A5012082461- Retinal Diseases and Treatments
- Retinal Development and Disorders
- Retinal Imaging and Analysis
- Retinopathy of Prematurity Studies
- Glaucoma and retinal disorders
- Photoreceptor and optogenetics research
- Neonatal Respiratory Health Research
- Virus-based gene therapy research
- Neonatal and fetal brain pathology
- Single-cell and spatial transcriptomics
- RNA Interference and Gene Delivery
- Neuroinflammation and Neurodegeneration Mechanisms
- Ophthalmology and Visual Impairment Studies
- Trypanosoma species research and implications
- bioluminescence and chemiluminescence research
- Advanced Fluorescence Microscopy Techniques
- Cardiomyopathy and Myosin Studies
- Hematopoietic Stem Cell Transplantation
- RNA modifications and cancer
- Liver Disease Diagnosis and Treatment
- Advanced Proteomics Techniques and Applications
- RNA Research and Splicing
- Congenital heart defects research
- Protein purification and stability
- Advanced biosensing and bioanalysis techniques
Howard Hughes Medical Institute
2019-2025
Stanford University
2013-2025
Harvard University
2017-2025
City Of Hope National Medical Center
2014-2025
University of California, Los Angeles
2023-2025
New York University Shanghai
2024
Smith-Kettlewell Eye Research Institute
2017-2024
Atrium Health Wake Forest Baptist
2023-2024
University of California, Irvine
2023
Wake Forest University
2023
Abstract Circular RNAs (circRNAs) are stable and prevalent in eukaryotic cells that arise from back-splicing. Synthetic circRNAs some endogenous can encode proteins, raising the promise of circRNA as a platform for gene expression. In this study, we developed systematic approach rapid assembly testing features affect protein production synthetic circRNAs. To maximize translation, optimized five elements: vector topology, 5′ 3′ untranslated regions, internal ribosome entry sites aptamers...
Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their many advantages, including reduced inflammatory profile compared with that of other viruses. However, even in areas immune privilege such as the eye, AAV are capable eliciting host-cell responses. To investigate effects responses on several ocular cell types, we tested multiple genome structures and capsid types using subretinal injections mice. Assays morphology, inflammation, physiology were performed....
Incorporating TLR9-inhibitory sequences in the AAV vector genome inhibits immunogenicity and enhances transgene expression multiple animal models.
Myopia has reached epidemic levels among children in regions of East and Southeast Asia. High myopia is associated with myopic macular degeneration, glaucoma, retinal detachment.To determine the incidence high based on refraction without cycloplegia primary junior schools China.This observational cohort study was completed Guangzhou, China. It consisted a from 19 schools, who were followed up 2010 to 2015, 22 2012. All randomly chosen at rates proportional number each city's 11 districts....
Genome-wide association studies (GWASs) of eye disorders have identified hundreds genetic variants associated with ocular disease. However, the vast majority these are noncoding, making it challenging to interpret their function. Here we present a joint single-cell atlas gene expression and chromatin accessibility adult human retina more than 50,000 cells, which used analyze single-nucleotide polymorphisms (SNPs) implicated by GWASs age-related macular degeneration, glaucoma, diabetic...
Vision is initiated by the reception of light photoreceptors and subsequent processing via downstream retinal neurons. Proper circuit organization depends on multifunctional tissue polarity protein FAT3, which required for amacrine cell connectivity lamination. Here, we investigated function Fat3 mutant mice found decreases in both electroretinography perceptual responses to high-frequency flashes. These defects did not correlate with abnormal wiring, pointing instead a role bipolar subtypes...
Human brain development requires generating diverse cell types, a process explored by single-cell transcriptomics. Through parallel meta-analyses of the human cortex in (seven datasets) and adulthood (16 datasets), we generated over 500 gene co-expression networks that can describe mechanisms cortical development, centering on peak stages neurogenesis. These meta-modules show dynamic subtype specificities throughout with several developmental displaying spatiotemporal expression patterns...
BACKGROUND AND OBJECTIVE: To report the 5-year results of Stanford University Network for Diagnosis Retinopathy Prematurity (SUNDROP) telemedicine initiative. PATIENTS METHODS: Infants requiring retinopathy prematurity (ROP) screening at six neonatal intensive care units from December 1, 2005, to November 30, 2010, were evaluated with remote retinal photography by an ROP specialist. Every infant received outpatient binocular indirect ophthalmoscope examinations until termination criteria...
Retinitis pigmentosa (RP) is a genetically heterogenous group of eye diseases in which initial degeneration rods triggers secondary cones, leading to significant loss daylight, color, and high-acuity vision. Gene complementation with adeno-associated viral (AAV) vectors one strategy treat RP. Its implementation faces substantial challenges, however; for example, the tremendous number loci causal mutations. therapy targeting cone an alternative approach that could provide much-needed generic...
Nrf2, a transcription factor that regulates the response to oxidative stress, has been shown rescue cone photoreceptors and slow vision loss in mouse models of retinal degeneration (rd). The pigment epithelium (RPE) is damaged these models, but whether it also could be rescued by Nrf2 not previously examined. We used an adeno-associated virus (AAV) with RPE-specific (Best1) promoter overexpress RPE rd mice. Control mice showed disruption regular array RPE, as well cells. Cones were lost...
Retinitis pigmentosa (RP) is an inherited retinal disease affecting >20 million people worldwide. Loss of daylight vision typically occurs due to the dysfunction/loss cone photoreceptors, cell type that initiates our color and high-acuity vision. Currently, there no effective treatment for RP, other than gene therapy a limited number specific genes. To develop gene-agnostic therapy, we screened 20 genes their ability prolong photoreceptor survival in vivo. Here, report adeno-associated...
Abstract The introduction of more effective and selective mRNA delivery systems is required for the advancement many emerging biomedical technologies including development prophylactic therapeutic vaccines, immunotherapies cancer strategies genome editing. While polymers oligomers have served as promising systems, their efficacy in hard-to-transfect cells such primary T lymphocytes often limited cell organ tropism. To address these problems, considerable attention has been placed on...
Significance Retinitis pigmentosa (RP) is a genetically heterogenous disease that lacks effective treatment. In RP, there degeneration of cone photoreceptors in the eye, often leading to complete blindness. The reasons for remain largely unknown. Development therapies preserve cones and vision patients with particularly mutation-independent manner, would benefit from greater understanding underlying pathological mechanisms. We examined immune responses RP mice during period identified...
Retinitis pigmentosa (RP) is an ocular disease characterized by the loss of night vision, followed daylight vision. Daylight vision initiated in retina cone photoreceptors, which are gradually lost RP, often as bystanders a process that initiates their neighboring rod photoreceptors. Using physiological assays, we investigated timing electroretinogram (ERG) decline RP mouse models. A correlation between time ERG and rods was found. To investigate potential role visual chromophore supply this...
Selective RNA delivery is required for the broad implementation of clinical applications, including prophylactic and therapeutic vaccinations, immunotherapies cancer, genome editing. Current polyanion relies heavily on cationic amines, while guanidinium systems have received limited attention due in part to their strong association, which impedes intracellular release. Here, we disclose a general solution this problem groups are used form stable complexes upon formulation but at...
NKp46 is a critical regulator of natural killer (NK) cell immunity, but its function in non-NK innate immune cells remains unclear. Here, we show that indispensable for expressing IL-2 receptor-α (IL-2Rα) by liver-resident type-1 lymphoid (ILC1s). Deletion reduces IL-2Rα on ILC1s downregulating NF-κB signaling, thus impairing ILC1 proliferation and cytotoxicity vitro vivo. The binding anti-NKp46 antibody to triggers the activation NF-κB, expression IL-2Rα, interferon-γ (IFN-γ), tumor...