Zobrazeno 1 - 10
of 34
pro vyhledávání: '"Adrian M. Timmers"'
Autor:
Guo-jie Ye, Chunjuan Song, Judith A Newmark, Artur V. Cideciyan, Paulette M. Robinson, William A. Beltran, Adrian M. Timmers, David R. Knop, Jeffrey D. Chulay, Valerie L. Dufour, Mark S. Shearman, Malgorzata Swider, Samuel G. Jacobson, Gustavo D. Aguirre
Publikováno v:
Human Gene Therapy. 31:743-755
Recombinant adeno-associated viral (rAAV) vector-mediated gene therapy is being developed to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase...
Autor:
Savitri Mandapati, Chunjuan Song, Alok K. Sharma, Mailin Van Hoosear, William W. Hauswirth, Guo-jie Ye, Jeffrey D. Chulay, Adrian M. Timmers, Cayrn Plummer, Ping Zhu, David R. Knop, Kari B. Green, Paulette M. Robinson, Peter Sonnentag, Wen-Tao Deng, Mark S. Shearman, Thomas J. Conlon, Kirsten E. Coleman
Publikováno v:
Human Gene Therapy Clinical Development. 29:188-197
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated AAV2tYF-GRK1-RPGRco, to treat retinitis pigmentosa (RP) in patients with mutations in the retinitis pigmentosa
Autor:
Chunjuan, Song, Valérie L, Dufour, Artur V, Cideciyan, Guo-Jie, Ye, Malgorzata, Swider, Judith A, Newmark, Adrian M, Timmers, Paulette M, Robinson, David R, Knop, Jeffrey D, Chulay, Samuel G, Jacobson, Gustavo D, Aguirre, William A, Beltran, Mark S, Shearman
Publikováno v:
Hum Gene Ther
Recombinant adeno-associated viral (rAAV) vector-mediated gene therapy is being developed to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retinitis pigmentosa GTPase regulator (RPGR) gene. In preparation for a clinical
Autor:
Chunjuan Song, Mark S. Shearman, Guo-jie Ye, Artur V. Cideciyan, Paulette M. Robinson, Gui-Shuang Ying, Gustavo D. Aguirre, Valerie L. Dufour, Malgorzata Swider, Adrian M. Timmers, William A. Beltran, Nicole M. Weinstein, Perry L. Habecker, Wei Pan, David R. Knop, Samuel G. Jacobson, Jeffrey D. Chulay, Amy C. Durham
Publikováno v:
Hum Gene Ther
Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-associated virus (rAAV) vector AGTC-501, also designated rAAV2tYF-GRK1-hRPGRco, to treat X-linked retinitis pigmentosa (XLRP) in patients with mutations in the retiniti
Autor:
Chantelle Gaskin, Tanaz Farivar, Heikki T Turunen, David R. Knop, Chunjuan Song, Judith A Newmark, Mark S. Shearman, Steven Pennock, Guo-jie Ye, Adrian M. Timmers, Jilin Liu
Publikováno v:
Human gene therapy. 31(1-2)
Both subretinal dosing and intravitreal (IVT) dosing of adeno-associated virus (AAV) in higher species induce mild and transient inflammatory responses that increase with dose. Foreign protein and foreign DNA are known inducers of inflammation, which
Autor:
Mark S. Sands, Judith Mosinger Ogilvie, William W. Hauswirth, Anne K. Hennig, Kevin K. Ohlemiller, Adrian M. Timmers
Publikováno v:
Molecular Therapy. 10(1):106-116
The beta-glucuronidase-deficient mucopolysaccharidosis type VII (MPS VII) mouse accumulates partially degraded glycosaminoglycans in many cell types, including retinal pigmented epithelial (RPE) cells in the eye. This lysosomal storage in RPE cells l
Autor:
Adrian M. Timmers, Judith D. Ochrietor, Takashi Muramatsu, Tatiana P. Moroz, Paul J. Linser, Michael F. Clamp
Publikováno v:
Vision Research. 42:447-453
5A11/Basigin is an immunoglobulin-like glycoprotein expressed on the surface of Müller cells, the apical and basal surfaces of the retinal pigmented epithelium, and photoreceptor cell bodies and their inner segments. Disruption of the 5A11/Basigin g
Autor:
Seok Hong Min, Yumiko Umino, Drew Everhart, Jijing Pang, William W. Hauswirth, Robert B. Barlow, Qiuhong Li, Adrian M. Timmers, Bo Chang, Norman L. Hawes, John J. Alexander
Publikováno v:
Nature Medicine. 13:685-687
Loss of cone function in the central retina is a pivotal event in the development of severe vision impairment for many prevalent blinding diseases. Complete achromatopsia is a genetic defect resulting in cone vision loss in 1 in 30,000 individuals. U
Autor:
J.H. McDowell, Krzysztof Palczewski, W. C. Smith, H. Van Der Wel, Robert J. Ulshafer, Adrian M. Timmers, Grazyna Adamus, Paul A. Hargrave
Publikováno v:
Experimental Eye Research. 61:569-578
We sequenced selected peptides of alligator rhodopsin that accounted for about half of the total protein. These sequences were confirmed when the total primary structure of alligator rhodopsin was deduced from the cDNA sequence. Differences in the am
Autor:
Adrian M. Timmers, Charles A. Thornton, Karen A. Johnstone, Maurice S. Swanson, Douglas W. Esson, William W. Hauswirth, Codrin Lungu, Ami Mankodi, Rahul N. Kanadia
Publikováno v:
Science. 302:1978-1980
The neuromuscular disease myotonic dystrophy (DM) is caused by microsatellite repeat expansions at two different genomic loci. Mutant DM transcripts are retained in the nucleus together with the muscleblind (Mbnl) proteins, and these abnormal RNAs so