CoChat
Sign Up
Cited by 554

The DOI matches its Crossref record; 5 of the 5 references it cites were found in a registry or an index.

Gene therapy: trials and tribulations

Somia, Nikunj, Verma, Inder M.·2000·Nature Reviews Genetics
DOI 10.1038/35038533
Open this paper in CoChat — free account

Abstract

The art and science of gene therapy has received much attention of late. The tragic death of 18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the successful treatment of three children suffering from a rare but fatal immunological disease. In the light of the success and tragedy, it is timely to consider the challenges faced by gene therapy--a novel form of molecular medicine that may be poised to have an important impact on human health in the new millennium.

Overview

Integrity signals

Registry

Crossref · 10.1038/35038533

Cites

5 of 5 references verified0 citation errors · 0 not checkable

Statistics

Recomputing p-values needs the paper’s full text.

Verification

Claims the paper makes

This paper’s full text isn’t openly available, so its claims and self-check weren’t run. The registry and citation checks still apply.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Citations

Cites

Show all 5 references

Research works printed but not linked: 72

  1. Li, S. & Huang, L. Nonviral gene therapy: promises and challenges. Gene Ther. 7, 31– 34 (2000). · DOI
  2. Templeton, N. S. & Lasic, D. D. (eds) Gene Therapy: Therapeutic Mechanisms and Strategies (Marcel Dekker, Inc., New York, 2000).This book has several excellent chapters on viral and non-viral vectors written by experts in the field. It also describes a number of therapeutic approaches to diseases and requirements for regulatory affairs. · DOI
  3. Friedmann, T. (ed.) The Development of Human Gene Therapy (Cold Spring Harbor Laboratory Press, New York, 1999).
  4. Verma, I. M. Gene therapy. Sci. Am. 263, 68– 72 (1990). · DOI
  5. Anderson, W. F. Human gene therapy. Nature 392, 25– 30 (1998). · DOI
  6. Mulligan, R. C. The basic science of gene therapy. Science 260, 926–932 (1993). · DOI
  7. Miller, A. D. Human gene therapy comes of age. Nature 357, 455–460 (1992). · DOI
  8. Markowitz, D., Goff, S. & Bank, A. A safe packaging line for gene transfer: separating viral genes on two different plasmids. J. Virol. 62, 1120– 1124 (1988). · DOI
  9. Danos, O. & Mulligan, R. C. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl Acad. Sci. USA 85, 6460– 6464 (1988). · DOI
  10. Burns, J. C., Friedmann, T., Driever, W., Burrascano, M. & Yee, J. K. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and non-mammalian cells. Proc. Natl Acad. Sci. USA 90, 8033–8037 (1993).Use of vesicular stomatitis virus glycoprotein (VSVG) to replace the envelope protein in MLV vectors. The resulting chimeric virus, containing VSVG protein, is pantropic and can be concentrated by ultracentrifugation. · DOI · Paper page
  11. Yu, S. F. et al. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl Acad. Sci. USA 83, 3194–3198 (1986). · DOI
  12. Morgan, R. A. & Anderson, W. F. Human gene therapy. Annu. Rev. Biochem. 62, 191–217 (1993). · DOI
  13. St Louis, D. & Verma, I. M. An alternative approach to somatic cell gene therapy. Proc. Natl Acad. Sci. USA 85, 3150–3154 (1988). On transplantation of retrovirally-transduced mouse fibroblasts, producing and secreting human factor IX protein, the transcription of the transgene is `shut off'. · DOI
  14. Palmer, T. D., Rosman, G. J., Osborne, W. R. & Miller, A. D. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc. Natl Acad. Sci. USA 88, 1330–1334 ( 1991). · DOI
  15. Lewis, P., Hensel, M. & Emerman, M. Human immunodeficiency virus infection of cells arrested in the cell cycle. EMBO J. 11, 3053– 3058 (1992). · DOI
  16. Bukrinsky, M. I. et al. A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365, 666–669 (1993). · DOI
  17. Frankel, A. D. & Young, J. A. HIV-1: fifteen proteins and an RNA. Annu. Rev. Biochem. 67, 1–25 (1998). · DOI
  18. Kafri, T., Blomer, U., Peterson, D. A., Gage, F. H. & Verma, I. M. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nature Genet. 17, 314–317 ( 1997). · DOI
  19. Zennou, V. et al. HIV-1 genome nuclear import is mediated by a central DNA flap . Cell 101, 173–185 (2000). · DOI
  20. Follenzi, A., Ailles, L. E., Bakovic, S., Geuna, M. & Naldini, L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences . Nature Genet. 25, 217– 222 (2000). · DOI
  21. Zufferey, R., Donello, J. E., Trono, D. & Hope, T. J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2886–2892 ( 1999). · DOI
  22. Miyoshi, H., Blomer, U., Takahashi, M., Gage, F. H. & Verma, I. M. Development of a self-inactivating lentivirus vector. J. Virol. 72, 8150– 8157 (1998). · DOI · Paper page
  23. Zufferey, R. et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873–9880 (1998). · DOI · Paper page
  24. Johnston, J. C. et al. Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors. J. Virol. 73, 4991–5000 (1999). · DOI
  25. Schnell, T., Foley, P., Wirth, M., Munch, J. & Uberla, K. Development of a self-inactivating, minimal lentivirus vector based on simian immunodeficiency virus. Hum. Gene Ther. 11, 439–447 ( 2000). · DOI
  26. Poeschla, E. M., Wong-Staal, F. & Looney, D. J. Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nature Med. 4, 354–357 ( 1998). · DOI
  27. Mitrophanous, K. et al. Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Ther. 6, 1808– 1818 (1999). · DOI
  28. Carter, P. J. & Samulski, R. J. Adeno-associated viral vectors as gene delivery vehicles. Int. J. Mol. Med. 6, 17–27 (2000).
  29. Berns, K. I. in Fields Virology (eds Fields, B. N., Knipe, D. M. & Howley P. M.,) 2173–2198 (Lippincott–Raven, Philadelphia, 1996).
  30. Kotin, R. M. et al. Site-specific integration by adeno-associated virus. Proc. Natl Acad. Sci. USA 87, 2211– 2215 (1990). · DOI
  31. Inoue, N., Hirata, R. K. & Russell, D. W. High-fidelity correction of mutations at multiple chromosomal positions by adeno-associated virus vectors. J. Virol. 73, 7376–7380 ( 1999). · DOI · Paper page
  32. Summerford, C. & Samulski, R. J. Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions. J. Virol. 72, 1438– 1445 (1998). · DOI
  33. Girod, A. et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nature Med. 5, 1052–1056 (1999). · DOI · Paper page
  34. Malik, P. et al. Recombinant adeno-associated virus mediates a high level of gene transfer but less efficient integration in the K562 human hematopoietic cell line. J. Virol. 71, 1776– 1783 (1997). · DOI
  35. Kay, M. A. et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nature Genet. 24, 257–261 ( 2000).Gives the first hints of successful gene therapy in haemophilia B patients by using recombinant adeno-associated viral vectors. Expression of transduced factor IX could be detected in one patient. · DOI
  36. Nakai, H., Storm, T. A. & Kay, M. A. Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nature Biotechnol. 18, 527–532 (2000). · DOI · Paper page
  37. Shenk, T. in Fields Virology (eds Fields, B. N., Knipe D. M. & Howley, P. M.) 2111–2148 (Lippincott–Raven, Philadelphia, 1996).
  38. Yeh, P. & Perricaudet, M. Advances in adenoviral vectors: from genetic engineering to their biology. FASEB J. 11, 615–623 (1997). · DOI
  39. Kochanek, S. et al. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc. Natl Acad. Sci. USA 93, 5731–5736 (1996). · DOI
  40. Parks, R. J. et al. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc. Natl Acad. Sci. USA 93, 13565– 13570 (1996).References 42 and 43 describe the generation of new adenoviral, `gutless', vectors in which all the viral genes required for viral propagation are provided in trans . The gutless vectors show long-term expression of the transgene. · DOI · Paper page
  41. Morral, N. et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc. Natl Acad. Sci. USA 96, 12816–12821 (1999). · DOI
  42. Dai, Y. et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl Acad. Sci. USA 92, 1401–1405 (1995). · DOI
  43. Kafri, T. et al. Cellular immune response to adenoviral vector infected cells does not require de novo viral gene expression: implications for gene therapy. Proc. Natl Acad. Sci. USA 95, 11377 –11382 (1998).Even physically inactivated adenoviral particles can generate a cytotoxic T-cell response, raising concerns about adenoviral vectors as suitable tools for long-term gene therapy. · DOI
  44. Tripathy, S. K., Black, H. B., Goldwasser, E. & Leiden, J. M. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med. 2, 545–550 ( 1996). · DOI
  45. Wagner, R. R., & Rose, J. K. in Fields Virology (eds Fields, B. N., Knipe D. M. & Howley, P. M.) 1121– 1136 (Lippincott–Raven, Philadelphia, 1996).
  46. Halbert, C. L., Rutledge, E. A., Allen, J. M., Russell, D. W. & Miller, A. D. Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J. Virol. 74, 1524–1532 (2000). · DOI · Paper page
  47. Fields, P. A. et al. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. Mol. Ther. 1, 225–235 ( 2000). · DOI
  48. Xiao, W. et al. Route of adminstration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol. Ther. 1, 323–329 ( 2000). · DOI
  49. Wang, L., Takabe, K., Bidlingmaier, S. M., Ill, C. R. & Verma, I. M. Sustained correction of bleeding disorder in hemophilia B mice by gene therapy. Proc. Natl Acad. Sci. USA 96, 3906–3910 ( 1999). · DOI
  50. Scriver, C. R. S., Beaudet, A. L., Sly, W. S. & Valle, D. V. (eds) The Metabolic Basis of Inherited Disease (McGraw–Hill, New York, 1989).
  51. Cavazzana-Calvo, M. et al. Gene therapy of human severe combined immunodeficiency (SCID)-XI disease. Science 288, 669– 672 (2000).The first definitive example of successful gene therapy, in three children suffering from SCID-XI. The haematopoietic stem cells from the patients were transduced by recombinant retroviruses expressing the γc–subunit, which is common to many interleukin receptors. · DOI
  52. Kohn, D. B. et al. T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Nature Med. 4, 775–780 (1998). · DOI
  53. Blaese, R. M. et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4 years. Science 270, 475– 480 (1995). · DOI
  54. Bordignon, C. et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270, 470–475 (1995). · DOI
  55. Miyoshi, H., Smith, K. A., Mosier, D. E., Verma, I. M. & Torbett, B. E. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283, 682– 686 (1999). · DOI
  56. Guenechea, G. et al. Transduction of human CD34+CD38− bone marrow and cord-derived SCID-repopulating cells with third-generation lentiviral vectors. Mol. Ther. 1, 566– 573 (2000).References 59 and 60 show successful long-term transduction of human haematopoietic stem cells by lentiviral vectors, without the use of agents such as growth factors and cytokines. · DOI
  57. Snyder, R. O. et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270–276 (1997). · DOI
  58. Wang, L., Nichols, T. C., Read, M. S., Bellinger, D. A. & Verma, I. M. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol. Ther. 1, 154– 158 (2000). · DOI
  59. May, C. et al. Therapeutic haemoglobin synthesis in thalassaemic mice expressing lentivirus-encoded human-globin. Nature 406, 82–86 (2000). · DOI · Paper page
  60. Cosset, F. L. & Russell, S. J. Targeting retrovirus entry. Gene Ther. 3, 946–956 ( 1996).
  61. Wickham, T. J. Targeting adenovirus. Gene Ther. 7, 110– 114 (2000). · DOI
  62. Boerger, A. L., Snitkovsky, S. & Young, J. A. Retroviral vectors preloaded with a viral receptor-ligand bridge protein are targeted to specific cell types. Proc. Natl Acad. Sci. USA 96, 9867–9872 (1999). · DOI
  63. Bartlett, J. S., Kleinschmidt, J., Boucher, R. C. & Samulski, R. J. Targeted adeno-associated virus vector transduction of nonpermissive cells mediated by a bispecific F(ab'γ)2 antibody. Nature Biotechnol. 17, 181–186 ( 1999); erratum 17, 393 (1999). · DOI
  64. Urlinger, S. et al. Exploring the sequence space for tetracycline-dependent transcriptional activators: Novel mutations yield expanded range and sensitivity. Proc. Natl Acad. Sci. USA 97, 7963– 7968 (2000). · DOI · Paper page
  65. Maxwell, I. H., Spitzer, A. L., Long, C. J. & Maxwell, F. Autonomous parvovirus transduction of a gene under control of tissue-specific or inducible promoters. Gene Ther. 3, 28 –36 (1996).
  66. Kafri, T., Van Praag, H., Gage, F. H. & Verma, I. M. Lentiviral vectors: Regulated gene expression. Mol. Ther. 1, 516–521 (2000). · DOI
  67. Wang, Y., O'Malley, B. W. Jr, Tsai, S. Y. & O'Malley, B. W. A regulatory system for use in gene transfer. Proc. Natl Acad. Sci. USA 91, 8180– 8184 (1994). · DOI
  68. No, D., Yao, T. P. & Evans, R. M. Ecdysone-inducible gene expression in mammalian cells and transgenic mice. Proc. Natl Acad. Sci. USA 93, 3346–3351 (1996). · DOI
  69. Suhr, S. T., Gil, E. B., Senut, M. C. & Gage, F. H. High level transactivation by a modified Bombyx ecdysone receptor in mammalian cells without exogenous retinoid X receptor. Proc. Natl Acad. Sci. USA 95, 7999–8004 (1998). · DOI
  70. Spencer, D. M., Wandless, T. J., Schreiber, S. L. & Crabtree, G. R. Controlling signal transduction with synthetic ligands. Science 262, 1019–1024 ( 1993). · DOI
  71. Ye, X. et al. Regulated delivery of therapeutic proteins after in vivo somatic cell gene transfer. Science 283, 88–91 (1999). · DOI
  72. Miyoshi, H., Takahashi, M., Gage, F. H. & Verma, I. M. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc. Natl Acad. Sci. USA 16, 10319 –10323 (1997). · DOI

Cited by

Sorted by citations

Metadata

Authors
Somia, N., & Verma, I. M.
Venue
Nature Reviews Genetics
Publisher
Springer Science and Business Media LLC
Published
2000-11
Volume
1
Issue
2
Pages
91-99
ISSN
  • 1471-0056
  • 1471-0064
DOI
10.1038/35038533
Cited by
554
References
5
Language
en
Licence
www.springer.com/tdm (tdm)

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review