{"id":10409,"date":"2026-07-08T11:35:32","date_gmt":"2026-07-08T18:35:32","guid":{"rendered":"https:\/\/keck.usc.edu\/news\/?p=10409"},"modified":"2026-07-08T14:43:04","modified_gmt":"2026-07-08T21:43:04","slug":"usc-researcher-longwei-liu-uses-ultrasound-and-genetic-engineering-to-develop-targeted-treatments-for-blindness-and-other-diseases","status":"publish","type":"post","link":"https:\/\/keck.usc.edu\/news\/usc-researcher-longwei-liu-uses-ultrasound-and-genetic-engineering-to-develop-targeted-treatments-for-blindness-and-other-diseases\/","title":{"rendered":"USC researcher Longwei Liu uses ultrasound and genetic engineering to develop targeted treatments for blindness and other diseases"},"content":{"rendered":"\n

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\n\n \n Campus News<\/span>\n\n\n\n<\/div>\n \n \n
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USC researcher Longwei Liu uses ultrasound and genetic engineering to develop targeted treatments for blindness and other diseases<\/h1>\n\n\n<\/div>\n \n \n
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The ophthalmology researcher and biomedical engineer aims to create cures that remotely trigger cell and gene therapies for conditions ranging from a genetic disorder that can rob infants of their vision to cancer.<\/p>\n\n\n\n<\/div>\n \n

\n Wayne Lewis<\/span>\n \n July 08, 2026<\/span>\n <\/div>\n \n \n
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Longwei Liu (Photo by Shanshan Qin)<\/figcaption>\n <\/figure>\n \n \n \n\n<\/div>\n \n\n <\/div><\/div>\n\n\n\n\n \n \n\n\n\n\n\n\n\n
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USC researcher Longwei Liu, PhD<\/a>, has made it his mission to develop a therapeutic strategy to narrowly target immunotherapy and gene editing by using focused ultrasound directed inside the body. This technology could have applications for diseases ranging from pediatric blindness to cancer.<\/p>\n

His goal is to address a hard-to-shake dynamic that shows up too often in medical care. Treatments effective against serious diseases such as cancer can be toxic for patients because they also affect healthy tissue. This is a problem seen even in many targeted therapies.<\/p>\n

Liu\u2019s solution combines ultrasound with genetic engineering to act as an \u201con-off switch,\u201d noninvasively activating treatment only when and where it\u2019s needed.<\/p>\n

\u201cTurning on only in the diseased region would maximize efficacy,\u201d said Liu, an assistant professor of ophthalmology<\/a> and biomedical engineering<\/a> at the Keck School of Medicine of USC<\/a> and USC Viterbi School of Engineering<\/a>, and a Baxter Foundation faculty fellow. \u201cAnd ensuring other organs don\u2019t receive any stimulation would make the therapy safer.\u201d<\/p>\n

Hot on the trail of biomedical advances<\/strong><\/h2>\n

To develop this new therapeutic strategy, Liu and his team piggybacked on a survival mechanism that makes our bodies resilient to hot temperatures. Environmental stress, such as heat shock, kicks off a cascade of biochemical signals that ultimately stabilize fundamental cellular processes.<\/p>\n

In this cascade, the researchers identified a key signaling protein and developed an engineered version of it to activate treatment. From there, energy from focused ultrasound generates heat that can work as an on switch for this engineered protein.<\/p>\n

\u201cEvolution gave human beings a protection mechanism when temperatures get high,\u201d Liu said. \u201cMy research repurposes that natural protection to make the cells respond to the heat generated by sound.\u201d<\/p>\n

A pilot application for this technology is gene therapy for an inherited retinal disorder known as Leber congenital amaurosis (LCA). This rare disease is a leading cause of blindness in children. Liu seeks to improve outcomes by triggering gene therapy only within the eye.<\/p>\n

\u201cWe are looking for a fundamental way to directly change the patient\u2019s genetic code, fix the mutated gene using CRISPR technology,\u201d he said.<\/p>\n

His aspirations stretch beyond any one condition, though. LCA is one of 6,000 genetic disorders in humans, and most of them lack an effective treatment.<\/p>\n

\u201cThis is a platform technology,\u201d Liu said. \u201cWe don\u2019t want to limit ourselves to one specific disease.\u201d<\/p>\n

How imaging innovations inform bold treatment strategies<\/strong><\/h2>\n

Liu earned his PhD from Tsinghua University in China in 2019 before completing postdoctoral training in real-time cellular imaging and molecular engineering at the University of California San Diego in the lab of Peter Yingxiao Wang, PhD<\/a>, who now holds USC\u2019s Dwight C. and Hildagarde E. Baum Chair in Biomedical Engineering. In 2025, Liu launched his own independent lab at the Keck School of Medicine.<\/p>\n

In previous work, Liu has demonstrated that local activation with ultrasound can enhance an immunotherapy for cancer<\/a> called CAR T cell therapy. CAR T therapy, currently approved for blood cancers, involves engineering a patient\u2019s own immune cells to express receptors that specifically target cancer.<\/p>\n

The researchers\u2019 \u201cEchoback CAR T cell\u201d technology is designed for solid cancers by using high-frequency soundwaves to drive the growth of extra targeting receptors on CAR T cells when they\u2019re near the disease site. This concentrates therapeutic activity within the tumor while reducing toxicity elsewhere.<\/p>\n

Lab experiments indicated that the approach extended the amount of time therapeutic cells stay active against prostate and brain tumors.<\/p>\n

Those results were only possible due to the groundwork laid by the researchers\u2019 work developing imaging innovations, including biosensors that light up proteins to trace cellular functions as they occur. It gave Liu unique insight into the biochemistry that controls T cells.<\/p>\n

\u201cPeople often ask how we fished out those elements in our research,\u201d he said. \u201cI knew which signaling pathways were being activated because I spent several years studying T cell behavior using our fluorescent imaging.\u201d<\/p>\n

Balancing curiosity and practicality<\/strong><\/h2>\n

The team also had to surmount another hurdle along the path to ultrasound-triggered treatments: Although the natural protective mechanism is associated with heat shock, it\u2019s also spurred by other stressors.<\/p>\n

Therefore, they needed to identify a signaling element that responds only to heat.<\/p>\n

Liu approached Wang with an unconventional idea. In addition to the typical approach of screening a library of naturally occurring signaling proteins related to the body\u2019s protection against heat stroke, why not also screen engineered ones created by their team?<\/p>\n

It turned out that one of their engineered circuits had superior heat-specific behavior.<\/p>\n

Liu is grateful for the mentorship he\u2019s received from Wang and, before that, his graduate studies adviser at Tsinghua University in China, Yanan Du, PhD.<\/p>\n

\u201cThey encouraged me to ask bold questions and gave me advice on how to make the engineering approach practical,\u201d he said. \u201cThat\u2019s the philosophy I bring to my lab. As mentors, the way we protect trainees\u2019 curiosity is by helping them step-by-step to make sure what they\u2019re doing is realistic.\u201d<\/p>\n

Vital links<\/strong><\/h2>\n

Trojan connections were a major factor in Liu\u2019s choice to launch his independent lab at USC.<\/p>\n

He also values the synergy between USC\u2019s schools of medicine and engineering, exemplified by the recent move to make the Alfred E. Mann Department of Biomedical Engineering a joint department<\/a> of the Keck School of Medicine and USC Viterbi.<\/p>\n

\u201cI was really inspired by the university\u2019s vision in bringing the schools together,\u201d Liu said.<\/p>\n

Being embedded in the medical school allows him opportunities to work with clinicians whose perspectives help shape his studies. For example, he\u2019s working to translate one of his biosensors into humans for tracking drug response, at the behest of physician colleagues.<\/p>\n

\u201cI have seen the power of engineering in medicine,\u201d Liu said. \u201cThe concept is putting myself into an environment where I have a chance to talk to medical doctors. They\u2019re the first line, seeing the patients and delivering therapies, so they know exactly what the unmet needs are.\u201d<\/p>\n\n\n\n<\/div>\n <\/div>\n\n\n <\/div><\/div>\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":176,"featured_media":10410,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"advgb_blocks_editor_width":"","advgb_blocks_columns_visual_guide":"","footnotes":"","_links_to":"","_links_to_target":""},"categories":[7],"tags":[635,627,446,169,20,520,230],"class_list":["post-10409","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-campus-news","tag-department-of-biomedical-engineering","tag-department-of-ophthalmology","tag-homepage","tag-latest","tag-research","tag-usc-roski-eye-institute","tag-usc-viterbi-school-of-engineering"],"acf":[],"yoast_head":"\nUSC researcher Longwei Liu uses ultrasound and genetic engineering to develop targeted treatments for blindness and other diseases<\/title>\n<meta name=\"description\" content=\"USC researcher Longwei Liu, PhD, has made it his mission to develop a therapeutic strategy to narrowly target immunotherapy and gene editing by using focused ultrasound directed inside the body. 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