Deep learning-guided design of cell type-specific AAV promoters
Single-cell multiome and enhancer connectome of human retinal pigment epithelium and choroid nominate pathogenic variants in age-related macular degeneration
Engineering circular RNA for enhanced protein production
Single-cell multiome of the human retina and deep learning nominate causal variants in complex eye diseases
Augmentation of CD47-SIRPα signaling protects cones in genetic models of retinal degeneration
In situ detection of adeno-associated viral vector genomes with SABER-FISH
Microglia modulation by TGF-β1 protects cones in mouse models of retinal degeneration
Soluble CX3CL1 gene therapy improves cone survival and function in mouse models of retinitis pigmentosa
Multiomics and deep learning dissect regulatory syntax in human development
Genetic elements promote retention of extrachromosomal DNA in cancer cells
Discrete Immolative Guanidinium Transporters deliver mRNA to specific organs and red blood cells
ERG responses to high-frequency flickers require FAT3 signaling in mouse retinal bipolar cells
Organ- and cell-selective delivery of mRNA in vivo using guanidinylated serinol charge-altering releasable transporters
Charge-altering releasable transporters enhance mRNA delivery in vitro and exhibit in vivo tropism
Chromophore supply modulates cone function and survival in retinitis pigmentosa mouse models
AAV-Txnip prolongs cone survival and vision in mouse models of retinitis pigmentosa
Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses
Nrf2 overexpression rescues the RPE in mouse models of retinitis pigmentosa
Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity
AAV cis-regulatory sequences are correlated with ocular toxicity
Hoxb5 marks long-term haematopoietic stem cells and reveals a homogenous perivascular niche
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