Targeting of Drugs 5: Strategies for Oligonucleotide and Gene Delivery in Therapy

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.