Gene therapy and informed consent decision making: Nursing research directions

Biological Research for Nursing

Volumn 11, Issue 1, 2009, Pages 98-107

  Kaspar, Rita W ^a   Wills, Celia E ^a   Kaspar, Brian K ^a  

^a OHIO STATE UNIVERSITY   (United States)

Author keywords

Decision making; Gene therapy; Informed consent; Neuromuscular disorders

Indexed keywords

ADENO ASSOCIATED VIRUS; ARTICLE; CLINICAL TRIAL; DECISION MAKING; DECISION SUPPORT SYSTEM; FORECASTING; GENE THERAPY; GENE VECTOR; GENETICS; HEALTH SERVICE; HUMAN; INFORMED CONSENT; METHODOLOGY; NEUROMUSCULAR DISEASE; NURSE ATTITUDE; NURSING; NURSING RESEARCH; ORGANIZATION AND MANAGEMENT; PATIENT EDUCATION; PATIENT PARTICIPATION; PSYCHOLOGICAL ASPECT; RESEARCH SUBJECT; RISK FACTOR;

CLINICAL TRIALS AS TOPIC; DECISION MAKING; DECISION SUPPORT TECHNIQUES; DEPENDOVIRUS; FORECASTING; GENE THERAPY; GENETIC VECTORS; HEALTH SERVICES NEEDS AND DEMAND; HUMANS; INFORMED CONSENT; NEUROMUSCULAR DISEASES; NURSE'S ROLE; NURSING RESEARCH; PATIENT EDUCATION AS TOPIC; PATIENT PARTICIPATION; RESEARCH SUBJECTS; RISK FACTORS;

EID: 67949123279     PISSN: 10998004     EISSN: 15524175     Source Type: Journal    
DOI: 10.1177/1099800409333169     Document Type: Article

References (58)

1. American Society of Gene Therapy. (2008). About the society. Retrieved May 11, 2008, from http://www.asgt.org/the-society/history.php#3
2. Bainbridge, J.W., Smith, A.J., Barker, S.S., Robbie, S., Henderson, R., Balaggan, K., et al. (2008). Effect of gene therapy on visual function in Leber's congenital amaurosis. New England Journal of Medicine, 358, 2231-2239.
3. Bank, A., Dorazio, R., & Leboulch, P. (2005). A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia. Annals of the New York Academy of Sciences, 1054, 308-316.
4. Blaese, R.M., Culver, K.W., Miller, A.D., Carter, C.S., Fleisher, T., Clerici, M., et al. (1995). T lymphocyte-directed gene therapy for ADA-SCID: Initial trial results after 4 years. Science, 270, 475-480.
5. Cavazzana-Calvo, M., Hacein-Bey, S., de Saint Basile, G., Gross, F., Yvon, E., Nusbaum, P., et al. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science, 288, 669-672. (Pubitemid 30241569)
6. Chamberlin, N.L., Du, B., de Lacalle, S., & Saper, C.B. (1998). Recombinant adeno-associated virus vector: Use for transgene expression and anterograde tract tracing in the CNS. Brain Research, 793, 169-175.
7. Conley, Y.P., & Tinkle, M.B. (2007). The future of genomic nursing research. Journal of Nursing Scholarship: An Official Publication of Sigma Theta Tau International Honor Society of Nursing/Sigma Theta Tau, 39, 17-24.
8. Cotrim, A.P., & Baum, B.J. (2008). Gene therapy: Some history, applications, problems, and prospects. Toxicologic Pathology, 36, 97-103.
9. Davidson, B.L., Stein, C.S., Heth, J.A., Martins, I., Kotin, R.M., Derksen, T.A., et al. (2000). Recombinant adeno-associated virus type 2, 4, and 5 vectors: Transduction of variant cell types and regions in the mammalian central nervous system. Proceedings of the National Academy of Sciences of the United States of America, 97, 3428-3432.
10. Dawes, R.M. (1988). Rational choice in an uncertain world. San Diego, CA: Harcourt Brace Jovanovich.
11. Dodge, J.C., Haidet, A.M., Yang, W., Passini, M.A., Hester, M., Clarke, J., et al. (2008). Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. Molecular Therapy: The Journal of the American Society of Gene Therapy, 16, 1056-1064.
12. Evans, C., Ghivizzani, S., & Robbins, P. (2008). Arthritis gene therapy's first death. Arthritis Research and Therapy, 10, 110.
13. Ferrari, F., Xiao, X., McCarty, D., & Samulski, R. (1997). New developments in the generation of Ad-free, high-titer rAAV gene therapy vectors. Nature Medicine, 3, 1295-1297.
14. Foust, K., Nurre, E., Montgomery, C., Hernandez, A., Chan, C., & Kaspar, B. (2009). Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nature Biotechnology, 27, 59-65.
15. Gavrilina, T.O., McGovern, V.L., Workman, E., Crawford, T.O., Gogliotti, R.G., DiDonato, C.J., et al. (2008). Neuronal SMN expression corrects spinal muscular atrophy in severe SMA mice while muscle-specific SMN expression has no phenotypic effect. Human Molecular Genetics, 17, 1063-1075.
16. Gene Therapy Clinical Trials Worldwide. (2008). Journal of Gene Medicine. Retrieved November 10, 2008, from http://www.wiley.co.uk/genetherapy/clinical/
17. Glorioso, J.C., Mata, M., & Fink, D.J. (2003). Gene therapy for chronic pain. Current Opinion in Molecular Therapeutics, 5, 483-488.
18. Hacein-Bey-Abina, S., Garrigue, A., Wang, G.P., Soulier, J., Lim, A., Morillon, E., et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. Journal of Clinical Investigation, 118, 3132-3142.
19. Haidet, A.M., Rizo, L., Handy, C., Umapathi, P., Eagle, A., Shilling, C., et al. (2008). Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proceedings of the National Academy of Sciences of the United States of America, 105, 4318-4322.
20. Holmes-Rovner, M., & Wills, C.E. (2002). Improving informed consent: Insights from behavioral decision research. Medical Care, 40 (Suppl. 9). V30 - V38.
21. Jaff, A., Prasad, S., Larcher, V., & Hart, S. (2006). Gene therapy for children with cystic fibrosis-who has the right to choose ? Journal of Medical Ethics, 32, 361-364.
22. Jaroff, L. (1999). Fixing the genes. Time, 153, 68-70.
23. Kaspar, B.K., & Gage, F.H. (2006). Adeno associated viral vectors for the nervous system. In P. R. Lowenstein, & M. G. Castro (Eds.), Gene therapy for neurological disorders (pp. 33-38). New York: Taylor & Francis Group, LLC.
24. Kim, S.Y., Holloway, R.G., Frank, S., Beck, C.A., Zimmerman, C., Wilson, R., et al. (2006). Volunteering for early phase gene transfer research in Parkinson disease. Neurology, 66, 1010-1015.
25. Kimmelman, J. (2005). Recent developments in gene transfer: Risk and ethics. BMJ, 330, 79-82.
26. Klein, R.L., Meyer, E.M., Peel, A.L., Zolotukhin, S., Meyers, C., Muzyczka, N., et al. (1998). Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors. Experimental Neurology, 150, 183-194.
27. MacGregor, R.R. (2001). Clinical protocol. A phase 1 open-label clinical trial of the safety and tolerability of single escalating doses of autologous CD4 T cells transduced with VRX496 in HIV-positive subjects. Human Gene Therapy, 12, 2028-2029.
28. Manno, C.S., Pierce, G.F., Arruda, V.R., Glader, B., Ragni, M., Rasko, J.J., et al. (2006). Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nature Medicine, 12, 342-347.
29. Marshall, E. (2000). Gene therapy on trial. Science, 288, 951-957.
30. Mata, M., Chattopadhyay, M., & Fink, D.J. (2006). Gene therapy for the treatment of sensory neuropathy. Expert Opinion on Biological Therapy, 6, 499-507.
31. McCarty, D. (2008). Self-complementary AAV vectors: Advances and applications. Molecular Therapy, 16, 1648-1656.
32. Mendell, J.R. (2002). [Gene therapy clinical trial for limb-girdle muscular dystrophy type 2D]. Unpublished raw data.
33. Mendell, J.R. (2008). [Gene therapy clinical trial for Duchenne muscular dystrophy]. Unpublished raw data.
34. Metcalfe, K., Poll, A., O'Connor, A., Gershman, S., Armel, S., Finch, A., et al. (2007). Development and testing of a decision aid for breast cancer prevention for women with a BRCA1 or BRCA2 mutation. Clinical Genetics, 72, 208-217.
35. Miller, T., Smith, R., Kordasiewicz, H., & Kaspar, B. (2008). Gene-targeted therapies for the central nervous system. Archives of Neurology, 65, 447-451.
36. Nationwide Children's Hospital. (2008 a). Consent to participate in a clinical research study:Phase I gene transfer of AAV1.tMCK.h±SG for LGMD2D (IRB07-00329/ Ame00002169).
37. Nationwide Children's Hospital. (2008 b). Subject assent form (for subjects 9 to 18 years old). Phase I gene transfer of AAV1.tMCK.h±SG for LGMD2D (IRB07-00329/ Ame00002169).
38. NIH Office of Biotechnology Activities. (2008). Human gene transfer protocols. Retrieved January 30, 2009, from http://oba.od.nih.gov/oba/rac/ PROTOCOL.pdf
39. NIH Office of Biotechnology Activities. (2002). Guidelines for research involving recombinant DNA molecules. Retrieved June 18, 2008, from http://oba.od.nih.gov/oba/rac/guidelines-02/NIH-Gdlnes-lnk-2002z.pdf
40. O'Connor, A.M. (1995). Validation of a decisional conflict scale. Medical Decision Making: An International Journal of the Society for Medical Decision Making, 15, 25-30.
41. O'Connor, A.M., Drake, E.R., Fiset, V., Graham, I.D., Laupacis, A., & Tugwell, P. (1999). The Ottawa patient decision aids. Effective Clinical Practice: ECP, 2, 163-170.
42. O'Connor, A.M., Stacey, D., Entwistle, V., Llewellyn-Thomas, H., Rovner, D., Holmes-Rovner, M., et al. (2003). Decision aids for people facing health treatment or screening decisions. Cochrane Database of Systematic Reviews (Online), 2, CD001431.
43. O'Connor, A.M., Wennberg, J.E., Legare, F., Llewellyn-Thomas, H.A., Moulton, B.W., Sepucha, K.R., et al. (2007). Toward the 'tipping point': Decision aids and informed patient choice. Health Affairs (Project Hope), 26, 716-725.
44. Ohio State University. (1999). Consent for gene therapy in limb girdle muscular dystrophy (Protocol OSU #98H0283).
45. Ott, M.G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., et al. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nature Medicine, 12, 401-409.
46. Phillips, K., Warner, E., Meschino, W., Hunter, J., Abdolell, M., Glendon, G., et al. (2000). Perceptions of Ashkenazi Jewish breast cancer patients on genetic testing for mutations in BRCA1 and BRCA2. Clinical Genetics, 57, 376-383. (Pubitemid 30245824)
47. Raper, S.E., Chirmule, N., Lee, F.S., Wivel, N.A., Bagg, A., Gao, G.P., et al. (2003). Fatal systemic inflammatory response syndrome in an ornithine transcarbamylase deficient patient following adenoviral gene transfer. Molecular Genetics and Metabolism, 80, 148-158.
48. Redelmeier, D.A., Rozin, P., & Kahneman, D. (1993). Understanding patients' decisions. Cognitive and emotional perspectives. JAMA, 270, 72-76.
49. Reynolds, W.W., & Nelson, R.M. (2007). Risk perception and decision processes underlying informed consent to research participation. Social Science & Medicine, 65, 2105-2115.
50. Rodino-Klapac, L.R., Chicoine, L.G., Kaspar, B.K., & Mendell, J.R. (2007). Gene therapy for duchenne muscular dystrophy: Expectations and challenges. Archives of Neurology, 64, 1236-1241.
51. Rodino-Klapac, L.R., Lee, J.S., Mulligan, R.C., Clark, K.R., & Mendell, J.R. (2008). Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D. Neurology, 71, 240-247.
52. Rosenberg, S.A., Aebersold, P., Cornetta, K., Kasid, A., Morgan, R.A., Moen, R., et al. (1990). Gene transfer into humans-immunotherapy of patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral gene transduction. New England Journal of Medicine, 323, 570-578.
53. Shiloh, S., Gerad, L., & Goldman, B. (2006). Patients' information needs and decision-making processes: What can be learned from genetic counselees ? Health Psychology, 25, 211-219.
54. Thompson, L. (2000, September-October). Human gene therapy: Harsh lessons, high hopes. FDA Consumer Magazine, 34 (5). Retrieved April 15, 2008, from http:// www.fda.gov/Fdac/features/2000/500-gene.html
55. Tversky, A., & Kahneman, D. (1974). Judgment under uncertainty: Heuristics and biases. Science, 185, 1124-1131.
56. Warrington KH Jr, & Herzog, R. (2006). Treatment of human disease by adeno-associated viral gene transfer. Human Genetics, 119, 571-603.
57. Wills, C.E., & Holmes-Rovner, M. (2003). Patient comprehension of information for shared treatment decision making: State of the art and future directions. Patient Education and Counseling, 50, 285-290.
58. Worgall, S., Sondhi, D., Hackett, N.R., Kosofsky, B., Kekatpure, M.V., Neyzi, N., et al. (2008). Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Human Gene Therapy, 19, 463-474.