How does US NIH regulate gene therapy study?
A. NIH provides guidelines for investigators & institutions (such as universities & hospitals) to follow when conducting clinical trials with gene therapy.
B. These guidelines state that clinical trials at institutions receiving NIH funding for this type of research must be registered with the NIH Office of Biotechnology Activities.
C. The protocol, or plan, for each clinical trial is then reviewed by the NIH Recombinant DNA Advisory Committee (RAC) to determine whether it raises medical, ethical, or safety issues that warrant further discussion at one of the RAC's public meetings.
D. NIH does not regulate gene therapy trials that are not funded by NIH.
Whichofthefollowingstatementson"NIHGuidelinesforResearchInvolvingRecombinantorSyntheticNucleicAcidMolecules(2016)"arecorrect?
A. Theguidelinesdetailsafetypracticesandcontainmentproceduresforbasicandclinicalresearchinvolvingrecombinantorsyntheticnucleicacidmolecules,includingthecreationanduseoforganismsandvirusescontainingrecombinantorsyntheticnucleicacidmolecules.
B. ThepurposeoftheNIHGuidelinesistospecifythepracticesforconstructingandhandling:(i)recombinantnucleicacidmolecules,(ii)syntheticnucleicacidmolecules,includingthosethatarechemicallyorotherwisemodifiedbutcanbasepairwithnaturallyoccurringnucleicacidmolecules,and(iii)cells,organisms,andvirusescontainingsuchmolecules.
C. InthecontextoftheNIHGuidelines,recombinantandsyntheticnucleicacidsaredefinedas:(i)moleculesthata)areconstructedbyjoiningnucleicacidmoleculesandb)thatcanreplicateinalivingcell,i.e.,recombinantnucleicacids;(ii)nucleicacidmoleculesthatarechemicallyorbyothermeanssynthesizedoramplified,includingthosethatarechemicallyorotherwisemodifiedbutcanbasepairwithnaturallyoccurringnucleicacidmolecules,i.e.,syntheticnucleicacids,or(iii)moleculesthatresultfromthereplicationofthosedescribedin(i)or(ii)above.
D. ForaclinicaltrialsitethatisaddedafterthecompletionoftheNIHprotocolregistrationprocess,noparticipantsshouldbeenrolledattheclinicaltrialsiteuntilIBCapprovalandIRBapprovalfromthatsitehavebeenobtained.
Whichofthefollowingstatementsongenetherapyarecorrect?
A. Twodecadesaftertheinitialgenetherapytrialsandmorethan2,000approvedclinicaltrialsworldwidewenotonlyhavegainedmuchnewinformationandknowledgeregardinggenetherapyingeneral,butalsolearnedtounderstandtheconcernthathaspersistedinsociety.
B. Nevertheless,ourknowledgecontinuestogrowandduringthecourseoftimemoresafetydatahasbecomeavailablethathelpsustodevelopbettergenetherapyapproaches.Also,withtheincreasedunderstandingofmolecularmedicine,wehavebeenabletodevelopmorespecificandefficientgenetransfervectorswhicharenowproducingclinicalresults.
C. Despitethesetbacksgenetherapyhasfaced,successstorieshaveincreasinglyemerged.Withoutdoubt,theethicalaspectsregardinggenetherapyneedtobeaddressedthesamewayasthequestionabouttheirefficacyandsafety.
D. Weneedtoacknowledgethefearofsomepatientsinrespecttogenetherapy.Ultimatelyitisthepatientwhodecides,whethershe/hewantstobetreatedwithgenetherapy.
Whyshouldweengagethepublicwhenarguingaboutgermlinegeneediting?
A. Ethicalandsocialvaluesregardinggermlinegenomeeditingneedtobesolicitedandconsidered.
B. Therearethreegeneralapproachestoaddressingtheethicaljustificationandstakeholderassessmentofgermlinegenomeediting:conductingprimaryresearch;conductingsecondaryanalysesofpublishedliteratureontheperceptions,acceptability,qualityoflife,attitudes,orvaluesofstakeholders;andcommissioninganexpertreview.
C. Engagingbroaderstakeholdergroups,includingthemedicalandscientificcommunities,personsandfamiliesdealingwithgeneticallybaseddisabilities,andthegeneralpublic,wouldbewarrantedgiventhepotentialusesandimpactsofgermlinegenome-editingtechnology.
D. Thesedebatesandengagementsshouldweightherisks,benefits,alternatives,unknownconsequences,andaccess,aswellasdistributiveandproceduraljustice,bothonasocietallevel(acrossandwithinsocieties)andonanindividualorcommunitybasis.Ultimately,thesedebatesandengagementswillinformtheframeworkstoenableethicalusesofthetechnologywhileprohibitingunethicalones.
How does the European Medicines Agency (EMA) define gene therapy medicinal products (GTMPs)?
A. EMA defines "gene therapy medicinal products (GTMPs) generally consist of a vector or delivery formulation/system containing a genetic construct engineered to express a specific transgene ('therapeutic sequence') for the regulation, repair, replacement, addition or deletion of a genetic sequence. The active substance is the nucleic acid sequence(s), or genetically modified microorganism(s), virus(es) or cells.
B. EMA defines that a gene therapy medicinal product is a biological medicinal product.
C. EMA defines that a gene therapy medicinal product fulfills the following two characteristics: (a) it contains an active substance which contains or consists of a recombinant nucleic acid used in or administered to human beings with a view to regulating, repairing, replacing, adding or deleting a genetic sequence; (b) its therapeutic, prophylactic or diagnostic effect relates directly to the recombinant nucleic acid sequence it contains, or to the product of genetic expression of this sequence.
D. Gene therapy medicinal products shall not include vaccines against infectious diseases.
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