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Stargardt’s macular degeneration; development of microchip-based screening technologies; gene therapy

Place of work — Department
of Ophthalmology,
Columbia University,
New York, USA
Contacts — Dept.
of Ophthalmology, Columbia University, 630 West 168th Street, New York, NY 1003.
(212) 305–8989;
rla22@columbia.edu
http://www.cumc.columbia.
edu/dept/eye/research/fac_al-
likmets.html
Publications — Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene
therapy. Kong J, Kim SR, Binley K, Pata I, Doi K, Mannik J, Zernant-Rajang J, Kan O,
Iqball S, Naylor S, Sparrow JR, Gouras P, Allikmets R. Gene Ther. 2008 Oct;15(19):1311-
1320.
Small molecule RPE65 antagonists limit the visual cycle and prevent lipofuscin formation.
Maiti P, Kong J, Kim SR, Sparrow JR, Allikmets R, Rando RR. Biochemistry. 2006 Jan
24;45(3):852–860.
Genotyping microarray (disease chip) for Leber congenital amaurosis: detection of modifier alleles.
Zernant, J., Külm, M., Dharmaraj, S., den Hollander, A.I., Perrault, I., Preising, M.N.,
Lorenz, B., Kaplan, J., Cremers, F.P.M., Maumenee, I., Koenekoop, R.K., and Allikmets, R.
Invest. Ophthalmol. Vis. Sci. 46:3052–3059, 2005.