Fetoscopic gene therapy for congenital lung disease

Journal of Pediatric Surgery

Volumn 32, Issue 7, 1997, Pages 964-969

  Sylvester, Karl G ^b   Yang, Edmund Y ^b   Cass, Darrell L ^b   Crombleholme, Timothy M ^b   Adzick, N Scott ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b NONE

Author keywords

Cystic fibrosis; Fetal gene therapy; Fetoscopy; Surfactant protein B deficiency; Transgene

Indexed keywords

BETA GALACTOSIDASE; SURFACTANT;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; BRONCHOSCOPY; CONFERENCE PAPER; CONGENITAL LUNG DISEASE; CYSTIC FIBROSIS; CYTOTOXIC LYMPHOCYTE; FEMALE; FETOSCOPY; FETUS LUNG; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; LUNG ALVEOLUS CELL TYPE 2; NONHUMAN; PRIORITY JOURNAL; SHEEP; TRANSGENE;

EID: 0030748567     PISSN: 00223468     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0022-3468(97)90378-X     Document Type: Conference Paper

References (22)

1. Nogee LM, DeMello DE, Dehner LP, et al: Deficiency of pulmonary surfactant protein B in congenital alveolar proteinosis. N Engl J Med 328:406-410, 1993
2. Nogee LM, Garnier G, Dietz HC, et al: A mutation in the surfactant protein B gene responsible for fatal neonatal respiratory disease in multiple kindreds. J Clin Invest 93:1860-1863, 1994
3. Yei SP, Bachinski CJ, Weaver TE, et al: Adenoviral mediated gene transfer of human surfactant protein B to respiratory epithelial cells. Am J Respir Cell Mol Biol 11:329-336, 1994
4. Rich DP, Couture LA, Cardoza LM, et al: Development and analysis of recombinant adenovirus for gene therapy of cystic fibrosis. Hum Gene Ther 4:461-476, 1993
5. Rosenfeld MA, Yoshimura BC, Trapnell BC, et al: In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68:143-155, 1992
6. Englehardt JF, Simon RH, Yang Y, et al: Adenovirus-mediated transfer of the CFTR gene to lung of non-human primates-biological efficacy study. Hum Gene Ther 4:759-769, 1993
7. Grubb BR, Pickles RJ, Ye H, et al: Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelium of mice and humans. Nature 371:802-806, 1994
8. Crystal RG, McElvaney NG, Rosenfeld MA, et al: Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis. Nature Genetics 8:42-51, 1994
9. McCray PB, Armstrong K, Zabner J, et al: Adenoviral mediated gene transfer to fetal pulmonary epithelia in vitro and in vivo. J Clin Invest 95:2620-2632, 1995
10. Holzinger A, Trapnell BC, Weaver TE, et al: Intraamniotic administration of an adenoviral vector for gene transfer to fetal sheep and mouse tissues. Ped Res 38:844-850, 1995
11. Vincent MC, Trapnell BC, Baughman RP, et al: Adenovirus mediated gene transfer to the respiratory tract of fetal sheep in utero. Hum Gene Ther 6:1019-1028, 1995
12. Kass-Eisler A, Falck-Pedersen E, Elfenbein DH, et al: The impact of developmental stage, route of administration and the immune system on adenovirus-mediated gene transfer. Gene Therapy 1:395-402, 1994
13. Alcorn DG, Adamson TM, Maloney JR, et al: A morphologic and morphometric analysis of fetal lung development in the sheep. Anat Rec 201:655-667, 1981
14. Adzick NS, Harrison MR: Fetal surgical therapy. Lancet 343:897-902, 1994
15. Luks FI, DePrest JA, Vandenberghe K, et al: Fetoscopy guided fetal endoscopy in a sheep model. J Am Coll Surg 178:609-612, 1994
16. Bout A, Perricaudet M, Baskin G, et al: Lung gene therapy: In vivo adenovirus mediated gene transfer to rhesus monkey airway epithelium. Hum Gene Ther 5:3-10, 1994
17. Trapnell BC: Adenoviral vectors for gene transfer. Advanced Drug Delivery Rev 12:185-199, 1993
18. Berkner KL: Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 6:616-624, 1988
19. Nogee LM, Hull W, Whitsett JA, et al: Inherited surfactant protein B deficiency: Prenatal diagnosis. Pediatr Res 33:227A, 1993
20. Galan HL, Kuehl TJ: Effect of intra-amniotic administration of Exosurf in preterm rabbit fetuses. Obstet Gynecol 80:604-608, 1992
21. Zabner J, Petersen DM, Puga AP, et al: Safety and efficacy of repetitive adenovirus-mediated transfer of CFTR cDNA to airway epithelium of primates and cotton rats. Nature Genetics 6:75-83, 1994
22. Yang Y, Nunes FA, Berencsi K, et al: Cellular immunity to viral antigens limits El-deleted adenoviruses for gene therapy. Proc Natl Acad Sci U S A 91:4407-4411, 1994