The promise of gene therapy in otolaryngology - head and neck surgery

Current Opinion in Otolaryngology and Head and Neck Surgery

Volumn 5, Issue 3, 1997, Pages 197-202

  Lalwani, Anil K ^a   Han, Jay J ^a   Carvalho, Gerard J ^a   Mhatre, Anand N ^a  

^a Laboratory of Molecular Otology   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CARDIOVASCULAR DISEASE; CENTRAL NERVOUS SYSTEM DISEASE; CLINICAL TRIAL; CYSTIC FIBROSIS; GENE THERAPY; HEAD CANCER; HEARING DISORDER; HUMAN; INFECTION; NECK CANCER; OTORHINOLARYNGOLOGY; PRIORITY JOURNAL; RENDU OSLER WEBER DISEASE; REVIEW; SUICIDE GENE; TUMOR SUPPRESSOR GENE;

EID: 0030794290     PISSN: 10689508     EISSN: None     Source Type: Journal    
DOI: 10.1097/00020840-199706000-00010     Document Type: Review

References (49)

1. Parkman R, Gelfand E: Severe combined immunodeficiency disease, adenosinedeaminase deficiency and gene therapy. Curr Opin Immunol 1991, 3:547-551.
2. Dorudi S, Northover J, Vile R: Gene transfer therapy in cancer. Br J Surg 1993, 80:566-572.
3. Cournoyer D, Caskey C: Gene thrapy of the immune system. Ann Rev Immunol 1993, 11:297-329.
4. Nabel E, Pompili V, Plautz G, Nabel G: Gene transfer and vascular disease. Cardiovasc Res 1994, 28:445-455.
5. Dunnett S, Svendsen C: Huntington's disease: animal models and transplantation repair. Curr Opin Neurobiol 1993, 3:790-796.
6. Ross G, Erickson R, Knorr D, Motulsky A, Parkman R, Samulski J, Strauss S, Smith B: Gene therapy in the United States: a five year status report. Hum Gene Ther 1996, 7:1781-1790. This article reviews the current status of gene therapy and clinical trials and includes a brief outline of different delivery vehicles in use for gene transfer and a summary of clinical trials categorized by protocol. This paper also provides the most current clinical data and recommendations concerning future directions of gene therapy studies.
7. O'Malley B, Ledley F: Somatic gene therapy in otolaryngology-head and neck surgery. Arch Otolaryngol Head Neck Surg 1993, 119:1191-1197.
8. O'Malley B, Finegold M, Ledley F: Autologous orthotopic thyroid follicular cell transplantation: a surgical component of ex vivo somatic gene therapy. Otolaryngol Head Neck Surg 1993, 108:51-62.
9. O'Malley B, Ledley F: Somatic gene therapy. Arch Otolaryngol Head Neck Surg 1993, 119:1100-1107.
10. Recombinant DNA advisory committee: Points to consider in the design and submission of human somatic cell gene therapy protocols. Federal Register 1990, 54:36698-36703.
11. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage F, Verma I, Trono D: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996, 272:263-267. A novel retroviral vector system based on HIV is developed. Initial results with lentivirus as a gene delivery agent shows promise as it demonstrates in vivo gene delivery and transduction of neurons.
12. Ali, M, Lemoine N, Ring C: The use of DNA viruses as vectors for gene therapy. Gene Ther 1994, 1:367-384.
13. Hanania E, Kavanagh J, Hortobagyi G, Giles R, Champlin R, Deisseroth A: Recent advances in the application of gene therapy to human disease. Am J Med 1995, 99:537-552. This comprehensive review covers recent advances in the application of genetic modification strategies to the treatment of human diseases. It includes organized summary compilation of marking trials, gene replacement trials, chemoprotection and chemotherapy sensitization trials, cancer vaccine trials, antisense oligonucleotide trials, and molecular immunotherapy trials. Contains reference tables for clinical trials, respective diseases, investigators, and institutions.
14. Zheng J, Stewart R, Gao W: Neurotrophin-4/5, brain-derived neurotrophic factor, and neurotrophin-3 promote survival of cultured vestibular ganglion neurons and protect them against neurotoxicity of ototoxins. J Neurobiol 1995, 28:330-340.
15. Boucher R: Current status of CF gene therapy. Trends Genet 1996, 12:81-84. This article briefly but thoroughly reviews the goals of cystic fibrosis gene therapy, preclinical data, results of recent human clinical trials, and directions for the future.
16. Knowles M, Hohneker K, Zhou Z, Olsen J, Noah T, Hu P, Leigh M, Engelhardt J, Edwards L, Jones K: A controlled study of adenoviralvector-mediated gene transfer in the nasal epithelium of a patient with cystic fibrosis. N Engl J Med 1995, 333:823-831.
17. McAllister K, Grogg K, Johnson D, Gallione C, Baldwin M, Jackson C, Helmbold E, Markel D, McKinnon W, Murrell J, et al.: Endoglin, a TGF-β binding protein of endothelial cells, is the gene for hereditary haemorrhagic telangiectasia type 1. Nat Genet 1994, 8:345-351.
18. McAllister K, Grogg K, Lennon F, Bowles-Biesecker B, McKinnon W, Helmbold E, Markel D, Jackson C, Guttmacher A, Pericak-Vance M, Marchuk D: Genetic herterogeneity in hereditary haemorrhagic telangiectasia: possible correlation with clinical phenotype. J Med Genet 1994, 31:927-932.
19. Vincent P, Plauchu H, Hazan J, Faure S, Weissenbach J, Godet J: A third locus for hereditary haemorrhagic telangiectasia maps to chromosome 12q. Hum Mol Genet 1995, 4:945-949.
20. Vokes E, Weichselbaum R, Lippman S, Hong W: Head and neck cancer [see comments]. N Engl J Med 1993, 328:184-194.
21. Liu T, Zhang W, Taylor D, Roth J, Goepfert H, Clayman G: Growth suppression of human head and neck cancer cells by the introduction of a wild-type p53 gene via a recombinant adenovirus. Cancer Res 1994, 54:3662-3667.
22. Clayman G, Liu T, Overholt S, Mobley S, Wang M, Janot F, Goepfert H: Gene therapy for head and neck cancer: comparing the tumor suppressor gene p53 and a cell cycle regulator WAF1/CIP1 (p21). Arch Otolaryngol Head Neck Surg 1996, 122:489-493.
23. Clayman G, El-Naggar A, Roth J, Zhang W, Goepfert H, Taylor D, Liu T: In vivo molecular therapy with p53 adenovirus for microscopic residual head and neck squamous carcinoma. Cancer Res 1995, 55:1-6.
24. Liu T, El-Naggar A, McDonnell T, Steck K, Wang M, Taylor D, Clayman G: Apoptosis induction mediated by wild-type p53 adenoviral gene transfer in squamous cell carcinoma of the head and neck. Cancer Res 1995, 55:3117-3122.
25. Moolten F, Wells J, Heyman R, Evans RM: Lymphoma regression induced by ganciclovir in mice bearing a herpes thymidine kinase transgene. Hum Gene Ther 1990, 1:125-134.
26. Wilson K, Stambrook P, Bi W, Pavelic Z, Pavelic L, Gluckman J: HSV-tk gene therapy in head and neck squamous cell carcinoma: enhancement by the local and distant bystander effect Arch Otolaryngol Head Neck Surg 1996, 122:746-749.
27. O'Malley B, Chen S, Schwartz M, Woo S: Adenovirus-mediated gene therapy for human head and neck squamous cell cancer in a nude mouse model. Cancer Res 1995, 55:1080-1085.
28. Whiteside T, Chikamatsu K, Nagashima S, Okada K: Antitumor effects of cytolytic T lymphocytes (CTL) and natural killer (NK) cells in head and neck cancer. Anticancer Res 1996, 16(4C):2357-2364.
29. Nabel G, Chang A, Nabel E: Clinical protocol: immunotherapy of malignancy by in vivo gene transfer into tumors. Hum Gene Ther 1992, 3:399-410.
30. Rosenberg S, Anderson WF, Blaese MR: Clinical Research Project: immunization of cancer patients using autologous cancer cell modified by insertion of the gene for interleukin-2. Hum Gene Ther 1992, 3:75-90
31. Rosenberg S, Anderson WF, Asher A: Clinical research project: immunization of cancer patients using autologous cancer cells modified by insertion of the gene for tumor necrosis factor. Hum Gene Ther 1993, 4:57-73.
32. Reardon W: Genetic deafness. J Med Genet 1992, 29:521-526.
33. Gorlin R, Toriello H, Cohen M: Genetic Deafness and Its Syndromes, edn 2. Oxford: Oxford University Press; 1995.
34. Coyle B, Coffey R, Armour J, Gausden E, Hochberg Z, Grossman A, Britton K, Pembrey M, Reardon W, Trembath R: Pendred syndrome (goitre and sensorineural hearing loss) maps to chromosome 7 in the region containing the nonsyndromic deafness gene DFNB4. Nat Genet 1996, 12:421-423.
35. Cao M, Thonnard J, Deggouj N, Gersdorff M, Philippe M, Osselaer JC, Tomasi JP: HLA class II-associated genetic susceptibility in idiopathic progressive sensorineural hearing loss. Ann Otol Rhinol Laryngol 1996, 105:628-633.
36. Forrest D, Erway L, Ng L, Altschuler R, Curran T: Thyroid hormone receptor β is essential for development of auditory function. Nat Genet 1996, 13:354-357.
37. Fujiyoshi T, Hood L, Yoo T: Restoration of brain stem auditory evoked potentials by gene transfer in shiverer mice. Ann Otol Rhinol Laryngol 1994, 103:449-455.
38. Eide F, Lowenstein D, Reichardt L: Neurotrophins and their receptors current concepts and implications for neurologic disease. Exp Neurol 1993, 121:200-214.
39. Lefebvre P, Van de Water T, Represa J, Liu W, Bernd P, Modlin S, Moonen G, Mayer M: Temporal pattern of nerve growth factor binding in vivo and the in vitro effects of NGF on cultures of developing auditory and vestibular neurons. Acta Otolaryngol 1991, 111:304-311.
40. Ylikoski J, Pirvola U, Moshnyakov M, Palgi J, Arumae U, Saarma M: Expression patterns of neurotrophins and their receptor mRNA in the rat inner ear. Hear Res 1993, 56:69-78.
41. Despres G, Romand R: Neurotrophins and the development of cochlear innervation. Life Sci 1994, 54:1291-1297.
42. Ernfors P, Van de Water T, Loring J, Jaenisch R: Complementary roles of BDNF and NT-3 in vestibular and auditory development Neuron 1995, 14:1153-1164. This original research article investigates the physiologic roles of BDNF and neurotrophin-3 in the development of the vestibular and auditory systems in mice that carry deleted BDNF or neurotrophin-3 genes. The introduction summarizes the recent neurotrophin studies.
43. Van de Water T, Staecker H, Kopke R: Neurotrophic factors: maintenance and injury-repair of auditory neurons [abstract 7]. The Molecular Biology of Hearing and Deafness. Bethesda, MD: NIH; 1995.
44. Zheng J, Stewart R, Gao W: Neurotrophin-4/5 enhances survival of cultured spiral ganglion neurons and protects them from cisplatin neurotoxicity. J Neurosci 1995, 15:5079-5087.
45. Zheng J, Gao W: Differential damage to auditory neurons and hair cells by ototoxins and neuroprotection by specific neurotrophins in rat cochlear organotypic cultures. Eur J Neurosci 1996, 8:1897-1905.
46. Ernfors P, Duan M, EIShamy W, Canlon B: Protection of auditory neurons from aminoglycoside toxicity by neurotrophin-3. Nat Med 1996, 2:463-467.
47. Geschwind M, Hartnick C, Liu W, Amat J, Van de Water T, Ferderoff H: Defective HSV-1 vector expressing BDNF in auditory ganglia elicits neurite outgrowth: model for treatment of neuron loss following cochlear degeneration. Hum Gene Ther 1996, 7:173-182. The study demonstrates that HSV vectors can be used to introduce and express BDNF gene in auditory neurons in vitro. The article suggests possible application of exogenous neurotrophins introduced via viral vectors in treatment of some forms of deafness.
48. Lalwani A, Walsh B, Reilly P, Muzyczka N, Mhatre A: Development of in vivo gene therapy for hearing disorders: introduction of adeno-associated virus into the cochlea of the guinea pig. Gene Ther 1996, 3:588-592. This study demonstrates for the first time in vivo gene transfer and expression of exogenously introduced reporter gene, β-galactosidase gene, in the cochlea. The reporter gene was delivered via adeno-associated virus using direct intracochlear infusion with osmotic minipump.
49. Raphael Y, Frisancho J, Roessler B: Adenoviral-mediated gene transfer into guinea pig cochlear cells in vivo. Neurosci Lett 1996, 207:137-141. This study used adenovirus vector for reporter gene transfer into the cochlea of a guinea pig. The investigators concluded that efficient gene transfer into multiple types of cochlear cells in vivo can be achieved without major morphologic signs of pathology or toxicity.