The major risks for gene therapy include:
A. Gene therapy can trigger the immune response in the patient, that could be harmful to them. Retroviruses has raised skepticism about their safety. Deactivated viruses are really deactivated?
B. Viral vectors may cause toxicity, infection, inflammation, etc.
C. Gene therapy may cause off-target effects— there is a risk that the new gene will insert itself into the path of another gene, disrupting its activity, or cause insertional mutagenesis.
D. Gene therapy may cause chronic granulomatous disease and cancer.
Ethicalissues and concernswithgenetherapyare:
A. Howcan"good" and "bad"usesofgenetherapybedistinguished?Whatactuallyhappenswhenthingsgowrong?Whodecideswhichtraitsarenormal&whichconstituteadisabilityordisorder(decidingproperindications)?
B. Dowehaveenoughknowledgetomaketherightdecisions?Areweabletocontrolgenetherapy?Aretheresituations,whengenetherapyisethicallymoreacceptable?Whatarethecostsforthistypeoftherapyandwhoispayingforit?Willthehighcostsofgenetherapymakeitavailableonlytothewealthy?
Couldthewidespreaduseofgenetherapymakesocietylessacceptingofpeoplewhoaredifferent?Whatisthesocioeconomicimpactofgenetherapyinoursociety?
D. Shouldpeoplebeallowedtousegenetherapytoenhancebasichumantraitssuchasheight,intelligence(IQ),orathleticability(socalledgeneticenhancement)?Whatisthepotentialimpactofgermlinegenetransferonfuturegenerations?
Whatarethemajorconcernswithgermlinegeneediting?
A. Someoftheconcernsraisedaboutsomaticgenetransferarerelatedtothepossibilitythatitcouldinadvertentlyleadtogermlinegenetransfer.Germlinegenetransferatthegamete and zygotestageshavesignificantrisks.
Beyondthemedicalriskstothepotentialchild,anumberoflong-standingethicalconcernsexistregardingthepossiblepracticeofgermlinegenetransferinbothhuman and nonhumancases.
Concernsinvolveissuesrangingfromtheautonomyoffutureindividualstodistributivejustice,fairness, and theapplicationofthesetechnologiesto"enhancement".Concernsrelatedtoeugenicsandsocialjusticeandequalaccesstotechnologies.
D. Unequalaccessandculturaldifferencesaffectinguptakecouldcreatelargedifferencesintherelativeincidenceofagivenconditionbyregion,ethnicgroup,orsocioeconomicstatus.
Which of the following statements aboutsomatic cell gene therapy (SCGT) are correct?
A. In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell.
B. Any such modifications affect the individual patient only, and are not inherited by offspring.
C. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the genome or as an external episome or plasmid) is used to treat disease.
D. Most focus on severe genetic disorders, including immunodeficiencies, hemophilia, thalassemia, and cystic fibrosis. Such single gene disorders are good candidates for somatic cell therapy.
Which of the following statements about germline gene therapy (GGT) are correct?
A. GGT involves the introduction of new genetic material into either germ cells (sperm or oocytes) or into zygotes in vitro prior to fertilization or implantation.
B. In GGT, germ cells (sperm or egg cells) are modified by the introduction of functional genes into their genomes.
C. Modifying a germ cell causes all the organism's cells to contain the modified gene. The change is therefore heritable and passed on to later generations.
D. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations and higher risks versus somatic cell gene therapy (SCGT).
微信支付 
支付宝支付