Results 111 to 120 of about 971 (147)

Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models. [PDF]

Sci Adv
Ferla R, Pugni E, Lupo M, Tiberi P, Fioretto F, Perota A, Duchi R, Lagutina I, Gesualdo C, Rossi S, Ventrella D, Elmi A, McClinton B, Toomes C, Xu T, Molday RS, Surace EM, Simonelli F, Bacci ML, Galli C, Memon MA, Shams N, Auricchio A, Trapani I.   +23 more
europepmc   +1 more source

Automated Quantification of Decreased Fundus Autofluorescence in Stargardt Disease Using <i>Starguage</i>: Validation against Manual Grading Standards. [PDF]

Ophthalmol Sci
Ahmed MI, Yucel H, Afridi R, de Guimarães TAC, Sendino-Tenorio I, Nguyen NV, Kiran R, Khan S, Khan U, Sharaf Un Nisa S, Campigotto M, Hariri A, Michaelides M, Scholl HPN, Mata N, Nguyen QD, Sepah YJ.   +16 more
europepmc   +1 more source

Activation of GSDME by all-<i>trans</i>-retinal increases sensitivity to photoreceptor ferroptosis. [PDF]

Int J Biol Sci
Yang B, Yang K, Chen Y, Xi R, Han J, Li S, Chen J, Wu Y.   +7 more
europepmc   +1 more source

Pathonign variants in recessive disorders: How extremely hypomorphic variants can be pathogenic and benign depending on the allele in trans. [PDF]

Intractable Rare Dis Res
Fabre A, Guerry P.
europepmc   +1 more source

Clinical and Genetic Characteristics of 18 Patients from Southeast China with ABCA4-Associated Stargardt Disease. [PDF]

Int J Mol Sci
Liu X, Liu Z, Cui J, Tan C, Sun W, Lin Y.   +5 more
europepmc   +1 more source

Quantifying the Progression of Stargardt Disease in Double-Null ABCA4 Carriers Using Fundus Autofluorescence Imaging. [PDF]

Transl Vis Sci Technol
Mihalek I, De Bruyn H, Glavan T, Lancos AM, Ciolfi CM, Malendowicz K, Aslaksen S, Molday LL, Molday RS, Fulton AB.   +9 more
europepmc   +1 more source

Therapeutic Efficacy of Multi-Characteristic Opsin Gene Therapy in a Mouse Model of Stargardt Disease. [PDF]

Bioengineering (Basel)
Mohanty S, Batabyal S, Kim S, Carlson M, Dibas A.   +4 more
europepmc   +1 more source

Rationally Designed, Short-Acting RPE65 Inhibitors for Visual Cycle-Associated Retinopathies. [PDF]

J Med Chem
Bassetto M, Hu Y, Li B, Chen X, Saraswat V, Damacio F, Smidak R, Palczewski K, Tochtrop GP, Kiser PD.   +9 more
europepmc   +1 more source

Safety and efficacy of MCO-010 optogenetic therapy in patients with Stargardt disease in USA (STARLIGHT): an open-label multi-center Ph2 trial. [PDF]

EClinicalMedicine
Lam BL, Zak V, Gonzalez VH, Gregori NZ, Chavala SH, Batabyal S, Carlson M, Kim S, Ayyagari A, Chang J, Lane H, Choudry N, Koester J, Von Tress M, Piltz-Seymour J, Sharif NA, Tsang SH, Mahajan VB, Boyer DS, Ho AC, Barone SB, Mohanty SK.   +21 more
europepmc   +1 more source

Identification and functional characterization of <i>ABCA4</i> gene variants in three patients with Stargardt disease or retinitis pigmentosa. [PDF]

Front Genet
Luo Q, Huang J, Shi L, Zhang G, Xue L, Wu K, Li X, Yang L, Li D, Mao L, Luo J.   +10 more
europepmc   +1 more source