Cómo citar
Descargas
Métricas Alternativas
Dimensions
Resumen
La terapia génica consiste en la inserción de ADN en células humanas con fines terapéuticos. Aunque inicialmente se propuso para el tratamiento de trastornos monogénicos, su uso se extendió hacia el cáncer y enfermedades infecciosas como el SIDA. Más de 1000 ensayos clínicos han sido realizados desde 1990 y cerca de 5000 pacientes en el mundo portan en sus células ADN exógeno. La eficacia y los riesgos de la terapia génica dependen de los vehículos usados para la transferencia del material genético. En este artículo se discute el estado actual del conocimiento, perspectivas y consideraciones éticas para la implementación de esta alternativa terapéutica en el tratamiento de errores innatos del metabolismo.
Palabras clave
Citas
Amabile, P. G., J. M. Waugh, T. N. Lewis. 2001. High-efficiency endovascular gene delivery via therapeutic ultrasound. J Am Coll Cardiol 37: 1975-1980. American Society of Gene Therapy Website: http://www.asgt.org.
Bagley, J., J. Iacomini. 2003. Gene therapy progress and prospects: gene therapy in organ transplantation. Gene Ther 10: 605-611.
Barrera, LA. 2003. Desarrollo de un modelo de vectores usando virus adenoasociados libre de adenovirus para corregir la deficiencia enzimática en las mucopolisacaridosis. IV Congreso de Errores Innatos del Metabolismo y Pesquisa Neonatal. Octubre 24-27. Iguazú, Argentina.
Barrera, LA., Sáenz, H., Cuellar, YM., Ospina, S., Garzón, K., Cabrera, MA., Márquez, W., Torres, AL. 2004. Manual de Enfermedades Metabólicas. Editorial Panamericana SA. Bogotá. 250 p.
Berns, KI. Parvoviridae: The viruses and their replication in: Fields Virology. Fields, BN., Knipe, DM., Howley, PM. Lippicott – Raven Publishers. Philadelphia. 1996. pp 2173-2192.
Bossis, I., Chiorini, JA. 2003. Cloning of an avian adeno-associated (AAA V) and generation of recombinant AAAV particles. J Virol 77: 6799-6810.
Braun, SE., Aranovich, EL., Anderson, RA., Crotty, PL., McIvor, RS., Whitley, CB. 1993. Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate –2- sulfatase. Proc Natl Acad Sci USA 90:11830–11834.
Brooks, AR., Harkins, RN., Wang, P. 2004. Transcriptional silencing is associated with extensive methylation of the CMV promoter following adenoviral gene delivery to muscle. J Gene Med 6: 395-404.
Burton, EA., Glorioso, JC., Fink, DJ. 2003. Gene therapy progress and prospects: Parkinson ́s disease. Gene Ther 10: 1721-1727.
Cabrera-Salazar, MA., Novelli, E., Barranger, JA. 2002. Gene therapy for the lysosomal storage disorders. Curr Opin Mol Ther 4: 1464-8431.
Cabrera-Salazar, MA., Barranger, JA. 2003. Lysosomal Storage disorders: gene therapy. Nature Encyclopedia of the Human Genome pp. 757-766.
Cartier, N. 2001. Gene therapy strategies for X-linked adrenoleukodystrophy. Curr Opin Mol Ther 3: 357-361.
Cavazzana-Calvo, M., Hacein-Bey, S., de Saint Basile, G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J.L., Bousso, P., Deist, F.L., & Fischer, A. 2000 Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science. 288: 669-672.
Chao, H., Mansfield, GS., Bartel, RC., 2003. Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing. Nat Med 9: 1015-1019.
Cheng, SH., Smith, AE. 2003. Gene therapy progress and prospects: gene therapy of lysosomal storage disorders. Gene Ther 10: 1275-1281.
Cheng, SH., Ziegler, R., Barbon, S., Disnick, R., Schuchman, E. 2004. Efficacy of AAV-mediated expression of ASM in Niemann-Pick B mice. J Inherit Metab Dis 27(suppl 1): 162.
Collins, J., Herman, P., Schuch, C., Babgy, G. 1992. cmyc antisense oligonucleotides inhibit the colony-forming capacity of Colon 320 colonic carcinoma cells. J. Clin Invest 89: 1523-1527.
Daly, TM., Vogler, C., Levy, B. 1999. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc Natl Acad Sci USA 96: 2296-2300.
Dauty, E., Remy, JS., Blessing, T., Behr, JP. 2001. Dimerizable cationic detergents with a low cmc condense plasmid DNA into nanometric particles and transfect cells in culture. J Am Chem Soc 123: 9227–9234.
DiFerrante, NM., Nichols, BL., Jr., Donnelly, PV., Neri, G., Hrgovcic, R., Berglund, RK. 1971. Induced degradation of glycosaminoglycans in Hurler’s and Hunter’s syndromes by plasma infusion. Proc Nat Acad Sci USA 68: 303-307.
Dimitriev, IP., Kashentseva, EA., Curieh, DT. 2002. Engineering of adenovirus vectors containing heterologous peptide sequences in the C terminus of capsid protein IX. J Virol 76: 6893-6899.
Ding, Z., Harding, C., Thöny, B. 2004. State-of-the-art 2003 on PKU gene therapy. Mol Genet Metab 81: 3-8.
Dudley, RWR., Lu, Y., Gilbert, R. 2004. Sustained improvement of muscle function one year after full-length dystrophin gene transfer into mdx mice by a gutted helper-dependent adenoviral vector. Hum Gene Ther 15: 145-156.
Edelstein, ML., Adebi, MR., Wixon, J., Edelstein, RM. 2004. Gene therapy clinical trials worldwide 1989-2004 – an overview. J Gene Med 6: 597-602.
Ellinwood, NM., Vite, C., Haskins, M. 2004. Gene therapy for lysosomal storage diseases: the lessons and promise of animal models. J Gene Med 6: 481-506.
Felgner, JH., Kumar, R., Sridhar, CN., Wheeler, CJ., Tasi, YJ., Border, R., Ramsey, P., Martin, M., Felgner, PL. 1994. Enhanced gene delivery system and mechanism studies with a novel series of cationic lipid formulations. J Biol Chem 269: 2550-2561.
Fisher, KJ., Kelley, WM., Burda, JF. 1996. A novel adenovirus-adeno-associated virus hybrid vector that displays efficient rescue and delivery of the AAV genome. Hum Gene Ther 7: 2079-2087.
Fraites, TJ., Schleissing, MR., Shanely, RA. 2002. Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors. Mol Ther 5: 571-578.
Gao, C., Sands, MS., Haskins, ME., Ponder, KP. 2000. Delivery of a retroviral vector expressing human α-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice. Mol Ther 2: 233-244.
Gao, G., Alvira, MR., Wang, L. 2002. Novel adeno-associated virus from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854-11859.
García, F., Barrera, LA. 2001. La terapia génica. Innovación y ciencia 3-4: 88-97.
García-Blanco, MA. 2003. Messenger RNA reprogramming by spliceosome-mediated RNA trans-splicing. J Clin Invest 112: 475-480.
Gaspar, HB., Howe, S., Thrasher, AJ. 2003. Gene therapy progress and prospects: gene therapy for severe combined immunodeficiency. Gene Ther 10: 1999-2004.
Ghosh, A., Allamarvdasht, M., Pan, CJ., Sun, MS., Mansfield, BC., Byrne, BJ., Chou, JY. 2006. Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer. Gene Ther 13:321-329.
Griesenbach, U., Ferrari, S., Geddes, DM., Alton, EWFW. 2002. Gene therapy progress and prospects: cystic fibrosis. Gene Ther 9: 1344-1350.
Groves, KA., Barnett, SC., Franklin, RJM., Noble, M. 1993. Repair of demyelinated lesions by transplantation of purified O-2A progenitors cells. Nature 362: 453-455.
Gutiérrez, MA., Cerón F., García, F., Barrera, LA. 2001. Construcción y evaluación de un vector de expresión episomal conteniendo el cDNA de la IDS para corregir la deficiencia enzimática en fibroblastos de paciente con síndrome de Hunter. III Congreso Latinoamericano de Errores Innatos del Metabolismo y Pesquisa Neonatal. Octubre 21-24. Cartagena, Colombia.
Hacein-Bey-Abina, S., von Kallen, C., Schmidt M. 2003. A Serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348: 255-256.
Harris, JD, Lemoine, NR. 1996. Strategies for targeted gene therapy. Trends in Genet 12: 400-404.
Herweijer, H., Wolff, JA. 2003. Progress and prospects: naked DNA gene transfer and therapy. Gene Ther 10: 453-458.
Hilman, BC., Sorensen, RU. 1994. Management Options: SCIDS with Adenosine Deaminase Deficiency. Ann Allergy 72: 395-402. Journal of Gene Medicine Clinical Trials Website: www.wiley.co.uk/ wileychi/genmed.
Kamili, S., Spelbring, J., Carson, D., Krawczynski, K. 2004. Protective efficacy of hepatitis E virus DNA vaccine administered by gene gun in the cynomolgus macaque model of infection. J Infect Dis 189: 258-264.
Kay, MA., Liu, D., Hoogerbrugge, PM. 1997. Gene Therapy. Proc Natl Acad Sci USA 94: 12744-12746.
Kim, I., Józkowicz, A., Piedra, PA. 2001. Lifetime correction of genetic deficiency in mice with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA 98: 13282-13287.
Koeberl DD, Sun BD, Damodaran TV, Brown T, Millington DS, Benjamin, DK., Jr, Bird, A., Schneider, A., Hillman, S., Jackson, M., Beaty, RM., Chen, YT. 2006. Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. Gene Ther. 13: 1281-1289.
Kuriyama, S., Mitoro, A., Nakatani, T. 2000. Particle-mediated gene transfer into murine livers using a newly developed gene gun. Gene Ther 7: 1132-1136.
Lacadena, JR. 2001. Terapia Génica. Consideraciones éticas. (N.I.C.E). http://cerezopntic.mec.es/jlacaden/presenOO.html.
Lanpher, B., Brunetti-Pierri, N., Lee, B. 2006. Inborn errors of metabolism: the from Mendelian to complex diseases. Nat Rev Genet. 7: 449-460.
Li, BW., Rush, A., Zhang, SR. 2004. Antibody responses to Brugia malayi antigens induced by DNA vaccination. Filaria J 22: 1-8.
Li, S., Ma, Z. 2001. Nonviral gene therapy. Curr Gene Ther 1: 201-226.
Lim, YB. Han, SO., Kong, HU. 2000. Biodegradable polyester, poly[a-(4-aminobutyl)-Lglycolic acid], as a non-toxic gene carrier. Pharm Res 17: 811-816.
Liu, F., Huang, L. 2002. A syringe electrode device for simultaneous injection of DNA an electrotransfer. Mol Ther 5: 323-328.
Lowenstein, PR. 2004. Immunological needles in the gene therapy haystack: applying a genetic paradigm to gene therapy. Gene Ther 11: 1-3.
Mah, C., Cresawn, KO., Fraites, TJ. Jr, Pacak, CA., Lewis, MA., Zolotukhin, I., Byrne, BJ. 2002. Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors. Gene Ther. 12: 1405-1409.
Manno, CS., Chew, AJ., Hutchison, S. 2003. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 101: 2963-2972.
Marshall, J., McEachern, K., Nietupski, J., Cavanagh, J., Grabowski, Cheng, SH. 2004a. AAV-mediated gene therapy of a murine model of Gaucher disease. J Inherit Metab Dis. 27(suppl 1): 160.
Marshall, J., Ziegler, R., Barbon, C., Bercury, Cheng, SH. 2004b. Efficacy following AAV2/8-mediated expression of α-galactosidase A in Fabry mice. J Inherit Metab Dis. 27(suppl 1): 173.
Martín-Touaux, E., Puech, JP., Château., D. 2002. Muscle as a putative producer of acid α-glucosidasa for glycogenosis type II gene therapy. Hum Mol Genet 11: 1637-1645.
McKenzie, DL., Kwok, KY., Rice, KG. 2000. A potent new class of reductively activated peptide gene delivery agents. J Biol Chem 275: 9970-9977.
McKinnis, EJ., Sulzbacher S., Rutledge, JC., Sanders, J., Scott, CR. 1996. Bone marrow transplantation in Hunter syndrome. J Pediatr 129: 145-148.
McNeish, IA., Bell, SJ., Lemoine NR. 2004. Gene therapy progress and prospects: cancer gene therapy using suppressor genes. Gene Ther 11: 497-503.
Min, JJ., Gambhir, SS. 2004. Gene therapy progress and prospects: Noninvasive imaging of gene therapy in living subjects. Gene Ther 11: 115-125.
Mount, JD., Herzog, RW., Tillson, M. 2002. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-direct gene therapy. Blood 99: 2670-2675.
Nakai, H., Herzog, RW., Hagstrom, N. 1998. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver Blood 91: 4600-4607.
Nakai, H., Montini, E., Fuess S. 2003. AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat Genet 14: 297-302.
Naldini, L., Verma, IM. 2000. Lentiviral vectors. Adv Virus Res 55, 599-609.
Neufeld, EF., Muenzer, J. Mucopolysaccharidosis in: The Metabolic and Molecular Bases of Inherited Diseases. Scriver, C.R., Beaudet A.L., Sly W.S. & Valle D. (eds). McGraw-Hill. New York. 2001. 3421-3452.
Nevin, NC. 2002. What has happened to gene therapy? Eur J Pediatr 159 (Suppl 3):S240-S242.
Nicklin, SA., Buening, H., Dishart, KL. 2001. Efficient and selective AAVs-mediated gene transfer directed to human vascular endothelial cells. Mol Ther 4: 174-181.
NIH Report. 2002. Assessment of adenoviral vector safety and toxicity: report of the National Institutes of Health Recombinant DNA Advisory Committee. Hum Gene Ther 13: 3-13
Niidome, T., Huang, L. 2002. Gene therapy progress and prospects: nonviral vectors. Gene Ther 9:1647-1652.
Oh, H., Park, E., Kang, S., Jo, I., Jung, S. 2004. Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer. Pediatr Res. 56: 1-7.
Orkin, L. 1995. Report and Recommendations of the Panel to Assess the NIH Investment in Research on Gene Therapy. Distributed by the National Institutes of Health. Bethesda. MD. Available: www.nih.gov. Dec. 07.
Park, J., Murray, GJ., Limaye, A. 2003. Long-term correction of globotriaosylceramida storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer. Proc Natl Acad Sci USA 100: 3450-3454.
Ponder, KP., Melniczek, JR., Xu, L. 2003. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proc Natl Acad Sci USA 99: 13102-13107.
Puttaraju, M., DiPasquale, J., Baker, CC. 2001. Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing. Mol Therp. 4: 105-114.
Recchia, A., Parks, RJ., Lamartina, S. 1999. Site-specific integration mediated by hybrid adenovirus/adeno-associated virus vector. Proc Natl Acad Sci USA 96: 2615-2620.
Rutledge, EA., Russell, DW. 1997. Adeno-associated virus vector integration junctions. J Virol 71: 8429-8436.
Sáenz, H., Gutiérrez, MA., Barrera, LA. 2004. Terapia génica con vectores virales para el tratamiento de mucopolisacaridosis y otras enfermedades genéticas. Universitas Medica 45: 22-31.
Sands, M., Davidson, B. 2006. Gene Therapy for Lysosomal Storage Diseases. Mol Ther 13: 839-849.
Sapolsky, R. 2003a. Altering behaviour with gene transfer in the limbic system. Physiol Behav. 79: 479-490.
Sapolsky, R. 2003b. Gene therapy for psychiatric disorders. Am J Psychiatry 162: 208-220.
Savulescu, J. 2001. Harm, ethics comities and gene therapy death. J Med Ethics 27: 148-150.
Smith, A.E. 1995. Viral vectors in gene therapy. Ann Rev Microbiol 49:807-838.
Smith, JS., Keller, JR., Lohrey, NC. 1999. Redirected infection of directly biotinylated recombinant adenovirus vectors through cell surface receptors and antigens. Proc Natl Acad Sci USA 96: 8855-8860.
Somiari, S., Glasspool-Malone, J., Drabick, JJ. 2000. Theory an in vivo application of electroporative gene delivery. Mol Ther 3:249-255.
St George, JA. 2003. Gene therapy progress and prospects: adenoviral vectors. Gene Ther 10: 1135-1141.
Stein, CS., Kang, Y., Sauter SL. 2001. In Vivo treatment of hemophilia A and mucopolysacchatidosis type VII using nonprimate lentiviral vectors. Mol Ther 3: 850-856.
Sun, B., Zhang, H., Franco, LM., Brown, T., Bird, A., Schneider, A., Koeberl, DD. 2005a. Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter. Mol Ther 11: 889-898.
Sun, B., Zhang, H., Franco, LM., Young, SP., Schneider, A., Bird, A., Amalfitano, A., Chen, YT., Koeberl, DD. 2005b. Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II. Mol Ther 2005 11: 57-65.
Taniyamma, Y., Tachibana, K., Hiraoka, K. 2002. Local delivery of plasmid DNA into rat carotid artery using ultrasound. Circulation 105: 1233-1239.
Tao, W., Hangoc, G., Cooper S., Bosmeyer, HE. 2004. SDF-1α/CXCL12 enhances retroviral-mediated gene transfer into immature subsets of human and murine hematopoietic progenitor cells. Gene Ther 11: 61-69.
Templeton NS and Lasic D. 2000. Gene therapy. Therapeutic mechanisms and strategies. Marcel Dekker, Inc. New York. 584 p.
Themis, M., May, D., Coutelle, C., Newbold, RF. 2003. Mutational effects of retrovirus insertion on the genome of V79 cells by an attenuated retrovirus vector: implications for gene therapy. Gene Ther 10: 1703-1711.
VandenDriessche, T., Vanslembrouck, V., Goovaerts, I. 1999. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc Natl Acad Sci USA 96: 10379-10384.
Vargas, J., Gusella, GL., Najfeld, V. 2004. Novel integrase-defective lentiviral episomal vectors for gene transfer. Hum Gene Ther 15: 361-372.
Volpers, C., Kochanek, S. 2004. Adenoviral vectors for gene transfer and therapy. J Gene Med 6: S164-S171.
Volpers, C., Thirion, C., Biermann, V. 2003. Antibody-mediated targeting of an adenovirus vector modified to contain a synthetic immunoglobulin g-binding domain in the capsid. J Virol 77: 2093-2104.
Vorburger, SA., Hunt, KK. 2002. Adenoviral gene therapy. The Oncologist. 7: 46-59.
Wagner, E., Cotten, M., Foisner, R., Birnstiel, ML. 1991. Transferrin-polycation-DNA complexes: the effect of polycations on the structure of the complex and DNA delivery to cells. Proc Natl Acad Sci USA 88: 4255-4259.
Walsh, CE. 2003. Gene therapy progress and prospects: gene therapy for the hemophilias. Gene Ther 10: 999-1003.
Walters, L. 1999. Ethical issues in human gene therapy. Human Genome News 10: 16-17.
Walters, L., Palmer, JG. 1997. The ethics of human gene therapy. Oxford University Press, New York. pp. 60-98.
Wheeler, CJ., Felgner, PL., Tsai, YJ., Marshall, J., Sukhu, L., Soeun, GH., Hartikka, J., Nietupski, J., Manthorpe, M., Nichols, M., Plewe, M., Liang, X., Norman, J., Smith, A., Cheng, SH. 1996. A novel cationic lipid greatly enhances plasmid delivery and expression in mouse lung. Proc Natl Acad Sci USA 93: 11454-11459.
Wickham, TJ., Segal, DM., Roelvink, PW. 1996. Targeted adenovirus gene transfer to endothelial and smooth muscle cells by using biospecific antibodies. J Virol 70: 6831-6838.
Widera, G., Austin, M., Rabussay, D. 2000. Increased DNA vaccine delivery and immunogenicity by electroporation in vivo. J Immunol 164: 463-4640.
Wightman, L., Kischeis, R., Rössler, V. 2001. Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J Gene Med 3: 362-372.
Xiao, X., Li, J., Samulski, J. 1998. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol. 72: 2224-2232.
Zarnitsyn, VG., Prausnitz, MR. 2004. Physical parameters influencing optimization of ultrasound-mediated DNA transfection. Ultrasound Med Biol 30: 527-538.
Zhou, X., Nicholas, M. 1998. In vitro packaging of adeno-associated virus DNA. J Virol 72: 3241-3247.
Zhu, W., Thomas, CE., Sparling, PF. 2004. DNA Immunization of mice with a plasmid encoding Neisseria gonorrhea PorB protein by intramuscular injection and epidermal particle bombardment. Vaccine 22: 661-670.
Esta obra está bajo una licencia internacional Creative Commons Atribución-NoComercial-SinDerivadas 4.0.
Derechos de autor 2023 https://creativecommons.org/licenses/by-nc-nd/4.0