Targeting of Drugs 5

TheNATO AdvancedStudiesInstituteseries"TargetingofDrugs"wasoriginatedin 1981. It is nowamajorinternationalforum,heldeverytwo yearsin CapeSounion,Greece,in whichthepresentandthefutureofthisimportantareaofresearch in drugdeliveryisdiscussed in greatdepth. PreviousASIsoftheseriesdealtwith drugcarriersofnaturalandsynthetic origin,theirinteractionswith thebiologicalmilieu, waysby whichthefunctionofdrugcarriers iscircumvented and,morerecently,with avarietyofapproaches to carrierdesignor modificationthatcontributeto optimalcarrierfunction. Thepresentbookcontainsthe proceedings ofthe8thNATO ASI, "TargetingofDrugs:Strategies for Oligonucleotideand GeneDelivery in Therapy", held in CapeSounionduring24June-5 July 1995. Asthetitle implies,thebookdealswith avarietyofsystemsin termsoftheirability to transportnucleic acidsto targetareasin vitro andin vivo in waysthateffectivelymodify,supplement, correct, orcurtailthefunctionofgenesin therapy. Weexpressourappreciation to Mrs. ConchaPerringfor herassistance with the organizationoftheASI. TheASI washeldunderthesponsorship ofNATO ScientificAffairs Division andco-sponsored andgenerouslyfinancedby SmithKlineBeechamPharmaceuticals (KingofPrussia). Financialassistance wasalsoprovidedby SandozPharma(Baseland Athens),GeneMedicine (Houston,USA), ChironCorporation(Emeryville,USA), BYK GuldenLombergChemische (Konstanz,Gernlany),HelpSA(Athens,Greece),Avanti Polar Lipids Inc (Birmingham,USA), OxfordMolecular(Oxford,UK), Pfizer(Kent,UK), andAlza Corporation(PaloAlto, USA). GregoryGregoriadis BrendaMcCormack v CONTENTS Gene Therapy for Inherited Genetic Disease: Possibilities and Problems c. ·Coutelle Gene Delivery and Therapy: The Case for Cystic Fibrosis 15 E. W. F. W. Alton Immune Responses with Direct Gene Transfer: DNA Vaccines and 21 Implications for Gene Therapy H. L. Davis Oligonucleotides: Molecular Versions for Optimal Use in Vivo 31 E. Saison-Behmoaras, A. Van Aerschot, I. Duroux, C. Hendrix, C. Helene, and P. Herdewijn Retrovirus Vectors in Gene Therapy: Targeting to Specific Cells 45 AJ. Kingsman, Y. Bae, J. c. Griffiths, N. Kim, E. E. Ramsdale, G. Romano, Y. Soneoka, P. M. Cannon, and S. M. Kingsman Adenovirus as Vectors for Gene Therapy 53 M. G. Lee Receptor-Mediated Gene Delivery with Synthetic Virus-like Particles 67 E. Wagner, M. Cotten, and K. Zatloukal Controllable Gene Therapy: Recent Advances in Non-Viral Gene Delivery 79 A.

Klappentext

Implications for Gene Therapy H. L. Davis Oligonucleotides: Molecular Versions for Optimal Use in Vivo 31 E. Saison-Behmoaras, A. Van Aerschot, I. Duroux, C. Hendrix, C. Helene, and P. Herdewijn Retrovirus Vectors in Gene Therapy: Targeting to Specific Cells 45 AJ. Kingsman, Y. Bae, J. c. Griffiths, N. Kim, E. E. Ramsdale, G. Romano, Y. Soneoka, P. M. Cannon, and S. M. Kingsman Adenovirus as Vectors for Gene Therapy 53 M. G. Lee Receptor-Mediated Gene Delivery with Synthetic Virus-like Particles 67 E. Wagner, M. Cotten, and K. Zatloukal Controllable Gene Therapy: Recent Advances in Non-Viral Gene Delivery 79 A.

Zusammenfassung
Proceedings of a NATO ASI held in Cape Sounion, Greece, June 24-July 5, 1995

Inhalt
Gene Therapy for Inherited Genetic Disease: Possibilities and Problems.- Gene Delivery and Therapy: The Case for Cystic Fibrosis.- Immune Responses with Direct Gene Transfer: DNA Vaccines and Implications for Gene Therapy.- Oligonucleotides: Molecular Versions for Optimal Use in Vivo.- Retrovirus Vectors in Gene Therapy: Targeting to Specific Cells.- Adenovirus as Vectors for Gene Therapy.- Receptor-Mediated Gene Delivery with Synthetic Virus-like Particles.- Controllable Gene Therapy: Recent Advances in Non-Viral Gene Delivery.- Genetic Chemistry: Towards Non-Enzymatic Ligation, Sequence-Selective Recognition of DNA, and Self-Assembling Systems for Gene Delivery.- Integrin-Mediated Gene Delivery.- Design, Synthesis, and Cellular Delivery of Antibody Targeted, Radiolabelled Oligonucleotide Conjugates for Cancer Therapy.- Poly(L lysine) Mediated Delivery of Nucleic Acids.- Complement Activation by Polylysine-DNA Complexes.- Nanoparticles as Carriers for Antisense Oligonucleotides.- Efficient Incorporation of Plasmid DNA within Liposomes of Varying Structural Characteristics: Liposomal DNA Integrity and Transfection Efficiency.- pH Sensitive Liposomes as Efficient Carriers for Intracellular Delivery of Oligonucleotides.- Liposomal Delivery of p-Ethoxy Antisense Oligodeoxynucleotides in Chronic Myelogenous Leukemia.- Liposome-Mediated Delivery of Genes and Oligonucleotides for the Treatment of Brain Tumors.- Enzyme Assisted Synthesis of Delivery Systems.- Participants' Photograph.- Contributors.