A Look to Future Directions in Gene Therapy Research for Monogenic Diseases 英文参考文献.docVIP

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A Look to Future Directions in Gene Therapy Research for Monogenic Diseases 英文参考文献.doc

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A Look to Future Directions in Gene Therapy Research for Monogenic Diseases 英文参考文献

Reviews ALooktoFutureDirectionsinGeneTherapy ResearchforMonogenicDiseases Matthew H.Porteus*,Jon P.Connelly, Shondra M. Pruett ABSTRACT adeno-associatedviruses,herpessimplexviruses,and retrovirus(includingfoamyandlentiviruses).Athorough discussionofthesevectordeliverysystemsisbeyondthe scopeofthisreview,andcomprehensivediscussionsofthe differentviralvectorsusedingenetherapycanbefound elsewhere[2–4]. T heconceptofgenetherapyhaslongappealedto biomedicalresearchersandcliniciansbecauseit promisedtotreatcertaindiseasesattheirorigins.In thelastseveralyears,therehavebeenseveraltrialsinwhich patientshavebene?tedfromgenetherapyprotocols.This progress,however,hasrevealedimportantproblems, includingtheproblemofinsertionaloncogenesis.Inthis review,whichfocusesonmonogenicdiseases,wediscussthe problemofinsertionaloncogenesisandidentifyareasfor futureresearch,suchasdevelopingmorequantitativeassays forriskandef?cacy,andwaysofminimizingthegenotoxic effectsofgenetherapyprotocols,whichwillbeimportantif genetherapyistoful?llitsconceptualpromise. SummaryofRecentClinicalTrialsofGeneTherapy forMonogenicDiseases Inthelastdecadetherehavebeenfourmonogenicdiseases forwhichseminalgenetherapytrialshavebeenconducted: ornithinetranscarbamylase(OTC)de?ciency,hemophilia causedbyfactorIXde?ciency,severecombined immunode?ciency(SCID),andchronicgranulomatous disease(CGD),theresultsofwhicharebrie?ysummarizedin Table1[5–12].Signi?cantattentionhasbeenpaidtothe seriousadverseeventsthathavecomefromthesetrials,and infactweusetheseadverseeventsasabasisforformulating futureareasofresearchingenetherapy.Itmustbe Introduction Withtherecognitionthatthousandsofdiseasesarecaused bychangesinsinglegenes,so-calledmonogenicdiseases,and theadventofrecombinantDNAtechnology,theideathatone couldmanipulatethenucleicacidcontentofdiseasedcellsto curedisease,‘‘genetherapy,’’wasborn.In1978,David Baltimoresummarizedthepromiseofgenetherapyandthe optimismthatmanyfelt: highlighted,however,thatanincreasingnumberofpatients, partic