Guided by RNAscope, Powerful Outcomes in Gene Therapy

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class="field_block:paragraph:h1_2_2_text_to_media_multi_cta:field_lg_sngl_frmtd_text_global"> <div><p>Visualize AAV Vector DNA, Transgene mRNA, small RNAs along with protein biomarkers! </p></div> </div> <div class="field_block:paragraph:h1_2_2_text_to_media_multi_cta:field_call_to_action_global"> <a href="/reagents/rnascope-ish-technology/probes/custom/request-form" class="btn btn-primary btn-cta mr-3 mb-3" type="link"> Submit New Probe Request </a> </div> </div> </div> <div class="layout__region layout__region--second"> <div class="hero-content-wrap"> <div class="field_block:paragraph:h1_2_2_text_to_media_multi_cta:field_indv_media"> <div> <img src="https://resources.bio-techne.com/bio-techne-assets/images/banners/sbd-hero-cd20-lgk-red-480x480.png" alt="SBD hero foreground CD20 lgK red VS" loading="lazy" > </div> </div> </div> </div> </div> </div> </div> </div> </div> </section> </div> </div> </header> </div> <div id="main" class="container-fluid container-wide"> <div class="row"> <div class="col-md-12"> <div> <div id="block-bio-techne-global-breadcrumbs" class="system_breadcrumb_block"> <nav role="navigation" aria-labelledby="system-breadcrumb"> <h2 id="system-breadcrumb" class="visually-hidden">Breadcrumb</h2> <ol> <li> <a href="/">Home</a> </li> <li> <a href="/reagents">Bio-Techne Reagents</a> </li> <li> <a href="/reagents/rnascope-ish-technology">RNAscope ISH Technology</a> </li> <li> Guided By RNAscope, Powerful Outcomes In Gene Therapy </li> </ol> </nav> </div> </div> </div> </div> <main> <section class="section"> <div> <div id="block-bio-techne-global-content" class="system_main_block"> <article> <div> <div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><p><a href="#why-rnascope">Why Choose RNAscope™ for Gene Therapy?</a> | <a href="#empower">Empower Biodistribution and Safety Analysis</a> | <a href="#products-services">Products & Services</a> | <a href="#resources">Resources</a></p></div> </div> </div> <div> <div class="paragraph paragraph--type--section paragraph--view-mode--default mb-4"> <div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><h2 lang="EN-US">Measure Biodistribution, Cellular Tropism and Transduction Efficiency </h2><p lang="EN-US">RNAscope™ in situ hybridization (ISH) assays may be used in <a href="/research-areas/gene-therapy">gene therapy</a> to provide spatial biodistribution analysis of viral AAV vector, transgene mRNA and non-viral oligonucleotide therapeutics (ASO/siRNA) with single cell resolution. RNAscope probes can be designed to target unique regions within the promoter, regulatory elements, the transgene of a viral vector or short oligonucleotide sequences used in RNAi therapies.</p><p lang="EN-US"> </p></div> </div> </div> <div> <div class="paragraph paragraph--type--button-row paragraph--view-mode--default"> <div class="row"> <div class="col"> <div class="list-grid-wrap"> <div class="row"> <div class="col-md-4"> <a href="/reagents/rnascope-ish-technology/gene-therapy/adeno-associated-viruses" class="btn btn-primary" type="link"> AAV/Vector-based Gene Therapy </a> </div> <div class="col-md-4"> <a href="/reagents/rnascope-ish-technology/gene-therapy/aso-rnai" class="btn btn-primary" type="link"> ASO and siRNA Gene Therapy </a> </div> </div> </div> </div> </div> </div> </div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><h2 lang="EN-US"><a name="why-rnascope"></a></h2><h2 lang="EN-US">Why Choose RNAscope Technology for Gene Therapy?</h2><ul><li><strong>Visualize </strong>tissue biodistribution and cellular tropism of viral vectors such as AAV, transgene and RNA therapeutics delivered by non-viral platforms with single-molecule sensitivity and sub-cellular resolution.</li><li><strong>Select </strong>engineered vectors and capsids for highest transduction efficiency.</li><li><strong>Quantify </strong>expression and persistence of transgenes including codon-optimized or gene-edited cargo.</li><li><strong>Evaluate </strong>therapeutic efficacy including RNA efficiency within targeted cells and tissues.</li><li><strong>Improve </strong>safety by evaluating dosing and identifying off-target events in relevant organs.</li></ul><p> </p></div> </div> </div> <div> <div class="paragraph paragraph--type--full-page-text-with-media mb-4 paragraph--view-mode--default"> <div><h2>Unlock Answers to Critical Questions</h2></div> <div> <img src="https://resources.bio-techne.com/bio-techne-assets/images/applications/spatial-biology-gene-therapy-critical-questions-banner.png" alt="Illustration of the type of questions relating to gene therapy that can be answered with RNAscope" loading="lazy" > </div> </div> </div> <div> <div class="paragraph paragraph--type--header"> <div> <div><a name="empower"></a><h2>Empowering Biodistribution and Safety Analysis with Powerful RNA Visualization Tools</h2></div> </div> </div> </div> <div> <div class="paragraph paragraph--type--card-paragraph paragraph--view-mode--default card-para-default"> <div class="row card_row"> <div class="col-md-4"> <div class="h-100"> <div class="bg-white border rounded flex-column d-flex h-100"> <div class="card-img-top text-center mb-3" style="background-repeat: no-repeat; background-color: #f5f5f5; height:200px; width: 100%;"> <div class="teaser-valign-container" style=""> <div class="valign"> <img src="https://resources.bio-techne.com/bio-techne-assets/images/applications/spatial-biology-gene-therapy-view-biodistribution.png" alt="Visualize gene therapy biodistribution with RNAscope" loading="lazy" > </div> </div> </div> <h3 class="card_title_htag"> <div><h4>Visualize Biodistribution</h4></div> </h3> <div class="card_description flex-grow-1"> <div><ul> <li lang="EN-US">Optimize the targeting efficiency of your therapeutic using any preclinical animal model.</li> <li lang="EN-US">Easily measure biodistribution, cellular tropism and transduction efficiency across multiple tissues of interest with high specificity.</li> </ul></div> </div> <div class="card-bottom-link"></div> </div> </div> </div> <div class="col-md-4"> <div class="h-100"> <div class="bg-white border rounded flex-column d-flex h-100"> <div class="card-img-top text-center mb-3" style="background-repeat: no-repeat; background-color: #f5f5f5; height:200px; width: 100%;"> <div class="teaser-valign-container" style=""> <div class="valign"> <img src="https://resources.bio-techne.com/bio-techne-assets/images/applications/spatial-biology-gene-therapy-gene-expression.png" alt="Visualize single cell gene expression with RNAscope " loading="lazy" > </div> </div> </div> <h3 class="card_title_htag"> <div><h4>Single Cell Gene Expression</h4></div> </h3> <div class="card_description flex-grow-1"> <div><ul> <li lang="EN-US">Evaluate the performance of your engineered cargo <em>in vivo</em>.</li> <li lang="EN-US">Quantify the expression and persistence of your transgene or therapeutic target with single-molecule sensitivity.</li> </ul></div> </div> <div class="card-bottom-link"></div> </div> </div> </div> <div class="col-md-4"> <div class="h-100"> <div class="bg-white border rounded flex-column d-flex h-100"> <div class="card-img-top text-center mb-3" style="background-repeat: no-repeat; background-color: #f5f5f5; height:200px; width: 100%;"> <div class="teaser-valign-container" style=""> <div class="valign"> <img src="https://resources.bio-techne.com/bio-techne-assets/images/applications/spatial-biology-gene-therapy-safety-toxicity.png" alt="Visualize changes in target cells with RNAscope " loading="lazy" > </div> </div> </div> <h3 class="card_title_htag"> <div><h4>Functional Impact</h4></div> </h3> <div class="card_description flex-grow-1"> <div><ul> <li lang="EN-US">Demonstrate the functional efficacy and safety in disease models.</li> <li lang="EN-US">Visualize changes in targeted cells and tissues in spatial context using both protein and RNA biomarkers.</li> <li lang="EN-US">Monitor off target events by visualizing biodistribution of the therapy in various organs.</li> </ul></div> </div> <div class="card-bottom-link"></div> </div> </div> </div> </div> </div> </div> <div> <div class="paragraph paragraph--type--header"> <div> <div><h2>More Informative than qPCR</h2></div> </div> </div> </div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><p><strong>Evaluate Subcellular Localization of Vector Delivery and Transgene Expression: </strong>Morphology-based biodistribution is extremely important where a therapy involves gene editing. While qPCR-based methods provide average values for copy number from extracted nucleic acids, the RNAscope technology provides morphology-based quantification.</p></div> </div> </div> <div> <div class="paragraph paragraph--type--_-2-text-to-media mb-4 paragraph--view-mode--default"> <div class="container-fluid container-wide"> <div class="layout layout--twocol-section layout--twocol-section--50-50"> <div class="layout__region layout__region--first"> <div class="hero-content-wrap"> <div class="field_block:paragraph:2_2_text_to_media:field_lg_sngl_frmtd_text_global"> <div><p> </p><p><em>Figure: RNAscope uniquely enables visualization of cellular uptake and cellular tropism. (A) AAV vector is primarily sequestered in the interstitial space. (B) HALO ® (Indica Labs) overlay identifies AAV-positive nuclei in red and AAV-negative nuclei in white. (C) Quantification of % of signal detected within the nuclear boundaries vs signal located outside of the nucleus.</em></p><p> </p></div> </div> </div> </div> <div class="layout__region layout__region--second"> <div class="hero-content-wrap"> <div class="field_block:paragraph:2_2_text_to_media:field_indv_media"> <div> <img src="https://resources.bio-techne.com/bio-techne-assets/images/applications/spatial-biology-gene-therapy-qpcr-comparison2.png" alt="RNAscope uniquely enables visualization of cellular uptake and cellular tropism." loading="lazy" > </div> </div> </div> </div> </div> </div> </div> </div> <div> <div class="paragraph paragraph--type--full-page-text-with-media mb-4 paragraph--view-mode--default"> <div> <img src="https://resources.bio-techne.com/bio-techne-assets/images/banners/fda-recommends-biodistribution-studies-full.png" alt="FDA strongly recommends biodistribution studies to characterize engineered products (1). RNAscope in situ hybridization assay is the most powerful spatial method available within the methods provided." loading="lazy" > </div> </div> </div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><h2>RNAscope Probes Redefine Biodistribution Monitoring with Unmatched Specificity </h2><p>RNAscope is a trusted technology used in <a href="/resources/rnascope-citations">>10,000 publications</a> with multiple ordering formats to choose from. We offer ultimate flexibility in RNAscope probe design and can easily design probes specific to your proprietary therapeutic, for use in any animal model system. Our probes enable precise distinction between human transgenes and cross-species orthologs in animal models and allow accurate differentiation between codon-optimized transgenes and native sequences. </p></div> </div> </div> <div> <div class="paragraph paragraph--type--individual-button mb-4 paragraph--view-mode--default w-100"> <div class="row"> <div class="col"> <a href="/resources/rnascope-citations" class="btn btn-primary" type="link" > Search RNAscope Citations </a> </div> </div> </div> </div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><h2><a name="products-services"></a>Products and Services for Gene Therapy</h2></div> </div> </div> <div> <div class="paragraph paragraph--type--_-2-text-to-media mb-4 paragraph--view-mode--default"> <div class="container-fluid container-wide"> <div class="layout layout--twocol-section layout--twocol-section--50-50"> <div class="layout__region layout__region--first"> <div class="hero-content-wrap"> <div class="field_block:paragraph:2_2_text_to_media:field_header_2_global"> <div><h2>RNAscope Professional Assay Services </h2></div> </div> <div class="field_block:paragraph:2_2_text_to_media:field_lg_sngl_frmtd_text_global"> <div><p>Our <a href="/services/professional-assay-services-rnascope">Professional Assay Services</a> offer you direct access to the developers of RNAscope in situ<em> </em>hybridization (ISH) technology. Accelerate your journey in target validation, preclinical, and clinical development with the promise that we will provide consistent and trustworthy data to drive your projects forward.</p><p> </p><p> </p></div> </div> <div class="field_block:paragraph:2_2_text_to_media:field_call_to_action_global"> <a href="https://info.bio-techne.com/WEB-ACD-2023-07-PAS-Lead-Capture-Assay-Services-7398_01-LP-Main.html" class="btn btn-primary" type="link"> Free Project Consultation </a> </div> </div> </div> <div class="layout__region layout__region--second"> <div class="hero-content-wrap"> <div class="field_block:paragraph:2_2_text_to_media:field_indv_media"> <div> <img src="https://resources.bio-techne.com/bio-techne-assets/images/applications/rnascope-professional-assay-services.png" alt="Illustration of the GCLP-compliant RNAscope assay services available from Bio-Techne" loading="lazy" > </div> </div> </div> </div> </div> </div> </div> </div> <div> <div class="paragraph paragraph--type--freeform mb-4 paragraph--view-mode--default"> <div><h2><strong>RNAscope ISH Technology - Product Formats</strong></h2><p>Multiomic Spatial Solutions for RNA and Protein Co-detection</p><p> </p><figure class="responsive-figure-table"><table class="table table-bordered overflow-auto"><tbody><tr><td class="w-25"><h3>RNAscope</h3><p> ~20 ZZ probe pairs  Designed to > 300 nt.</p><ul><li>Detect transgene expression</li><li>Detect AAV vector DNA</li><li>Detect cellular marker mRNA or protein</li></ul><p><a href="/reagents/rnascope-ish-technology/rnascope">Learn About RNAscope</a></p></td><td class="w-25"><h3>BaseScope™</h3><p>1-3 ZZ probe pairs</p><p>Designed to ~ 50-300 nt.</p><ul><li>Detect codon-optimized genes,  splice variants, exon junctions</li><li>Detect up to single-nucleotide differences</li><li>Detect cellular marker mRNA or protein</li></ul><p><a href="/reagents/rnascope-ish-technology/basescope/manual-ls">Learn About BaseScope</a></p></td><td><h3>miRNAscope™</h3><p>Designed to ~17-50 nt.</p><ul><li>Detect small non-coding RNAs (miRNA, ASO, siRNA)</li><li>Detect smRNA and protein</li></ul><p><a href="/reagents/rnascope-ish-technology/mirnascope">Learn About miRNAscope</a></p></td><td class="w-25"><h3>RNAscope Plus</h3><p>RNA species of varied sizes.</p><ul><li>Visualize - 1 small RNA (ASO, miRNA, siRNA) + up to 3 mRNAs</li><li>Evaluate expression – of the target gene and cell specific marker genes</li><li>Detect 1 smRNA, 2 mRNA, and protein (IF)</li></ul><p><a href="/reagents/rnascope-ish-technology/mirnascope/manual-ls">Learn About RNAscope Plus</a></p></td></tr></tbody></table></figure><p> </p></div> </div> </div> <div> <div class="paragraph paragraph--type--individual-button mb-4 paragraph--view-mode--default w-100"> <div class="row"> <div class="col"> <a href="/reagents/rnascope-ish-technology/probes/custom/request-form" class="btn btn-primary" type="link" > Submit New Probe Request </a> </div> </div> </div> </div> <div> <div class="paragraph paragraph--type--header"> <div> <div><a name="resources"></a><h2>Featured Resources for Gene Therapy Research</h2></div> </div> </div> </div> <div> <div class="paragraph paragraph--type--card-paragraph paragraph--view-mode--default card-para-default"> <div class="row card_row"> <div class="col-md-4"> <div class="h-100"> <div class="bg-white border rounded flex-column d-flex h-100"> <a href="/resources/literature/rnascope-gene-therapy-brochure" > <div class="card-img-top text-center mb-3" style="background-repeat: no-repeat; 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