12.07.2015 Views

GTMB 7 - Gene Therapy & Molecular Biology

GTMB 7 - Gene Therapy & Molecular Biology

GTMB 7 - Gene Therapy & Molecular Biology

SHOW MORE
SHOW LESS

Create successful ePaper yourself

Turn your PDF publications into a flip-book with our unique Google optimized e-Paper software.

David et al: Current status and future direction of fetal gene therapyThe decision to participate in a fetal gene therapy trialwould occur close to the time of prenatal diagnosis of thecondition. The parents may hear information in a highlybiased way and not consider the risk to future pregnancies.It will be important to ensure that parents are adequatelycounselled and understand these issues before agreeing totake part in any future research. The general publicremains concerned that ethical discussion about issuessuch as gene therapy, cloning and the Human GenomeProject are falling behind the technology (Brown, 2000). Itis therefore important to provide adequate informationwhich will allow the public to understand the risks andbenefits of these novel techniques and to enable aneducated involvement in the decision-making processalong with health professionals. This will also helpindividuals to give informed consent as these proceduresbecome used in clinical practice.VII. ConclusionsFetal gene therapy offers the potential forobstetricians and gene therapists not only to diagnose butalso to treat inherited genetic disease. However, for thetreatment to be acceptable, it must offer advantages overpostnatal gene therapy, be safe for both mother and fetus,and preferably avoid germ-line transmission. Currently, inutero gene therapy remains an experimental procedure.But in the future, better understanding of the developmentof genetic disease in the fetus, and improvements in vectordesign and targeting of fetal tissues should allow thistechnology to move into clinical practice.ReferencesAiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A,Morecki S, Andolfi G, Tabucchi A, Carlucci F, Marinello E,Cattaneo F, Vai S, Servida P, Miniero R, Roncarolo MG,Bordignon C (2002) Correction of ADA-SCID by stem cellgene therapy combined with nonmyeloablative conditioning.Science 296, 2410-2413.Akiyama M (1998) Severe congenital ichthyosis of the neonate.Int J Dermatol 37, 722-728.Anderson CC, Carroll JM, Gallucci S, Ridge JP, Cheever AW,Matzinger P (2001) Testing time-, ignorance-, and dangerbasedmodels of tolerance. J Immunol 166, 3663-3671.Badalian SS, Chao CR, Fox HE, Timor TI (1993) Fetalbreathing-related nasal fluid flow velocity in uncomplicatedpregnancies. Am J Obstet Gynecol 169, 563-567.Badawi N, Kurinczuk JJ, Keogh JM, Alessandri LM, O'SullivanF, Burton PR, Pemberton PJ, Stanley FJ (1998) Intrapartumrisk factors for newborn encephalopathy: the WesternAustralian case-control study. BMJ 317, 1549-1553.Baldeschi C, Gache Y, Rattenholl A, Bouille P, Danos O,Ortonne JP, Bruckener-Tuderman L, Meneguzzi G (2003).<strong>Gene</strong>tic correction of canine dystrophic epidermolysisbullosa mediated by retroviral vectors. Hum Mol <strong>Gene</strong>t 12,1897-1905.Barcroft J, Barron (1946) Observations upon the form andrelations of the maternal and fetal vessels in the placenta ofthe sheep. Anat Rec 94, 569-595.Barker DJ, Gluckman PD, Godfrey KM, Harding JE, Owens JA,Robinson JS (1993) Fetal nutrition and cardiovasculardisease in adult life. Lancet 341, 938-941.Baumgartner TL, Baumgartner BJ, Hudon L, Moise KJ (1999)Ultrasonographically guided direct gene transfer in utero:Successful induction of β-galactosidase in a rabbit model.Am J Obstet Gynecol 181, 848-852.Benirschke K, Kaufmann P (1990) Placental types. Pathology ofthe human placenta. Springer-Verlag, New York.Bennett M, Galan H, Owens G, Dewey R, Banks R, Hobbins J,Accurso F, Schaack J (2001) In utero gene delivery byintraamniotic injection of a retroviral vector producer cellline in a nonhuman primate model. Hum <strong>Gene</strong> Ther 12,1857-1865.Bigger B, Coutelle C (2001) Perspectives on gene therapy forcystic fibrosis airway disease. Biodrugs 15, 615-634.Billingham RE, Brent L, Medawar PB (1956) Quantitativestudies on tissue transplantation immunity III Activelyacquired tolerance. Phil Trans R Soc (London) B239, 357-369.Binns R (1967) Bone marrow and lymphoid cell injection of thepig fetus resulting in transplantation tolerance or immunity,and immunoglobulin production. Nature 214, 179-180.Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T,Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P,Greenblatt JJ, Rosenberg SA, Klein H, Berger M, MullenCA, Ramsey WJ, Muul L, Morgan RA, French Anderson W(1995) T lymphocyte-directed gene therapy for ADA- SCID:initial trial results after 4 years. Science 270, 475-480.Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G,Panina P, Mazzolari E, Maggioni D, Rossi C, Servida P,Ugazio AG, Mavilio F (1995) <strong>Gene</strong> therapy in peripheralblood lymphocytes and bone marrow for ADAimmunodeficientpatients. Science 270, 470-475.Boué A, Muller F, Nezelof C, Oury JF, Duchatel F, Dumez Y,Aubry MC, Boue J (1986) Prenatal diagnosis in 200pregnancies with a 1-in-4 risk of cystic fibrosis. Hum <strong>Gene</strong>t74, 288-297.Boyle MP, Enke RA, Adams RJ, Guggino WB, Zeitlin PL (2001)In utero AAV-mediated gene transfer to rabbit pulmonaryepithelium. Mol Ther 4, 115-121.Brandt ML, Moise KJJ, Eckert JW, Johnson L, Waltrip T, SaadeG, Wu Y, Finegold MJ (1997) Transuterine puncture of thefetal stomach provides access to the small bowel in therabbit. J Invest Surg 10, 41-46.Brosens JJ, Pijnenborg R, Brosens IA ( 2002) The myometrialjunctional zone spiral arteries in normal and abnormalpregnancies: a review of the literature. Am J ObstetGynecol 187, 1416-1423.Brown P (2000) Regulations not keeping up with developmentsin genetics, says poll. BMJ 321, 1369.Buckley RH, Schiff SE, Schiff RI, Markert L, Williams LW,Roberts JL, Myers LA, Ward FE (1999) Hematopoietic stemcelltransplantation for the treatment of severe combinedimmunodeficiency. New Engl J Med 340, 508-516.Buckley SMK, Waddington SN, Jezzard S, Themis M, ColledgeWH, Coutelle C (2003) Intra-amniotic application of CFTRexpressingadenovirus does not reverse cystic fibrosisphenotype in inbred Cftr-knockout mice. Mol Ther 7, S200.Burger IM, Wilfond BS (2000) Limitations of informed consentfor in utero gene transfer research: implications forinvestigators and institutional review boards. Hum <strong>Gene</strong>Ther 11, 1057-1063.Burlet P, Huber C, Bertrandy S, Ludosky MA, Zwaenepoel I,Clermont O, Roume J, Delezoide AL, Cartaud J, Munnich A,Lefebvre S (1998) The distribution of SMN protein complexin human fetal tissues and its alteration in spinal muscularatrophy. Hum Mol <strong>Gene</strong>t 7, 1927-1933.Case SS, Price MA, Jordan CT, Yu XJ, Wang L, Bauer G, HaasDL, Xu D, Stripecke R, Naldini L, Kohn DB, Crooks GM(1999) Stable transduction of quiescent CD34(+)CD38(-)human hematopoietic cells by HIV-1-based lentiviralvectors. Proc Natl Acad Sci 96, 2988-2993.202

Hooray! Your file is uploaded and ready to be published.

Saved successfully!

Ooh no, something went wrong!