Focused Ultrasound in CGTs |

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class="navDropToggle"></div> </li> <li> <a href="/support">Support</a> </li> </ul> <a href="asgct.org" class="px-1 is-external is-hidden-desktop">ASGCT.org</a> </div> </nav> </header> <main class="c-main" id="content"> <section class="c-breadcrumbs"> <div class="container"> <span><a href="/">Home</a> > </span> <a href="/quick-takes" title="Quick Takes" class="CMSBreadCrumbsLink">Quick Takes</a> > <span class="CMSBreadCrumbsCurrentItem">Focused Ultrasound in CGTs</span> </div> </section> <section class="section"> <div class="container"> <div class="columns"> <div class="column is-8"> <h1 class="is-like-h2">Focused Ultrasound in Cell and Gene Therapies</h1> </div> </div> <div class="columns"> <div class="column is-8"> <div class="c-leader"> <div class="c-leader-content"><p>We will be exploring how non-invasive focused ultrasound technology can enhance the delivery of cell and gene therapies in treating various diseases. This content was developed in partnership with the Focused Ultrasound Foundation.</p></div> </div> <div class="content mb-6"> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984"><a href="https://www.fusfoundation.org/" target="_blank"><img alt="" src="https://www.asgct.org/getmedia/5fc1206b-77af-4f03-a37b-ab48a41e9268/FUS-logo-(5).aspx?width=350&height=175" style="width: 350px; height: 175px; float: left; margin: 5px 10px;" /></a></p> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984"> </p> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984"> </p> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984"> </p> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984"> </p> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984"> </p> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{144}" paraid="1613902984">Listen to Quick Takes from experts in the community on the following questions (Time: 20 min):  </p> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="21" role="listitem"> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{156}" paraid="1388931310"><span style="font-size:18px;">What is focused ultrasound?  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="21" role="listitem"> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{162}" paraid="1147651296"><span style="font-size:18px;">Why is focused ultrasound a safe treatment option?  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="3" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="21" role="listitem"> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{168}" paraid="311591561"><span style="font-size:18px;">What are the latest advancements and clinical applications of focused ultrasound in brain disease treatments? </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="4" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="21" role="listitem"> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{174}" paraid="555550484"><span style="font-size:18px;">How can focused ultrasound enhance gene and cell therapy treatments for brain diseases, and what are the main challenges to making this a clinical reality? </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="5" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="21" role="listitem"> <p paraeid="{8693d51e-4613-413c-b145-0cb2216ee679}{180}" paraid="630854138"><span style="font-size:18px;">For non-brain diseases, how can focused ultrasound enhance gene and cell therapy treatments and what are the main challenges to make this a clinical reality? </span></p> </li> </ul> <p><iframe allow="accelerometer; autoplay; clipboard-write; encrypted-media; gyroscope; picture-in-picture; web-share" allowfullscreen="" frameborder="0" height="450" referrerpolicy="strict-origin-when-cross-origin" src="https://www.youtube.com/embed/94sPpXVh_GY?si=GyeumbZapzBDByec" title="YouTube video player" width="800"></iframe></p> <p><a class="c-button-primary" href="https://asgct.qualtrics.com/jfe/form/SV_7OrFIMbubh2ECCG" target="_blank">Take our survey</a></p> <h2>How it Works</h2> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{88}" paraid="2041340533">Unlike standard ultrasound used for imaging, focused ultrasound targets and treats tissues, aiming to address medical conditions like cancer and essential tremors non-invasively (meaning no medical instruments will enter the body). </p> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{102}" paraid="1699355082">Focused ultrasound involves high-frequency sound waves that interact with body tissues, either causing vibrations (mechanical effects) or heating tissues (thermal effects). Thermal effects are used to create small lesions in specific areas. </p> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{108}" paraid="1724660432">Focused ultrasound can treat essential tremors by creating tiny lesions in the brain to stop the tremors. This approach is non-invasive, does not require surgery, and avoids ionizing radiation. </p> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{114}" paraid="1140405504">Clinical trials over the past decade have demonstrated evidence for the safety of focused ultrasound. It has been FDA-approved for several applications, offering non-invasive alternatives to traditional surgeries. </p> <h2 paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{126}" paraid="1249052929">Applications of Focused Ultrasound</h2> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="13" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{134}" paraid="976050831"><span style="font-size:18px;"><strong>Prostate Cancer</strong>: Treats prostate tumors non-invasively, using a transrectal (through the rectum) approach to preserve surrounding nerves. </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="13" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{146}" paraid="2029512884"><span style="font-size:18px;"><strong>Uterine Fibroids</strong>: Ablates fibrous masses in the uterine wall that cause symptoms like bleeding or pain, using an external transabdominal (through the abdomen) approach. </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="3" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="13" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{158}" paraid="1504127370"><span style="font-size:18px;"><strong>Bone Metastasis Pain Relief</strong>: Provides palliative pain relief for bone metastasis by targeting bone tumors.  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="4" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="13" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{166}" paraid="2096201613"><span style="font-size:18px;"><strong>Liver Metastasis</strong>: Treats colorectal cancer metastasis in the liver using a method called histotripsy, given externally through the abdomen. </span></p> </li> </ul> <h2 paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{166}" paraid="2096201613">Potential and Benefits</h2> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{188}" paraid="1471626766">Focused ultrasound treatments are generally performed without incisions, anesthesia is optional, and patients avoid radiation exposure, making these therapies safer than many traditional treatments. </p> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{194}" paraid="735348330">Focused ultrasound can deliver large therapeutic compounds and cells safely across the blood-brain barrier. It enhances bioavailability in the brain while allowing precise targeting of diseased brain areas, which is critical in disorders like ALS, <a href="/disease-treatments/parkinsons-disease" target="_blank">Parkinson’s</a>, and <a href="/disease-treatments/huntingtons-disease" target="_blank">Huntington’s</a> disease. Focused ultrasound now offers a non-invasive alternative that may achieve comparable delivery efficacy without incisions. </p> <h2 paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{200}" paraid="322262654">Expanding Ultrasound for Gene Delivery Beyond the Brain</h2> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="14" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{208}" paraid="4451400"><span style="font-size:18px;">Genetic diseases affecting organs like the <strong>heart (e.g., cardiomyopathy)</strong>, <strong>muscles (e.g., <a href="/disease-treatments/muscular-dystrophy" target="_blank">muscular dystrophy</a>)</strong>, <strong>kidneys</strong>, and <strong>blood</strong> <strong>(e.g., <a href="/disease-treatments/sickle-cell-disease" target="_blank">sickle cell disease</a>)</strong> are potential targets for gene therapy.  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="14" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{230}" paraid="1221338298"><span style="font-size:18px;">Unlike sickle cell treatments that utilize genetically modified bone marrow, organ-specific therapies like those for the heart and kidney require direct gene insertion into tissues. </span></p> </li> </ul> <h2 paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{240}" paraid="1521078683">Challenges of Gene Delivery in Large Organs </h2> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="15" role="listitem"> <p paraeid="{df3fb0c0-a3a8-46c6-921e-b21a23ccbddf}{248}" paraid="1071320566"><span style="font-size:18px;"><strong>Broad Coverage:</strong> Focused ultrasound, suitable for pinpoint precision (as in the brain), is less applicable for large organ-wide gene delivery. A new approach must target the entire organ rather than a localized area.  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="15" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{1}" paraid="1370712668"><span style="font-size:18px;"><strong>Selective Targeting</strong>: Ultrasound must ensure genes enter the correct cells (e.g., heart muscle cells) while avoiding uptake by unintended cell types like blood vessel or support cells. </span></p> </li> </ul> <h2 paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{9}" paraid="1795439875">Ultrasound Techniques for Organ-Wide Gene Delivery</h2> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="16" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{17}" paraid="480609285"><span style="font-size:18px;"><strong>Whole-Organ Ultrasound Exposure</strong>: Involves using ultrasound across the entire organ rather than pinpoint-focused energy.  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="16" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{25}" paraid="348546278"><span style="font-size:18px;"><strong>Microbubble-Assisted Ultrasound</strong>: Tiny bubbles (contrast agents) vibrate in the ultrasound field to enhance gene delivery from blood vessels into targeted tissues. </span></p> </li> </ul> <h2 paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{33}" paraid="437878287">Key Research Hurdles</h2> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="17" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{41}" paraid="1594214722"><span style="font-size:18px;"><strong>3D Ultrasound Exposure</strong>: Requires generating ultrasound fields that cover the full three-dimensional structure of an organ with adequate intensity and frequency.  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="17" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{49}" paraid="2080821651"><span style="font-size:18px;"><strong>Cellular Specificity</strong>: Developing methods to guide genes to specific cells within organs (e.g., heart muscle cells) to address diverse cell types present in large organs. </span></p> </li> </ul> <h2 paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{57}" paraid="748872735">Research and Future Directions</h2> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"multilevel"}" data-listid="18" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{65}" paraid="1461379"><span style="font-size:18px;">Ongoing efforts aim to overcome the challenges of large-organ gene delivery by refining ultrasound techniques and optimizing targeting strategies. </span></p> </li> </ul> <h2 paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{65}" paraid="1461379">Continuing the Journey</h2> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{77}" paraid="385276589">Check out some helpful information to guide you through the exciting new field of Focused Ultrasound: </p> <ul role="list"> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="1" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="20" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{89}" paraid="1883585979"><span style="font-size:18px;"><a href="https://www.fusfoundation.org/" rel="noreferrer noopener" target="_blank">Focused Ultrasound Foundation</a>  </span></p> </li> <li aria-setsize="-1" data-aria-level="1" data-aria-posinset="2" data-font="Symbol" data-leveltext="" data-list-defn-props="{"335552541":1,"335559685":720,"335559991":360,"469769226":"Symbol","469769242":[8226],"469777803":"left","469777804":"","469777815":"hybridMultilevel"}" data-listid="20" role="listitem"> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{96}" paraid="895661649"><span style="font-size:18px;">Search <a href="https://clinicaltrials.gov/" rel="noreferrer noopener" target="_blank">Clinicaltrials.gov</a> for open trials researching this treatment approach  </span></p> </li> </ul> <h2 paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{96}" paraid="895661649">Call to Action</h2> <p paraeid="{a57204ad-8678-4211-ae2d-57b2728f8acb}{96}" paraid="895661649">Share this information among your community to help raise awareness and understanding of focused ultrasound. For more information on gene therapy, explore the rest of ASGCT's Patient Education site, which includes resources that are free to use by sharing on social media, embedding the video, or simply linking to this page! Please credit the American Society of Gene and Cell Therapy, or tag <a href="https://twitter.com/ASGCTherapy" rel="noreferrer noopener" target="_blank">@ASGCTherapy.</a> </p> <h2>Thank You to Our Sponsors</h2> <p>The following companies offer their generous support of ASGCT's Community Quick Takes</p> <p><a href="https://www.lexeotx.com/" target="_blank"><img alt="" src="https://asgct.org/getmedia/2dbe79d5-4ab4-4f66-98ac-c9740b8b4c52/lexeo.aspx?width=200&height=100" /></a><a href="https://www.ultragenyx.com/" target="_blank"><img alt="" src="https://asgct.org/getmedia/36ee16ef-164b-4402-b998-707be67bbdc8/ultra.aspx?width=200&height=100" /></a><a href="https://www.vrtx.com/home/"><img alt="" src="https://www.asgct.org/getmedia/50a8e037-ccbc-455a-869a-2d554afa7fc2/vertex-(3).aspx?width=200&height=100" style="width: 200px; height: 100px;" /></a></p> </div> </div> <div class="column is-4 pl-4"> <div class="c-nav-local-wrap"> <div class="asideBox"> <h3> Related Pages</h3> <hr class="is-secondary" /> <ul class="c-nav-local-list"> <li class="c-nav-local-item is-active"><a class="c-nav-local-link" href="/quick-takes/focused-ultrasound-in-cell-and-gene-therapies">Focused Ultrasound in CGTs</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/quick-takes/access-to-approved-cell-and-gene-therapies">Access to Approved CGTs</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/quick-takes/informed-consent">Informed Consent</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/quick-takes/state-of-the-field-cgt-highlights-and-approvals-20">State of the Field: CGT Highlights and Approvals of 2023</a> </li> <li class="c-nav-local-item"><a class="c-nav-local-link" href="/quick-takes/genetic-testing-and-clinical-trial-eligibility">Genetic Testing and Clinical Trial Eligibility</a> </li> </ul> </div> </div> </div> </div> </div> <div class="lastUpdatedTag"> <p>Last Updated: 11/22/2024</p> </div> </section> </main> <footer class="c-footer"> <div class="c-footer-primary"> <section class="section"> <div class="container"> <div class="columns"> <div class="column c-footer-section"> <p><img alt="" src="/PatientEducation/media/PatientEducation/logo_white.png" /></p> <p>20800 Swenson Dr., Suite 300<br /> Waukesha, WI 53186</p> </div> <div class="column is-two-fifths c-footer-section"> <ul class="c-utility-list"> <li><a href="#">Gene Therapy 101</a></li> <li><a href="#">Disease Treatments</a></li> <li><a href="#">Clinical Trials</a></li> <li><a href="/patient-journey">Patient Journey</a></li> <li><a href="/about-asgct">About</a></li> <li><a href="/support">Support</a></li> <li><a href="https://asgct.org/about/privacy-policy" target="_blank">Privacy Policy</a></li> </ul> </div> <div class="column c-footer-section"> <h3 class="mb-3">Connect with ASGCT</h3> <p class="mb-0"><span>Phone: 414</span>.278.1341</p> <p><a href="mailto:info@asgct.org">info@asgct.org</a></p> <p> <div class="c-social"> <ul class="c-social-list"> <li class="c-social-item"><a href="https://twitter.com/ASGCTherapy" target="_blank" class="c-social-link twitter"><span class="is-sr-only">Twitter</span></a></li> <li class="c-social-item"><a href="https://www.facebook.com/ASGCTherapy" target="_blank" class="c-social-link facebook"><span class="is-sr-only">Facebook</span></a></li> <li class="c-social-item"><a href="https://www.instagram.com/asgctherapy" target="_blank" class="c-social-link instagram"><span class="is-sr-only">Instagram</span></a></li> <li class="c-social-item"><a href="https://www.linkedin.com/company/asgct" target="_blank" class="c-social-link linkedin"><span class="is-sr-only">LinkedIn</span></a></li> <li class="c-social-item"><a href="https://www.youtube.com/user/ASGCT" target="_blank" class="c-social-link youtube"><span class="is-sr-only">YouTube</span></a></li> </ul> </div> </p> </div> </div> </div> </section> </div> <div class="c-footer-secondary"> <section class="section py-4"> <div class="container"> <div class="columns"> <div class="column"> <p class="is-small">© 2024 All rights reserved. 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Focused Ultrasound in CGTs |