Gene therapy for retinal dystrophies;Gentherapie bei Netzhautdystrophien

Ophthalmologe

Volumn 109, Issue 2, 2012, Pages 121-128

  Issa, P Charbel ^a,b   Groppe, M ^a   MacLaren, R E ^a,c  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b UNIVERSITY HOSPITAL BONN   (Germany)

^c MOORFIELDS EYE HOSPITAL   (United Kingdom)

Author keywords

Non viral gene therapy; Retinal degeneration; Retinal dystrophy; Retinal gene therapy

Indexed keywords

ARTICLE; GENE THERAPY; GENETICS; HUMAN; METHODOLOGY; RETINA DYSTROPHY; TREATMENT OUTCOME;

GENE THERAPY; HUMANS; RETINAL DYSTROPHIES; TREATMENT OUTCOME;

EID: 84857243443     PISSN: 0941293X     EISSN: None     Source Type: Journal    
DOI: 10.1007/s00347-011-2453-3     Document Type: Article

References (30)

1. Allocca M, Doria M, Petrillo M et al (2008) Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice. J Clin Invest 118:1955-1964
2. Bainbridge JW, Smith AJ, Barker SS et al (2008) Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med 358:2231-2239
3. Berger W, Kloeckener-Gruissem B, Neidhardt J (2010) The molecular basis of human retinal and vitreoretinal diseases. Prog Retin Eye Res 29:335-375
4. Bolz H (2005) Die Genetik der retinalen Dystrophien - ein Überblick. Ophthalmologe 102:661-673
5. Buch PK, Bainbridge JW, Ali RR (2008) AAV-mediated gene therapy for retinal disorders: from mouse to man. Gene Ther 15:849-857
6. Buch PK, Maclaren RE, Ali RR (2007) Neuroprotective gene therapy for the treatment of inherited retinal degeneration. Curr Gene Ther 7:434-445
7. Busskamp V, Roska B (2011) Optogenetic approaches to restoring visual function in retinitis pigmentosa. Curr Opin Neurobiol 21:942-946
8. Charbel Issa P, Maclaren RE (2012) Non-viral retinal gene-therapy: a review. Clin Experiment Ophthalmol 40:39-47
9. Charbel Issa P, Tröger E, Finger R et al (2010) Structur-function correlation of the human central retina. PLoS One 5:e12864
10. Cideciyan AV, Aleman TS, Boye SL et al (2008) Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proc Natl Acad Sci U S A 105:15112-15117
11. Cideciyan AV, Hauswirth WW, Aleman TS et al (2009) Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Hum Gene Ther 20:999-1004
12. Cideciyan AV, Hauswirth WW, Aleman TS et al (2009) Vision 1 year after gene therapy for Leber's congenital amaurosis. N Engl J Med 361:725-727
13. Deutsche Forschungsgemeinschaft (2006) Entwicklung der Gentherapie. Stellungnahme der Senatskommission für Grundsatzfragen der Genforschung. Mitteilung 5. Wiley-VCH
14. Frigg R, Wenzel A, Grimm C et al (2005) Überlebensfaktoren in der Therapie erblicher Netzhautdegenerationen. Ophthalmologe 102:757-763
15. Hauswirth WW, Aleman TS, Kaushal S et al (2008) Treatment of Leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther 19:979-990
16. Hirsch ML, Agbandje-Mckenna M, Samulski RJ (2010) Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus. Mol Ther 18:6-8
17. Jacobson SG, Cideciyan AV, Ratnakaram R et al (2011) Gene therapy for Leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Arch Ophthalmol, doi:10.1001/archophthalmol.2011.298
18. Lorenz B, Preising M, Stieger K (2010) Retinal blinding disorders and gene therapy- molecular and clinical aspects. Curr Gene Ther 10:350-370
19. Maclaren RE (2009) An analysis of retinal gene therapy clinical trials. Curr Opin Mol Ther 11:540-546
20. Maguire AM, High KA, Auricchio A et al (2009) Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial. Lancet 374:1597-1605
21. Maguire AM, Simonelli F, Pierce EA et al (2008) Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med 358:2240-2248
22. Neidhardt J, Wycisk K, Klöckener-Gruissem B (2005) Virale und nichtvirale Gentherapieansätze zur Behandlung von Netzhauterkrankungen. Ophthalmologe 102:764-771
23. Park TK, Wu Z, Kjellstrom S et al (2009) Intravitreal delivery of AAV8 retinoschisin results in cell type-specific gene expression and retinal rescue in the Rs1-KO mouse. Gene Ther 16:916-926
24. Peden MC, Min J, Meyers C et al (2011) Ab-externo AAV-mediated gene delivery to the suprachoroidal space using a 250 micron flexible microcatheter. PLoS One 6:e17140
25. Poloschek CM, Jägle H (2012) Pharmakologische Ansätze in der Therapie erblicher Netzhautdegenerationen. Ophthalmologe (im Druck)
26. Schlichtenbrede FC, Sarra GM, Ali RR et al (2002) Fortschritte in der somatischen Gentherapie von Netzhautdegenerationen am Tiermodell. Ophthalmologe 99:259-265
27. Schüle S, Renner M, Longhurst S et al (2010) Regulatory requirements for clinical trial and marketing authorisation application for gene therapy medicinal products. Bundesgesundheitsbl Gesundheitsforsch Gesundheitsschutz 53:30-37
28. Schüssler-Lenz M, Schneider CK (2010) Klinische Prüfung mit Arzneimitteln für Neuartige Therapien. Bundesgesundheitsbl Gesundheitsforsch Gesundheitsschutz 53:68-74
29. Schwerdtfeger WK (2005) Verordnung über die Anwendung der guten klinischen Praxis bei der Durchführung von klinischen Prüfungen mit Arzneimitteln zur Anwendung an Menschen (GCP-Verordnung). Bundesgesundheitsbl Gesundheitsforsch Gesundheitsschutz 48:147-154
30. Simonelli F, Maguire AM, Testa F et al (2010) Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration. Mol Ther 18:643-650