Results 71 to 80 of about 6,270 (121)

Compact Calm1 promoter enables AAV mediated neuron-targeted expression in human iPSC-derived brain organoids. [PDF]

Sci Rep
Klockner I, Yeturi M, Rust TE, Stein JL, Zylka MJ.   +4 more
europepmc   +1 more source

Development and Validation of a Quantitative LC-MS/MS Method for Measuring CYP4V2 Enzyme Activity via 12-Hydroxylauric Acid in rAAV-hCYP4V2 Gene Therapy Products. [PDF]

Molecules
Ren G, Qin X, Li Y, Fan W, Luo W, Cao Y, Wang Y, Zhou Y, Liang C.   +8 more
europepmc   +1 more source

The Impact of QSP Modeling on the Design and Optimization of Gene Therapy Approaches. [PDF]

CPT Pharmacometrics Syst Pharmacol
Rayad N, Chowdhury EA, Meno-Tetang GML.
europepmc   +1 more source

Development of a Novel Method to Detect AAV Vector Integration. [PDF]

Viruses
Zhang J, Dang TT, Lin TY, Yu X, Pellin D, Tian J, Simmons O, Kou E, Cornetta K, Xiao W.   +9 more
europepmc   +1 more source

Transcriptomic Profiling Reveals Divergent Immune Responses to AAV1 and AAV-ie in Mice Inner Ear. [PDF]

Int J Med Sci
Shi D, Han L, Li C, Geng G, Jiang L, Chen X, Yang F, Feng Y, Luo J, Shu Y.   +9 more
europepmc   +1 more source

Novel promoters drive therapeutic transgene expression and evade transgene-specific immune responses in a mouse model of Pompe disease. [PDF]

Mol Genet Metab
Sun B, Yi H, Han SO, Li S, Eisner W, Brooks ED, Bacon R, Koeberl DD.   +7 more
europepmc   +1 more source

Neuritin1 Cis-Regulatory Elements Enable Gene Expression Preferentially in Retinal Ganglion Cells. [PDF]

Invest Ophthalmol Vis Sci
Talla V, Koilkonda R, Kinane C, Panchal M, Khuu T, Park KK.   +5 more
europepmc   +1 more source

Adeno-associated virus and hepatocytes: frenemies? [PDF]

Blood Adv
Pierce GF.
europepmc   +1 more source

Gene therapy rescues cone function in an all-cone retina mouse model with the most common cone opsin C203R missense mutation. [PDF]

PLoS One
Cahill ME, Chmelik K, Brothers BA, Ashcraft ME, Guan T, Puja A, Xiang Y, Shaw LM, Du J, Deng WT.   +9 more
europepmc   +1 more source

Methodological validation of a cryo-TEM-based detection method for empty capsid ratio in recombinant adeno-associated virus. [PDF]

Biochem Biophys Rep
Xu L, Chen J, Zhu W, Zhao Y, Zheng F, Du R, Jiang Y, Yang Y, Chen Y, Dong Y, Zhang Z, Tong Y.   +11 more
europepmc   +1 more source