleotides.
S. Akhtar (1992)
10.1517/13543776.10.8.1201
Recent developments in hepatitis C antiviral research 1999 - 2000
M. Reding (2000)
10.1016/S0378-5173(97)04887-4
Antisense oligonucleotide delivery to cultured macrophages is improved by incorporation into sustained-release biodegradable polymer microspheres
S. Akhtar (1997)
10.1038/NBT0198-59
Mapping of RNA accessible sites for antisense experiments with oligonucleotide libraries
S. P. Ho (1998)
Inhibition of expression of the multidrug resistance-associated P-glycoprotein of by phosphorothioate and 5' cholesterol-conjugated phosphorothioate antisense oligonucleotides.
S. Alahari (1996)
10.1093/NAR/26.18.4241
Intracellular metabolism of a 2'-O-methyl-stabilized ribozyme after uptake by DOTAP transfection or asfree ribozyme. A study by capillary electrophoresis.
L. Prasmickaite (1998)
10.1016/S0197-0186(96)00106-4
Critical issues in the antisense inhibition of brain gene expression in vivo: Experiences targetting the 5-HT1A receptor
S. L. Corre (1997)
10.1385/MB:12:1:1
Preclinical and clinical pharmacology of antisense oligonucleotides
E. Marcusson (1999)
10.1016/0005-2736(92)90316-E
Effect of cationic cholesterol derivatives on gene transfer and protein kinase C activity.
H. Farhood (1992)
10.1038/sj.onc.1203069
Telomerase inhibition by peptide nucleic acids reverses `immortality' of transformed human cells
M. Shammas (1999)
10.1016/S0169-409X(00)00081-8
Selecting optimal antisense reagents.
M. Sohail (2000)
10.1211/0022357991773113
Lipoplexes and Tumours. A Review
C. Dass (1999)
10.1016/S0006-2952(96)00862-3
Interaction of oligonucleotide-conjugates with the dipeptide transporter system in Caco-2 cells.
V. A. Moore (1997)
Specific Inhibition of Macrophage TNF-α Expression by In Vivo Ribozyme Treatment
K. Kisich (1999)
10.1073/PNAS.86.10.3474
Characterization of oligonucleotide transport into living cells.
S. Loke (1989)
10.1006/BBRC.1996.1827
Phosphorothiolate ribozyme against the conserved sequence in V3 loop of HIV-1.
X. Zhang (1996)
10.1016/S0165-6147(99)01429-7
In vivo protein transduction: intracellular delivery of biologically active proteins, compounds and DNA.
S. Schwarze (2000)
10.1038/312162A0
Transferrin receptor on endothelium of brain capillaries
W. Jefferies (1984)
10.1126/SCIENCE.8351515
Antisense oligonucleotides as therapeutic agents--is the bullet really magical?
C. Stein (1993)
10.1089/OLI.1.1999.9.105
Brain as a unique antisense environment.
A. Szklarczyk (1999)
10.1074/jbc.272.7.3910
Antisense Inhibition of Silica-induced Tumor Necrosis Factor in Alveolar Macrophages*
Y. Rojanasakul (1997)
10.1083/JCB.117.5.1085
Neurite outgrowth in peripherin-depleted PC12 cells
C. Troy (1992)
10.1016/0014-5793(87)80171-0
Sequence‐dependent hydrolysis of RNA using modified oligonucleotide splints and RNase H
H. Inoue (1987)
Oligonucleotide therapeutics: clothing the emperor.
A. Gewirtz (1999)
10.1016/S0005-2736(98)00067-4
pH-sensitive liposomes as a carrier for oligonucleotides: a physico-chemical study of the interaction between DOPE and a 15-mer oligonucleotide in quasi-anhydrous samples.
M. C. de Oliveira (1998)
10.1074/jbc.271.46.29107
Optimizing the Cell Efficacy of Synthetic Ribozymes
T. Jarvis (1996)
10.1074/JBC.272.1.626
Combinatorial Screening and Rational Optimization for Hybridization to Folded Hepatitis C Virus RNA of Oligonucleotides with Biological Antisense Activity*
W. Lima (1997)
10.1016/S0014-5793(98)00984-3
pH‐sensitive liposomes for receptor‐mediated delivery to chicken hepatoma (LMH) cells
N. Škalko (1998)
10.1007/978-1-4615-6353-2_5
Use of a hammerhead ribozyme with cationic liposomes to reduce leukocyte type 12-lipoxygenase expression in vascular smooth muscle.
Jiali Gu (1997)
10.1177/095632029800900306
Specific Inhibition of Influenza Virus RNA Polymerase and Nucleoprotein Gene Expression by Liposomally Encapsulated Antisense Phosphorothioate Oligonucleotides in MDCK Cells
T. Abe (1998)
10.1093/NAR/25.14.2730
A new peptide vector for efficient delivery of oligonucleotides into mammalian cells.
M. Morris (1997)
10.1016/S0168-3659(97)00246-0
Biodegradable polyalkylcyanoacrylate nanoparticles for the delivery of oligonucleotides.
E. Fattal (1998)
10.1097/00007890-200002150-00019
Attenuation of cytomegalovirus-induced endothelial intercellular adhesion molecule-1 mRNA/protein expression and T lymphocyte adhesion by a 2'-O-methoxyethyl antisense oligonucleotide.
D. Knight (2000)
10.1021/JS960409F
Characterization of complexes of oligonucleotides with polyamidoamine starburst dendrimers and effects on intracellular delivery.
R. DeLong (1997)
10.1016/0168-9525(96)81422-3
DNA chips: analysing sequence by hybridization to oligonucleotides on a large scale.
E. Southern (1996)
10.1016/0168-3659(95)00100-M
Biodegradable poly(L-lactic acid) matrices for the sustained delivery of antisense oligonucleotides
K. Lewis (1995)
10.1016/S0005-2736(98)00046-7
Delivery of an anti-HIV-1 ribozyme into HIV-infected cells via cationic liposomes.
K. Konopka (1998)
Toward antisense oligonucleotide therapy for cancer: ISIS compounds in clinical development.
J. Holmlund (1999)
Probing accessible sites for ribozymes on human acetylcholinesterase RNA.
K. R. Birikh (1997)
10.3109/02652049809008253
Protein delivery from poly(lactic-co-glycolic acid) biodegradable microspheres: release kinetics and stability issues.
G. Crotts (1998)
10.1093/NAR/20.12.3252
Numbering system for the hammerhead.
K. J. Hertel (1992)
10.1038/NBT0697-537
Selecting effective antisense reagents on combinatorial oligonucleotide arrays
N. Milner (1997)
10.1016/S0006-2952(96)00763-0
Scavenger receptor-mediated delivery of antisense mini-exon phosphorothioate oligonucleotide to Leishmania-infected macrophages. Selective and efficient elimination of the parasite.
G. Chaudhuri (1997)
10.1002/(SICI)1098-2396(199904)32:1<51::AID-SYN7>3.0.CO;2-E
Dopamine D3 receptor antisense administration reduces basal c‐fos and NGFI‐B mRNA levels in the rat forebrain
M. Tremblay (1999)
10.1093/NAR/28.21.4225
Enhanced delivery of antisense oligonucleotides with fluorophore-conjugated PAMAM dendrimers.
H. Yoo (2000)
10.1074/jbc.270.43.25702
Chemical Modification of Hammerhead Ribozymes
L. Beigelman (1995)
10.1073/pnas.88.7.2702
Intracellular distribution of microinjected antisense oligonucleotides.
J. P. Leonetti (1991)
Oligonucleotides as inhibitors of human immunodeficiency virus.
Field Ak (1999)
10.1073/PNAS.96.24.13989
Antitumor activity and pharmacokinetics of a mixed-backbone antisense oligonucleotide targeted to the RIalpha subunit of protein kinase A after oral administration.
H. Wang (1999)
10.1006/BBRC.1999.0290
Uptake of liposomes containing phosphatidylserine by liver cells in vivo and by sinusoidal liver cells in primary culture: in vivo-in vitro differences.
J. Kamps (1999)
10.1093/NAR/24.10.1901
Potent antisense oligonucleotides to the human multidrug resistance-1 mRNA are rationally selected by mapping RNA-accessible sites with oligonucleotide libraries.
S. P. Ho (1996)
A nonantisense sequence-selective effect of a phosphorothioate oligodeoxynucleotide directed against the epidermal growth factor receptor in A431 cells.
J. Coulson (1996)
10.1089/OLI.1.2000.10.35
Absorption of antisense oligonucleotides in rat intestine: effect of chemistry and length.
O. Khatsenko (2000)
10.1021/BC00023A012
Polyamidoamine cascade polymers mediate efficient transfection of cells in culture.
J. Hänsler (1993)
10.1016/S0168-3659(97)00052-7
Microparticles of poly(dl-lactic-co-glycolic acid)/poly(ethylene glycol) blends for controlled drug delivery
R. Cleek (1997)
10.1089/OLI.1.2000.10.11
In vivo evaluation of a morpholino antisense oligomer directed against tumor necrosis factor-alpha.
G. Qin (2000)
10.1016/S0076-6879(00)13023-X
Preparation and application of liposome-incorporated oligodeoxynucleotides.
A. Tari (2000)
10.1016/0014-5793(90)81016-H
Amphipathic polyethyleneglycols effectively prolong the circulation time of liposomes
A. Klibanov (1990)



This paper is referenced by
10.1124/pr.55.1.5
Interleukins in Atherosclerosis: Molecular Pathways and Therapeutic Potential
J. H. von der Thüsen (2003)
10.2174/1566523052997541
Synthetic hammerhead ribozymes as therapeutic tools to control disease genes.
L. Citti (2005)
10.3390/v4040488
Nanotechnology and the Treatment of HIV Infection
R. Parboosing (2012)
10.2174/138161207780162999
Pharmaceutical and Biomedical Potential of PEGylated Dendrimers
V. Gajbhiye (2007)
10.1517/14728222.5.3.363
Telomerase - strategies to exploit an important chemotherapeutic target
T. Fletcher (2001)
10.1016/j.biomaterials.2010.11.074
Modulation of biological processes in the nucleus by delivery of DNA oligonucleotides conjugated with gold nanoparticles.
D. Kim (2011)
10.1007/s11095-011-0514-6
Solid Lipid Nanoparticles Loaded with Anti-microRNA Oligonucleotides (AMOs) for Suppression of MicroRNA-21 Functions in Human Lung Cancer Cells
San-jun Shi (2011)
Nano-ARTand NeuroAIDS
Malay Kumar Das (2016)
10.1007/s13346-016-0293-z
Nano-ART and NeuroAIDS
M. Das (2016)
10.1016/J.BMC.2005.05.024
Oligonucleotides containing 2'-O-[2-(2,3-dihydroxypropyl)amino-2-oxoethyl]uridine as suitable precursors of 2'-aldehyde oligonucleotides for chemoselective ligation.
E. Zubin (2005)
10.1016/J.BRAINRESREV.2006.01.006
Animal models of behavioral dysfunctions: Basic concepts and classifications, and an evaluation strategy
F. J. Staay (2006)
10.4103/0971-6866.27786
Clinical relevance of alternative splicing
T. Ravindra (2006)
10.1023/B:PHAM.0000016249.52831.a5
Uptake and Cytotoxicity of Chitosan Molecules and Nanoparticles: Effects of Molecular Weight and Degree of Deacetylation
M. Huang (2004)
10.1111/j.1442-9071.2005.00952.x
Treatments for choroidal and retinal neovascularization: a focus on oligonucleotide therapy and delivery for the regulation of gene function
R. J. Marano (2005)
10.1007/s13346-012-0116-9
Inorganic nanovectors for nucleic acid delivery
Sandhya Pranatharthiharan (2012)
10.1080/10611860400003858
Sustained Polymeric Delivery of Gene Silencing Antisense ODNs, siRNA, DNAzymes and Ribozymes: In Vitro and In Vivo Studies
A. Khan (2004)
10.6000/1927-5951.2011.01.01.12
Cellular Trafficking and Subcellular Interactions of Cationic Gene Delivery Nanomaterials
J. Barar (2011)
10.1089/OLI.2007.0097
Bioconjugation of oligonucleotides for treating liver fibrosis.
Z. Ye (2007)
10.1016/S0006-2952(02)00985-1
Sequence and chemistry requirements for a novel aptameric oligonucleotide inhibitor of EGF receptor tyrosine kinase activity.
S. Akhtar (2002)
10.2165/00063030-200418010-00004
A Multi-Model Approach to Nucleic Acid-Based Drug Development
Isabelle Gautherot (2012)
10.1002/APP.32508
Controlled Size Chitosan Nanoparticles as an Efficient, Biocompatible Oligonucleotides Delivery System
R. Manchanda (2010)
10.1007/s11010-005-2912-8
Cholesterol conjugated oligonucleotide and LNA: A comparison of cellular and nuclear uptake by Hep2 cells enhanced by Streptolysin-O
Šárka Holasová (2005)
10.1021/JA031815L
Hybrid QM/MM study of thio effects in transphosphorylation reactions: the role of solvation.
Brent A. Gregersen (2004)
10.1007/978-3-642-38815-6_9
Nano-encapsulation of Oligonucleotides for Therapeutic Use
Eveline E. Salcher (2014)
23 Toxicogenomics of Nonviral Cationic Gene Delivery Nanosystems
Y. Omidi (2012)
10.1021/JA035167H
Hybrid QM/MM study of thio effects in transphosphorylation reactions.
Brent A. Gregersen (2003)
Strategies for surviving down-regulation : effects on tumour cell growth potential and chemosensitivity profile
M. Pennati (2008)
10.1080/10611860400006257
The Design and Exogenous Delivery of siRNA for Post-transcriptional Gene Silencing
Ian R Gilmore (2004)
10.5772/24861
Chemotherapy-Induced Alopecia
S. Luanpitpong (2012)
Development of purification methods for tumor targeted polyplexes applied in vivo
Julia Fahrmeir (2007)
10.32657/10356/5114
Release studies of naked DNA, polyplexes and lipoplexes from polymeric films
Ramgopal Yamini (2007)
10.1016/B978-0-08-100557-6.00004-3
Nanoparticles for DNA delivery
Manohar Mahato (2017)
See more
Semantic Scholar Logo Some data provided by SemanticScholar