Results 141 to 150 of about 7,638 (169)

rAAV production cost analysis: Indication-specific cost per dose and reduction strategies. [PDF]

Gene Ther
Park MT, Matuszek D, Andaluz A, Motevalian SP.   +3 more
europepmc   +1 more source

Determination of the Population Frequency of Monoallelic and Biallelic Predicted Pathogenic RPE65 Variants in a Normal Database. [PDF]

Invest Ophthalmol Vis Sci
Abdul R, Liu X, Choi K, Huang M, Mack H, Savige J.   +5 more
europepmc   +1 more source

Chorioretinal Atrophy after Voretigene Neparvovec Therapy in <italic>RPE65</italic> Mutation-Associated Inherited Retinal Disease: Longitudinal Characterization in Multimodal Imaging.

Ophthalmologica
Sassen SH, Sassmannshausen M, Sassen J, Preising MN, Scholz JP, Holz FG, Lorenz B, Herrmann P.   +7 more
europepmc   +1 more source

Illuminating retinoid flux in the neurosensory retina. [PDF]

J Clin Invest
Moshiri A, Sodhi A.
europepmc   +1 more source

Ammonia-induced cell death patterns in glioblastoma: Immune landscape, prognostic signature, and therapeutic target identification. [PDF]

Genes Dis
Hu SQ, Wang J, Wang J, Zeng C, Chen R.
europepmc   +1 more source

The cone visual cycle and its disorders: insights from zebrafish. [PDF]

Front Mol Neurosci
Koutroumpa IS, Neuhauss SCF.
europepmc   +1 more source

Clinical endpoints special session, ISCEV 2025, Utrecht. [PDF]

Doc Ophthalmol
Hamilton R, Birch D, Stingl K, Fujinami K, Holopigian K, Boon CJF, Moseley J, McAnany JJ, Cideciyan AV, van Genderen MM.   +9 more
europepmc   +1 more source

Genome-wide identification and characterization of the carotenoid cleavage dioxygenase gene family in <i>Bixa orellana</i> L. [PDF]

Front Plant Sci
Narváez-Zapata JA, Garduño-Guadarrama G, Sierra-Ulín D, Cervera A, Tovar H, Rivera-Madrid R.   +5 more
europepmc   +1 more source

CRISPR/Cas-mediated activation of genes associated with inherited retinal dystrophies in human cells for diagnostic purposes. [PDF]

JCI Insight
Weber VJ, Reschigna A, Gerhardt MJ, Heigl T, Hinrichsmeyer KS, den Engel SV, Otify DY, Gavrilov Z, Blaser F, Meneau I, Betz C, Bolz HJ, Biel M, Michalakis S, Becirovic E.   +14 more
europepmc   +1 more source