Oligonucleotide Therapeutics First Annual Meetingof the Oligonucleotide Therapeutics Society, Volume 1082

by Tuschl , Thomas; Rossi, John J.

• ISBN13: 9781573315876
• ISBN10: 1573315877
• Edition: 1st
• Format: Paperback
• Copyright: 2006-12-15
• Publisher: Wiley-Blackwell

Summary

This volume concentrates on the most relevant oligonucleotide research and drug development areas and emphasizes the basic mechanisms and biology of oligonucleotide reagents.Chapters are devoted to RNAi, oligonucleotide delivery, and immunomodulation. Because of the advanced stages of oligonucleotide-based drug development, preclinical and clinical data are also presented by leaders in the field.NOTE: Annals volumes are available for sale as individual books or as a journal. For information on institutional journal subscriptions, please visit www.blackwellpublishing.com/nyas.ACADEMY MEMBERS: Please contact the New York Academy of Sciences directly to place your order (www.nyas.org). Members of the New York Academy of Science receive full-text access to the Annals online and discounts on print volumes. Please visit www.nyas.org/membership/main.asp for more information about becoming a member.

Author Biography

Thomas Tuschl is a German biochemist and molecular biologist, known for his research on RNA. John J. Rossi is the editor of Oligonucleotide Therapeutics: First Annual Meetingof the Oligonucleotide Therapeutics Society, Volume 1082, published by Wiley.

Table of Contents

Preface and Overview.
By THOMAS TUSCHL AND JOHN ROSSI	xi
Part I. Delivery
Surface-Modified LPD Nanoparticles for Tumor Targeting.
By SHYH-DAR LI AND LEAF HUANG	1
Atelocollagen-Mediated Systemic DDS for Nucleic Acid Medicines.
By KOJI HANAI, FUMITAKA TAKESHITA, KIMI HONMA, SHUNJI NAGAHARA, MIHO MAEDA, YOSHIKO MINAKUCHI, AKIHIKO SANO, AND TAKAHIRO OCHIYA	9
Intracellular Delivery of Oligonucleotide Conjugates and Dendrimer Complexes.
By R.L. JULIANO	18
Mechanism of PNA Transport to the Nuclear Compartment.
By LENKA STANKOVA, AMY J. ZIEMBA, ZHANNA V. ZHILINA, AND SCOT W. EBBINGHAUS	27
Part II. Identifying and Validating Targets and Systems
TLR9 and the Recognition of Self and Non-Self Nucleic Acids.
By MARC S. LAMPHIER, CHERILYN M. SIROIS, ANJALI VERMA, DOUGLAS T. GOLENBOCK, AND EICKE LATZ	31
RNA Silencing in the Struggle against Disease.
By VOLKER PATZEL, ISABELL DIETRICH, AND STEFAN H.E. KAUFMANN	44
Cellular Dynamics of Antisense Oligonucleotides and Short Interfering RNAs.
By LI KIM LEE, BRANDEE M. DUNHAM, ZHUTING LI, AND CHARLES M. ROTH	47
Inhibition of Hepatitis C IRES-Mediated Gene Expression By Small Hairpin RNAs in Human Hepatocytes and Mice.
By HEINI ILVES, ROGER L. KASPAR, QIAN WANG, ATTILA A. SEYHAN, ALEXANDER V. VLASSOV, CHRISTOPHER H. CONTAG, DEVIN LEAKE, AND BRIAN H. JOHNSTON	52
SiRNA-Mediated Selective Inhibition of Mutant Keratin mRNAs Responsible for the Skin Disorder Pachyonychia Congenita.
By ROBYN P. HICKERSON, FRANCES J.D. SMITH, W.H. IRWIN MCLEAN, MARKUS LANDTHALER, RUDOLF E. LEUBE, AND ROGER L. KASPAR	56
Multitargeted Approach Using Antisense Oligonucleotides for the Treatment of Asthma.
By Z. ALLAKHVERDI, M. ALLAM, A. GUIMOND, N. FERRARI, K. ZEMZOUMI, R. SÉGUIN, L. PAQUET, AND P.M. RENZI	62
Therapeutic Modulation of DMD Splicing By Blocking Exonic Splicing Enhancer Sites with Antisense Oligonucleotides.
By A. AARTSMA-RUS, A.A.M. JANSON, J.A. HEEMSKERK, C.L. DE WINTER, G.-J.B. VAN OMMEN, AND J.C.T. VAN DEUTEKOM	74
Neuromuscular Therapeutics By RNA-Targeted Suppression of ACHE Gene Expression.
By AMIR DORI AND HERMONA SOREQ	77
Characterization of Antisense Oligonucleotides Comprising 2'-Deoxy-2'-Fluoro-β-D-Arabinonucleic Acid (FANA): Specificity, Potency, and Duration of Activity.
By NICOLAY FERRARI, DENIS BERGERON, ANNA-LISA TEDESCHI, MARIA M. MANGOS, LUC PAQUET, PAOLO M. RENZI, AND MASAD J. DAMHA	91
Anti-HIV Activity of Steric Block Oligonucleotides.
By GABRIELA IVANOVA, ANDREY A. ARZUMANOV, JOHN J. TURNER, SANDRINE REIGADAS, JEAN-JACQUES TOULMÉ, DOUGLAS E. BROWN, ANDREW M.L. LEVER, AND MICHAEL J. GAIT	103
Selection of Thioaptamers for Diagnostics and Therapeutics.
By XIANBIN YANG, HE WANG, DAVID W.C. BEASLEY, DAVID E. VOLK, XU ZHAO, BRUCE A. LUXON, LEE O. LOMAS, NORBERT K. HERZOG, JUDITH F. ARONSON, ALAN D.T. BARRETT, JAMES F. LEARY, AND DAVID G. GORENSTEIN	116
Modification of the Pig CFTR Gene Mediated By Small Fragment Homologous Replacement.
By ROSALIE MAURISSE, JUDY CHEUNG, JONATHAN WIDDICOMBE, AND DIETER C. GRUENERT	120
Part III. Clinical Studies
Nucleic Acid Therapeutics for Hematologic Malignancies Theoretical Considerations.
By JOANNA B. OPALINSKA, ANNA KALOTA, JYOTI CHATTOPADHYAYA, MASAD DAMHA, AND ALAN M. GEWIRTZ	124
CpG Oligonucleotides Improve the Protective Immune Response Induced By the Licensed Anthrax Vaccine.
By DENNIS M. KLINMAN, HANG XIE, AND BRUCE E. IVINS	137
Anti-VEGF Aptamer (Pegaptanib) Therapy for Ocular Vascular Diseases.
By EUGENE W.M. NG AND ANTHONY P. ADAMIS	151
RNAi in Combination with a Ribozyme and TAR Decoy for Treatment of HIV Infection in Hematopoietic Cell Gene Therapy.
By MINGJIE LI, HAITANG LI, AND JOHN J. ROSSI	172
Index of Contributors	181