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Beyond the Lab and Into the Hospital: An Outlook on the Clinical Utility of Spatial Omics Technologies 走出实验室,走进医院:空间组学技术的临床应用展望
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.0030
Dean M. Pucciarelli, Benjamin Y. Lu, Inti Zlobec, Marcello DiStasio
{"title":"Beyond the Lab and Into the Hospital: An Outlook on the Clinical Utility of Spatial Omics Technologies","authors":"Dean M. Pucciarelli, Benjamin Y. Lu, Inti Zlobec, Marcello DiStasio","doi":"10.1089/genbio.2023.0030","DOIUrl":"https://doi.org/10.1089/genbio.2023.0030","url":null,"abstract":"Spatial omics technologies, including highly multiplexed histologic protein assays, nucleic acid abundance and/or sequence mapping, and spatial epigenetics assays, offer powerful tools for interrogating the complex biology of human tissues. These technologies have been broadly applied in basic and translational research, which presages deployment in clinical settings as well. In this article, we discuss spatial omics technologies with an emphasis on retrieval of disease-related information in single samples, with potential clinical applications in specialties such as oncology and immunology, and in the development of personalized treatment. Capable of localizing detailed molecular information within histologic structures, spatial omics technologies provide both cell-intrinsic information and microenvironmental interaction context. This will allow more precise diagnostic and prognostic classifications and more accurate predictions about treatment responses to be made. While technical and financial challenges to widespread deployment in clinical laboratories remain, spatial omics technologies are expected to dramatically expand actionable information obtained by human tissue sampling for pathologic analysis.","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"39 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135809417","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Spatial Omics Spotlights the Players in the Tumor Microenvironment 空间组学聚焦肿瘤微环境中的参与者
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.29115.sro
Sachin Rawat
{"title":"Spatial Omics Spotlights the Players in the Tumor Microenvironment","authors":"Sachin Rawat","doi":"10.1089/genbio.2023.29115.sro","DOIUrl":"https://doi.org/10.1089/genbio.2023.29115.sro","url":null,"abstract":"GEN BiotechnologyVol. 2, No. 5 News Feature: Spatial OmicsFree AccessSpatial Omics Spotlights the Players in the Tumor MicroenvironmentSachin RawatSachin Rawat*Address correspondence to: Sachin Rawat, Freelance Science Writer. E-mail Address: [email protected]Freelance Science Writer.Search for more papers by this authorPublished Online:16 Oct 2023https://doi.org/10.1089/genbio.2023.29115.sroAboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail Researchers are using spatial omics to look deeper into the tumor microenvironment and unravel tumor heterogeneity with an eye on gleaning important clinical insights.Tumor immune microenvironment of human colorectal cancer. Cancer cells in green and immune cells in magenta. (Credit: NanoString Technologies)Many hard-to-treat cancers often recur months or years after successful treatment. “You could have one cell that escapes treatment and it's that one cell that will populate and be resistant and allow for a recurrence to happen,” said Jasmine Plummer, founding director of the Center for Spatial Omics at St. Jude Children's Research Hospital.Investigating such tumors with even single-cell omics technologies could miss these resistant cells. Before analysis, single-cell technologies destroy the cancer tissue to look at what's happening in the tissue as a whole. However, a lot of the interesting stuff inside tumors happens at the level of individual cells and depends on the context in which they exist. Single-cell technologies lose this spatial context when the cells are broken up.This is where spatial omics come in.With advances in omics technologies, cancer biologists have extensive information on the genes, proteins, and other metabolites that make up the messy environment of a tumor. Single-cell omics goes further, enabling the identification of all cell types in a tumor sample. This has only deepened our understanding of the extreme heterogeneity of tumor cells. Spatial omics technologies are placing these insights in the spatial context.Jasmine Plummer, Founding Director of the Center for Spatial Omics at St. Jude Children's Research HospitalTake gene expression, for example. Single-cell transcriptomics reveals which genes are being expressed across different cell types. But it doesn't say where these cells are in the tumor. Spatial transcriptomics technologies fill this gap by simultaneously recording spatial coordinates with gene expression data. This is the crux of the growing field of spatial omics: assigning pin codes to omics data.Spatial transcriptomics technologies such as in situ hybridization and in situ sequencing allow researchers to capture transcriptomes without losing spatial information. The former uses fluorescent, gene-specific probes that bind mRNAs, whereas the latter sequences the transcripts directly in a section of a fixed tissue.Complementing these imaging-ba","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"31 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135809735","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Diffusion Evolution: New Artificial Intelligence Models Break Barriers in Protein Design 扩散进化:新的人工智能模型打破了蛋白质设计的障碍
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.29114.fli
Fay Lin
{"title":"Diffusion Evolution: New Artificial Intelligence Models Break Barriers in Protein Design","authors":"Fay Lin","doi":"10.1089/genbio.2023.29114.fli","DOIUrl":"https://doi.org/10.1089/genbio.2023.29114.fli","url":null,"abstract":"GEN BiotechnologyVol. 2, No. 5 News FeaturesFree AccessDiffusion Evolution: New Artificial Intelligence Models Break Barriers in Protein DesignFay LinFay LinE-mail Address: [email protected]Senior Editor, GEN BiotechnologySearch for more papers by this authorPublished Online:16 Oct 2023https://doi.org/10.1089/genbio.2023.29114.fliAboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail Diffusion models, a form of generative artificial intelligence, are a rising tool for protein design, showing improved experimental success and new potential for biotechnological applications.This protein fold is one of thousands designed from scratch using new machine learning methods. (Credit: Ian C. Haydon/UW Institute for Protein Design)In July 2023, scientists in David Baker's laboratory at the University of Washington (UW) published a report in Nature detailing a new deep-learning framework for de novo protein design called RoseTTAFold diffusion (RFdiffusion), in Nature.1 Since then, the scientific community has been buzzing about RFdiffusion's unprecedented experimental success rate and ease of use.David Juergens, a graduate student in Baker's laboratory and one of seven co-lead authors of the Nature article, shared an anecdote about a scientist working in a lab in China, who posted on social media how “they designed a protein in a browser, ordered the sequence, purified the protein, crystallized it, and then got a crystal structure that was half an angstrom away from the design that was on the computer. It was amazing!” Juergens told me.David Baker, Professor in Biochemistry and Director of the Institute for Protein Design at UWSome of the applications of RFdiffusion, documented with experimental validation in the Nature article, include design of symmetric oligomers for vaccine platforms and delivery vehicles and generation of high-affinity binders for therapeutics.1 In another project, the Baker laboratory has applied RFdiffusion to design proteins that bind peptide hormones—established biomarkers for clinical care and biomedical research—for diagnostic applications.2Box 1. Let's Generate interactionsGenerate: Biomedicines is a Boston-based therapeutics company at the intersection of machine learning, biological engineering, and medicine. Molly Gibson, cofounder and chief strategy and innovation officer, says the company focuses on designing protein–protein interactions for therapeutic applications.“If you think about biologics, the most important function that a protein takes is creating very specific and potent binding with its target. This could be things like an antibody where we know exactly where we want to neutralize a target, or where we want to agonize and potentiate function,” said Gibson.One project at Generate: Biomedicines has worked to create a broadly neutralizing antibody for coronavirus. Gibson notes that the virus activel","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"37 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135809736","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Technical Advances and Applications of Spatial Transcriptomics 空间转录组学的技术进展与应用
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.0032
Guohao Liang, Hong Yin, Fangyuan Ding
{"title":"Technical Advances and Applications of Spatial Transcriptomics","authors":"Guohao Liang, Hong Yin, Fangyuan Ding","doi":"10.1089/genbio.2023.0032","DOIUrl":"https://doi.org/10.1089/genbio.2023.0032","url":null,"abstract":"Transcriptomics is one of the largest areas of research in biological sciences. Aside from RNA expression levels, the significance of RNA spatial context has also been unveiled in the recent decade, playing a critical role in diverse biological processes, from subcellular kinetic regulation to cell communication, from tissue architecture to tumor microenvironment, and more. To systematically unravel the positional patterns of RNA molecules across subcellular, cellular, and tissue levels, spatial transcriptomics techniques have emerged and rapidly became an irreplaceable tool set. Herein, we review and compare current spatial transcriptomics techniques on their methods, advantages, and limitations, as well as applications across a wide range of biological investigations. This review serves as a comprehensive guide to spatial transcriptomics for researchers interested in adopting this powerful suite of technologies.","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"21 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135811698","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Influence of Alzheimer's Disease Related Neuropathology on Local Microenvironment Gene Expression in the Human Inferior Temporal Cortex 阿尔茨海默病相关神经病理对人类下颞叶皮层局部微环境基因表达的影响
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.0019
Sang Ho Kwon, Sowmya Parthiban, Madhavi Tippani, Heena R. Divecha, Nicholas J. Eagles, Jashandeep S. Lobana, Stephen R. Williams, Michelle Mak, Rahul A. Bharadwaj, Joel E. Kleinman, Thomas M. Hyde, Stephanie C. Page, Stephanie C. Hicks, Keri Martinowich, Kristen R. Maynard, Leonardo Collado-Torres
{"title":"Influence of Alzheimer's Disease Related Neuropathology on Local Microenvironment Gene Expression in the Human Inferior Temporal Cortex","authors":"Sang Ho Kwon, Sowmya Parthiban, Madhavi Tippani, Heena R. Divecha, Nicholas J. Eagles, Jashandeep S. Lobana, Stephen R. Williams, Michelle Mak, Rahul A. Bharadwaj, Joel E. Kleinman, Thomas M. Hyde, Stephanie C. Page, Stephanie C. Hicks, Keri Martinowich, Kristen R. Maynard, Leonardo Collado-Torres","doi":"10.1089/genbio.2023.0019","DOIUrl":"https://doi.org/10.1089/genbio.2023.0019","url":null,"abstract":"Neuropathological lesions in the brains of individuals affected with neurodegenerative disorders are hypothesized to trigger molecular and cellular processes that disturb the homeostasis of local microenvironments. Here, we applied the 10x Genomics Visium Spatial Proteogenomics (Visium-SPG) platform, which couples spatial gene expression with immunofluorescence (IF) protein co-detection, to evaluate its ability to quantify changes in spatial gene expression with respect to amyloid-beta (Aβ) and hyperphosphorylated tau (pTau) pathology in post-mortem human brain tissue from individuals with Alzheimer's disease (AD). We identified transcriptomic signatures associated with proximity to Aβ in the human inferior temporal cortex during late-stage AD, which we further investigated at cellular resolution with combined IF and single-molecule fluorescent in situ hybridization (smFISH). The study provides a data analysis workflow for Visium-SPG, and the data represent a proof-of-principle for the power of multi-omic profiling in identifying changes in molecular dynamics that are spatially associated with pathology in the human brain. We provide the scientific community with web-based, interactive resources to access the datasets of the spatially resolved AD-related transcriptomes.","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"135 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135809897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Tissue Spatial Omics Dissects Organoid Biomimicry 组织空间组学解剖类器官仿生学
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.0039
Nicholas Zhang, Denis Ohlstrom, Sicheng Pang, Nivik Sanjay Bharadwaj, Aaron Qu, Hans Grossniklaus, Ahmet F. Coskun
{"title":"Tissue Spatial Omics Dissects Organoid Biomimicry","authors":"Nicholas Zhang, Denis Ohlstrom, Sicheng Pang, Nivik Sanjay Bharadwaj, Aaron Qu, Hans Grossniklaus, Ahmet F. Coskun","doi":"10.1089/genbio.2023.0039","DOIUrl":"https://doi.org/10.1089/genbio.2023.0039","url":null,"abstract":"Recently, organoids, or three-dimensional (3D) cellular assemblies, have demonstrated promise as viable models for organ development and disease study. In contrast to challenging preclinical models, organoids are advantageous due to rapid fabrication times and greater patient specificity. The advent of spatial transcriptomics and single cell technologies has also enhanced the characterization of intraorganoid heterogeneity, thus highlighting 3D cell signaling and organ development at micro scales. In this study, we describe ongoing and future directions in spatial omics integrated with various imaging technologies for two-dimensional/3D organoid characterization. Utilizing both retinal organoids and native retinal tissues, we undertook an analysis to deconstruct the cellular compositions and structural attributes of their respective cell layers. Our findings indicate that the spatial organization of cell phenotypes is similar between organoids and native retinal tissue. However, it is noteworthy that native retinal tissue possesses thinner yet distinctly separated cell layers compared with the organoids.","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"49 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135810501","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Spatial Delivery 空间交付
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.29117.editorial
Rong Fan, Fay Lin
{"title":"Spatial Delivery","authors":"Rong Fan, Fay Lin","doi":"10.1089/genbio.2023.29117.editorial","DOIUrl":"https://doi.org/10.1089/genbio.2023.29117.editorial","url":null,"abstract":"GEN BiotechnologyVol. 2, No. 5 Guest Editorial: Spatial OmicsFree AccessSpatial DeliveryRong Fan and Fay LinRong Fan*Address correspondence to: Rong Fan E-mail Address: [email protected]Department of Biomedical Engineering, Yale University, New Haven, Connecticut, USA.Guest Editor, GEN Biotechnology.Search for more papers by this author and Fay Lin*Address correspondence to: Fay Lin E-mail Address: [email protected]Senior Editor, GEN Biotechnology.Search for more papers by this authorPublished Online:16 Oct 2023https://doi.org/10.1089/genbio.2023.29117.editorialAboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail Spatial omics enables the profiling of a variety of biomolecules with high spatial resolution across the central dogma of molecular biology directly in the natural tissue context. It has emerged as a powerful tool to analyze clinical samples for human biology research, therapeutic discovery, and translational medicine. As one of the fastest growing areas in the biotech industry, spatial omics is poised to drive the next biology revolution with broad impact across life science and medicine.In this debut special issue of GEN Biotechnology, we are delighted to feature a collection of spatial omics perspectives, reviews, and original research articles capturing the breadth of the field from cancer research to the newest advances in imaging methods.Lift OffThe historical roots of visualizing biological function date back 300 years with the invention of the compound microscope by Robert Hooke. Individual cells were seen for the first time in a plant leaf, and researchers could already visualize highly heterogeneous cell morphology implicated in distinct functions in various tissue regions. While the modern era of molecular and cell biology associates morphological heterogeneity with differential gene expression, it was the rise of single-cell genomewide gene expression measured by next-generation sequencing (NGS) platforms that allowed for detailed quantification of cellular heterogeneity in gene expression. Further breakthroughs in massively parallel single-cell sequencing via cellular barcoding enabled the gene expression profiling of thousands of single cells, thereby dissecting cell types and states in large cell populations. But despite these major breakthroughs in single-cell omics, analyzing cellular heterogeneity in the tissue context remained a challenge.Over the past decade, we have witnessed the exponential growth of an emerging field—spatial omics. The goal is to map genomewide biomolecular information pixel-by-pixel in undissociated tissue to yield a holistic view of cell type, state, and function in the native tissue context. Broadly speaking, there are two avenues to achieve this goal—one based on imaging, and the other based on NGS.Imaging-based spatial omicsAlthough single-molecule imaging and fluorescence ","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"46 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135811665","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Kallyope Is Digesting Gut–Brain Biology into Medicines Kallyope正在将肠道-大脑生物学转化为药物
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.29116.jgr
Jonathan D. Grinstein
{"title":"Kallyope Is Digesting Gut–Brain Biology into Medicines","authors":"Jonathan D. Grinstein","doi":"10.1089/genbio.2023.29116.jgr","DOIUrl":"https://doi.org/10.1089/genbio.2023.29116.jgr","url":null,"abstract":"GEN BiotechnologyVol. 2, No. 5 News FeaturesFree AccessKallyope Is Digesting Gut–Brain Biology into MedicinesJonathan D. GrinsteinJonathan D. GrinsteinE-mail Address: [email protected]Senior Editor, GEN Media Group.Search for more papers by this authorPublished Online:16 Oct 2023https://doi.org/10.1089/genbio.2023.29116.jgrAboutSectionsPDF/EPUB Permissions & CitationsPermissionsDownload CitationsTrack CitationsAdd to favorites Back To Publication ShareShare onFacebookTwitterLinked InRedditEmail Founded by Columbia University stalwarts Charles Zuker, Richard Axel, and Tom Maniatis, the New York City company is advancing a portfolio of oral small-molecule therapies across metabolism, gastrointestinal disease, and neurological disorders.Charles Zuker, Professor of Biochemistry & Molecular Biophysics and a Professor of Neuroscience at Columbia UniversityCharles Zuker had been studying taste for decades when his lab performed an experiment knocking out the receptor for sweetness in mice to test whether it would be able to distinguish sugar water from plain old water.At first, the mice lacking sweet receptors drink equal amounts of each type of water, whereas a wild-type mouse soon figures out that one of the two is sugar-laden and consequently favor the sweet one. But return 2 days later, the mice are drinking exclusively from the sugar-rich water, even if they lack the receptor for sweetness.1“We figured that there has to be some post-ingestive effect that's triggering this preference. We discovered that this maniacal desire to consume sugar—not sweet, but sugar in particular—was driven by the activation of the gut-brain circuit,” said Zuker when describing the discovery that was the basis for his a-ha moment.“That led to the idea that, my goodness, if activating the circuit can so dramatically transform an animal's behavior, then maybe accessing the gut-brain circuit could also be used to change physiology, metabolism, and so forth,” said Zuker, who is a Professor of Biochemistry and Molecular Biophysics and a Professor of Neuroscience at Columbia University and a Howard Hughes Medical Institute Investigator (Box 1).Box 1. The gut–brain axis: a critical conduit for neural signals informing the brain of the body's metabolic and physiologic stateSurvival requires the integration of external information from senses such as sight, smell, sound, touch, and taste as well as internal sensory cues from the digestive tract.2 To guarantee proper regulation of body physiological processes and behaviors and to promote overall health, informational elements, such as ingested food, energy homeostasis, inflammatory signals, and digestive progress, need to be monitored from the gut.3 The intricate network of neural, sympathetic, endocrine, immune, humoral, and gut microbiota connections—also known as the “brain–gut axis”—controls gastrointestinal homeostasis and connects the brain's emotional and cognitive centers to the gut's functions. This network enables two-wa","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"8 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135811830","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Developing Treatments for Rare Diseases on a Shoestring 小成本开发罕见疾病的治疗方法
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.0033
Ana C. Puhl, Sarah Negri, Maggie A.Z. Hupcey, Sean Ekins
{"title":"Developing Treatments for Rare Diseases on a Shoestring","authors":"Ana C. Puhl, Sarah Negri, Maggie A.Z. Hupcey, Sean Ekins","doi":"10.1089/genbio.2023.0033","DOIUrl":"https://doi.org/10.1089/genbio.2023.0033","url":null,"abstract":"There are thousands of rare genetic diseases lacking an approved treatment, many of which are life limiting to children. Those caused by a missing protein may represent a target for protein replacement either by enzyme replacement therapy or by gene therapy. One of the many challenges working on these types of genetic diseases is the availability of funding, as these diseases typically affect very small number of patients. Here we offer a novel case study of our approach to developing a treatment for one such rare disease, which has not required venture capital, angel investment, or funding by foundations to date. We have instead pursued NIH small business grants to fund the early preclinical work performed by our academic collaborators and ourselves. Our approach to developing a treatment for a rare disease on a shoestring budget is unlike any of the alternative approaches to funding.","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"53 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135273147","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
From Ultraviolet to Near-Infrared: Label-Free Reflection-Mode Hyperspectral Photoacoustic Microscopy for Single-Cell Biochemical Mapping 从紫外到近红外:用于单细胞生化制图的无标签反射模式高光谱光声显微镜
GEN biotechnology Pub Date : 2023-10-01 DOI: 10.1089/genbio.2023.0035
Qiangzhou Rong, Carlos Taboada, Ángela del Águila, Ilaria Merutka, Nishad Jayasundara, Yushun Zeng, Wei Yang, Qifa Zhou, Junjie Yao
{"title":"From Ultraviolet to Near-Infrared: Label-Free Reflection-Mode Hyperspectral Photoacoustic Microscopy for Single-Cell Biochemical Mapping","authors":"Qiangzhou Rong, Carlos Taboada, Ángela del Águila, Ilaria Merutka, Nishad Jayasundara, Yushun Zeng, Wei Yang, Qifa Zhou, Junjie Yao","doi":"10.1089/genbio.2023.0035","DOIUrl":"https://doi.org/10.1089/genbio.2023.0035","url":null,"abstract":"Hyperspectral imaging has emerged as a valuable technique for analyzing biological tissue compositions by probing intrinsic or exogenous biomolecules. However, conventional hyperspectral imaging methods predominantly rely on fluorescent signatures, limiting their application to nonfluorescent samples. To overcome this limitation, a label-free reflection-mode hyperspectral photoacoustic microscopy (RHS-PAM) system has been developed. RHS-PAM enables the imaging of thick biological samples with a wide range of intrinsic contrasts using excitation wavelengths ranging from ultraviolet to near infrared. RHS-PAM eliminates the need for tissue staining, and has achieved cellular-level spatial resolution and automatic image coregistrations at all wavelengths. Proof-of-concept applications of RHS-PAM have been demonstrated on various model organisms, including Caenorhabditis elegans, frog tadpole, zebrafish, and mouse. The technique has successfully imaged a wealth of structural and molecular features in these organisms, utilizing the optical absorption contrast of nucleic acids, proteins, hemoglobin, melanin, and lipids. The results highlight the capability of RHS-PAM to provide rich optical contrast, high spatial resolution, and an extended spectral range for label-free imaging. We believe that RHS-PAM represents a highly promising tool for single-cell biochemical mapping of diverse biological tissues.","PeriodicalId":73134,"journal":{"name":"GEN biotechnology","volume":"1 1","pages":"0"},"PeriodicalIF":0.0,"publicationDate":"2023-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"135809418","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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