Adeno-Associated Virus Serotype 2-hCHM Subretinal Delivery to the Macula in Choroideremia: Two-Year Interim Results of an Ongoing Phase I/II Gene Therapy Trial

Autor: Tomas S, Aleman, Rachel M, Huckfeldt, Leona W, Serrano, Denise J, Pearson, Grace K, Vergilio, Sarah, McCague, Kathleen A, Marshall, Manzar, Ashtari, Tu M, Doan, Carol A, Weigel-DiFranco, Bethany S, Biron, Xiao-Hong, Wen, Daniel C, Chung, Emily, Liu, Kevin, Ferenchak, Jessica I W, Morgan, Eric A, Pierce, Dean, Eliott, Jean, Bennett, Jason, Comander, Albert M, Maguire

Publikováno v: Ophthalmology. 129(10)

To assess the safety of the subretinal delivery of a recombinant adeno-associated virus serotype 2 (AAV2) vector carrying a human choroideremia (CHM)-encoding cDNA in CHM.Prospective, open-label, nonrandomized, dose-escalation, phase I/II clinical tr

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=pmid________::175d44fa052beacd88edf6041f266d41
https://pubmed.ncbi.nlm.nih.gov/35714735

Treatment Potential for LCA5-Associated Leber Congenital Amaurosis

Autor: Ivan Shpylchak, Vidyullatha Vasireddy, Katherine E. Uyhazi, Denise J. Pearson, Zhangyong Wei, Jean Bennett, Lanfranco Leo, Leona W. Serrano, Puya Aravand, Tomas S. Aleman, Brent A. Bell, Jennifer Pham

Publikováno v: Investigative Ophthalmology & Visual Science

Purpose To determine the therapeutic window for gene augmentation for Leber congenital amaurosis (LCA) associated with mutations in LCA5. Methods Five patients (ages 6-31) with LCA and biallelic LCA5 mutations underwent an ophthalmic examination incl

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c10a9b77b46bc6bacc44c2d13496703c
http://europepmc.org/articles/PMC7405811

Comparing clinical perimetry and population receptive field measures in patients with choroideremia

Autor: Denise J. Pearson, Albert M. Maguire, Tomas S. Aleman, Manzar Ashtari, Andreas M. Rauschecker, Chris I. Baker, Aimee E. Willett, Leona W. Serrano, Edward H. Silson, Jean Bennett

Publikováno v: Silson, E H, Aleman, T S, Willett, A, Serrano, L W, Pearson, D J, Rauschecker, A M, Maguire, A M, Baker, C I, Bennett, J & Ashtari, M 2018, ' Comparing clinical perimetry and population receptive field measures in patients with choroideremia ', Investigative Ophthalmology & Visual Science, vol. 59, no. 8, pp. 3249-3258 . https://doi.org/10.1167/iovs.18-23929
Investigative Ophthalmology & Visual Science

PURPOSE. Choroideremia (CHM) is an X-linked recessive form of hereditary retinal degeneration, which, at advanced stages, leaves only small central islands of preserved retinal tissue. Unlike many other retinal diseases, the spared tissue in CHM supp

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d084042f20aae31cdc8d7284991f9ef4
https://hdl.handle.net/20.500.11820/bdedc1e0-a1eb-4a95-a700-5ac2b95f4cb9

Late recurrence of retinopathy of prematurity after treatment with both intravitreal bevacizumab and laser

Autor: Gil Binenbaum, Wendy Chen, Agnieshka Baumritter, Graham E. Quinn, Karen A. Karp, Denise J. Pearson, Albert M. Maguire

Publikováno v: Journal of American Association for Pediatric Ophthalmology and Strabismus. 18:402-404

An infant of 36 weeks’ postmenstrual age (PMA) and 25 weeks’ gestation received bilateral intravitreal bevacizumab injections for type 1 retinopathy of prematurity. He underwent laser photocoagulation in both eyes 5 days later, confluent except f

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2241b57d7c82daf1407d117f0fd077f4
https://doi.org/10.1016/j.jaapos.2014.03.011

Training retinal imagers for retinopathy of prematurity (ROP) screening

Autor: Karen A. Karp, Agnieshka Baumritter, Michele Huynh, Kelli Satnes, Gui-Shuang Ying, Denise J. Pearson, Rachel J. Keith, Maxwell Pistilli, Darla N. Nyquist, Graham E. Quinn

Publikováno v: Journal of AAPOS : the official publication of the American Association for Pediatric Ophthalmology and Strabismus. 20(3)

To report the training/certification process of nonphysician imagers, image quality, and factors that affected image quality in the National Eye Institute sponsored multicentered e-ROP study.Nonphysician imagers underwent rigorous training and certif

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e6295d5fed21d37e227f9ece61641c43
https://pubmed.ncbi.nlm.nih.gov/27164425