# Topics and Trends in Most Cited Virus-based gene therapy research Papers, Class of 2026

*Canonical URL: https://pri.pepkio.com/top-papers/virus-based-gene-therapy-research/2026*

## What topics and trends defined most-cited Virus-based gene therapy research research in the Class of 2026?

Virus-based gene therapy is rapidly pivoting toward immuno-oncology, with massive growth in CAR T-cell therapy and CD8+ T cell infiltration. While traditional AAV vector design and systemic administration research are declining, the field is intensely focused on oncolytic viruses, intratumoral delivery, and rigorous clinical safety monitoring like aminotransferase elevation.

## At a glance

| Fact | Value |
| --- | --- |
| Field | Virus-based gene therapy research |
| Cohort label | Class of 2026 (2024 publications) |
| Papers analyzed | 6480 |
| Papers ranked | 20 |
| Top topics in ranked papers | Oncolytic virus, Adeno-Associated Virus, CAR T-cell therapy |
| Publication window | Jan 1, 2024 – Dec 31, 2024 |
| Eligibility | Research articles; reviews excluded |
| Citation window | 18 months post-publication |
| 18m citation range | 36–189 |
| Data source | OpenAlex · Retrieved July 2026 |
| License | CC BY 4.0 |

## Rankings

| Rank | Title | Authors | Corresponding authors | Affiliation | Journal | 18m citations | DOI |
| ---: | --- | --- | --- | --- | --- | ---: | --- |
| 1 | AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial | Jun Lv, Hui Wang, Xiaoting Cheng, Yuxin Chen, Daqi Wang, Longlong Zhang, Qi Cao, Honghai Tang, Shaowei Hu, Kaiyu Gao, Mengzhao Xun, Jinghan Wang, Zijing Wang, Biyun Zhu, Chong Cui, Ziwen Gao, Luo Guo, Sha Yu, Luoying Jiang, Yanbo Yin, Jiajia Zhang, Bing Chen, Wuqing Wang, Renjie Chai, Zheng-Yi Chen, Huawei Li, Yilai Shu | Yilai Shu | Fudan University, China | The Lancet | 189 | 10.1016/s0140-6736(23)02874-x |
| 2 | Risk of Second Tumors and T-Cell Lymphoma after CAR T-Cell Therapy | Mark P Hamilton, Takeshi Sugio, Troy Noordenbos, Shuyu Shi, Philip L Bulterys, Chih Long Liu, Xiaoman Kang, Mari N Olsen, Zinaida Good, Saurabh Dahiya, Matthew J Frank, Bita Sahaf, Crystal L Mackall, Dita Gratzinger, Maximilian Diehn, Ash A Alizadeh, David B Miklos | Mark P Hamilton | Stanford University, United States | New England Journal of Medicine | 138 | 10.1056/nejmoa2401361 |
| 3 | AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial | Jerry R Mendell, Francesco Muntoni, Craig M McDonald, Eugenio M Mercuri, Emma Ciafaloni, Hirofumi Komaki, Carmen Leon-Astudillo, Andrés Nascimento, Crystal Proud, Ulrike Schara-Schmidt, Aravindhan Veerapandiyan, Craig M Zaidman, Maitea Guridi, Alexander P Murphy, Carol Reid, Christoph Wandel, Damon R Asher, Eddie Darton, Stefanie Mason, Rachael A Potter, Teji Singh, Wenfei Zhang, Paulo Fontoura, Jacob S Elkins, Louise R Rodino-Klapac | Jerry R. Mendell | Nationwide Children's Hospital, United States | Nature Medicine | 136 | 10.1038/s41591-024-03304-z |
| 4 | Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo | Meirui An, Aditya Raguram, Samuel W Du, Samagya Banskota, Jessie R Davis, Gregory A Newby, Paul Z Chen, Krzysztof Palczewski, David R Liu | David R. Liu | Broad Institute of MIT and Harvard, United States | Nature Biotechnology | 106 | 10.1038/s41587-023-02078-y |
| 5 | In vivo human T cell engineering with enveloped delivery vehicles | Jennifer R Hamilton, Evelyn Chen, Barbara S Perez, Cindy R Sandoval Espinoza, Min Hyung Kang, Marena Trinidad, Wayne Ngo, Jennifer A Doudna | Jennifer A. Doudna | University of California, Berkeley, United States | Nature Biotechnology | 106 | 10.1038/s41587-023-02085-z |
| 6 | An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery | Qin Huang, Ken Y Chan, Jason Wu, Nuria R Botticello-Romero, Qingxia Zheng, Shan Lou, Casey Keyes, Alexander Svanbergsson, Jencilin Johnston, Allan Mills, Chin-Yen Lin, Pamela P Brauer, Gabrielle Clouse, Simon Pacouret, John W Harvey, Thomas Beddow, Jenna K Hurley, Isabelle G Tobey, Megan Powell, Albert T Chen, Andrew J Barry, Fatma-Elzahraa Eid, Yujia A Chan, Benjamin E Deverman | Qin Huang, Ken Y. Chan, Benjamin E. Deverman | Broad Institute of MIT and Harvard, United States | Science | 100 | 10.1126/science.adm8386 |
| 7 | Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B | Adam Cuker, Kaan Kavakli, Laurent Frenzel, Jiaan-Der Wang, Jan Astermark, Monica H Cerqueira, Alfonso Iorio, Olga Katsarou-Fasouli, Robert Klamroth, Amy D Shapiro, Cédric Hermans, Akira Ishiguro, Andrew D Leavitt, Johannes B Oldenburg, Margareth C Ozelo, Jerome Teitel, Francesca Biondo, Annie Fang, Joanne Fuiman, John McKay, Pengling Sun, John E J Rasko, Jeremy Rupon, BENEGENE-2 Trial Investigators | Adam Cuker | University of Pennsylvania, United States | New England Journal of Medicine | 75 | 10.1056/nejmoa2302982 |
| 8 | Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study | Peter A Campochiaro, Robert Avery, David M Brown, Jeffrey S Heier, Allen C Ho, Stephen M Huddleston, Glenn J Jaffe, Arshad M Khanani, Stephen Pakola, Dante J Pieramici, Charles C Wykoff, Sherri Van Everen | Peter A. Campochiaro | Johns Hopkins University School of Medicine, United States | The Lancet | 58 | 10.1016/s0140-6736(24)00310-6 |
| 9 | Directed evolution of engineered virus-like particles with improved production and transduction efficiencies | Aditya Raguram, Meirui An, Paul Z Chen, David R Liu | Aditya Raguram, David R. Liu | Broad Institute of MIT and Harvard, United States | Nature Biotechnology | 53 | 10.1038/s41587-024-02467-x |
| 10 | Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A | Alok Srivastava, Aby Abraham, Fouzia Aboobacker, Gurbind Singh, Tulasi Geevar, Uday Kulkarni, Sushil Selvarajan, Anu Korula, Rutvi Gautam Dave, Mohana Shankar, Abraham S Singh, Anbu Jeba, Navien Kumaar, Christopher Benjamin, Kavitha M Lakshmi, Vivi Miriam Srivastava, Ramachandran V Shaji, Sukesh C Nair, Harrison C Brown, Gabriela Denning, Pete Lollar, Christopher B Doering, Trent Spencer | Alok Srivastava | Christian Medical College Vellore, India | New England Journal of Medicine | 51 | 10.1056/nejmoa2410597 |
| 11 | Split intein-mediated protein trans-splicing to express large dystrophins | Hichem Tasfaout, Christine L Halbert, Timothy S McMillen, James M Allen, Theodore R Reyes, Galina V Flint, Dirk Grimm, Stephen D Hauschka, Michael Regnier, Jeffrey S Chamberlain | Hichem Tasfaout, Jeffrey S. Chamberlain | University of Washington, United States | Nature | 50 | 10.1038/s41586-024-07710-8 |
| 12 | Expression of tumor antigens within an oncolytic virus enhances the anti-tumor T cell response | Mason J Webb, Thanich Sangsuwannukul, Jacob van Vloten, Laura Evgin, Benjamin Kendall, Jason Tonne, Jill Thompson, Muriel Metko, Madelyn Moore, Maria P Chiriboga Yerovi, Michael Olin, Antonella Borgatti, Mark McNiven, Satdarshan P S Monga, Mitesh J Borad, Alan Melcher, Lewis R Roberts, Richard Vile | Richard G. Vile | Mayo Clinic, United States | Nature Communications | 46 | 10.1038/s41467-024-49286-x |
| 13 | In vivo CAR T-cell generation in nonhuman primates using lentiviral vectors displaying a multidomain fusion ligand | Christopher J Nicolai, Maura H Parker, Jim Qin, Weiliang Tang, Justin T Ulrich-Lewis, Rebecca J Gottschalk, Sara E Cooper, Susana A Hernandez Lopez, Don Parrilla, Richard S Mangio, Nolan G Ericson, Alissa H Brandes, Saluwa Umuhoza, Kathryn R Michels, Mollie M McDonnell, Lisa Y Park, Seungjin Shin, Wai-Hang Leung, Andrew M Scharenberg, Hans-Peter Kiem, Ryan P Larson, Laurie O Beitz, Byoung Y Ryu | Christopher J Nicolai | Umoja Biopharma, United States | Blood | 45 | 10.1182/blood.2024024523 |
| 14 | Combination therapy with oncolytic virus and T cells or mRNA vaccine amplifies antitumor effects | Rao Fu, Ruoyao Qi, Hualong Xiong, Xing Lei, Yao Jiang, Jinhang He, Feng Chen, Liang Zhang, Dekui Qiu, Yiyi Chen, Meifeng Nie, Xueran Guo, Yuhe Zhu, Jinlei Zhang, Mingxi Yue, Jiali Cao, Guosong Wang, Yuqiong Que, Mujing Fang, Yingbin Wang, Yixin Chen, Tong Cheng, Shengxiang Ge, Jun Zhang, Quan Yuan, Tianying Zhang, Ningshao Xia | Quan Yuan, Tianying Zhang, Ningshao Xia | Xiamen University, China | Signal Transduction and Targeted Therapy | 44 | 10.1038/s41392-024-01824-1 |
| 15 | Chimeric Antigen Receptor T Cells Targeting CD19 and GCC in Metastatic Colorectal Cancer | Naifei Chen, Chengfei Pu, Lingling Zhao, Wei Li, Chang Wang, Ruihong Zhu, Tingting Liang, Chao Niu, Xi Huang, Haiyang Tang, Yizhuo Wang, Hang Yang, Beibei Jia, Xianyang Jiang, Guiting Han, Wensheng Wang, Dongqi Chen, Yiming Wang, Eric K Rowinsky, Eugene Kennedy, Victor X Lu, Guozhen Cui, Zhao Wu, Lei Xiao, Jiuwei Cui | Lei Xiao, Jiuwei Cui | the First Hospital of Jilin University, China | JAMA Oncology | 44 | 10.1001/jamaoncol.2024.3891 |
| 16 | An oncolytic virus–T cell chimera for cancer immunotherapy | Yuxuan Chen, Xiaohong Chen, Weier Bao, Gang Liu, Wei Wei, Yuan Ping | Ping Yuan | Zhejiang University, China | Nature Biotechnology | 42 | 10.1038/s41587-023-02118-7 |
| 17 | LOAd703, an oncolytic virus-based immunostimulatory gene therapy, combined with chemotherapy for unresectable or metastatic pancreatic cancer (LOKON001): results from arm 1 of a non-randomised, single-centre, phase 1/2 study | Benjamin L Musher, Eric K Rowinsky, Brandon G Smaglo, Wasif Abidi, Mohamed Othman, Kalpesh Patel, Salmaan Jawaid, James Jing, Amanda Brisco, Ann M Leen, Mengfen Wu, Linda C Sandin, Jessica Wenthe, Emma Eriksson, Gustav J Ullenhag, Bambi Grilley, Justyna Leja-Jarblad, Susan G Hilsenbeck, Malcolm K Brenner, Angelica S I Loskog | Benjamin Musher | Baylor College of Medicine, United States | The Lancet Oncology | 40 | 10.1016/s1470-2045(24)00079-2 |
| 18 | Systematic multi-trait AAV capsid engineering for efficient gene delivery | Fatma-Elzahraa Eid, Albert T Chen, Ken Y Chan, Qin Huang, Qingxia Zheng, Isabelle G Tobey, Simon Pacouret, Pamela P Brauer, Casey Keyes, Megan Powell, Jencilin Johnston, Binhui Zhao, Kasper Lage, Alice F Tarantal, Yujia A Chan, Benjamin E Deverman | Fatma-Elzahraa Eid, Benjamin E. Deverman | Broad Institute of MIT and Harvard, United States | Nature Communications | 38 | 10.1038/s41467-024-50555-y |
| 19 | Carcinoembryonic antigen-expressing oncolytic measles virus derivative in recurrent glioblastoma: a phase 1 trial | Evanthia Galanis, Katharine E Dooley, S Keith Anderson, Cheyne B Kurokawa, Xiomara W Carrero, Joon H Uhm, Mark J Federspiel, Alexey A Leontovich, Ileana Aderca, Kimberly B Viker, Julie E Hammack, Randolph S Marks, Steven I Robinson, Derek R Johnson, Timothy J Kaufmann, Jan C Buckner, Daniel H Lachance, Terry C Burns, Caterina Giannini, Aditya Raghunathan, Ianko D Iankov, Ian F Parney | Evanthia Galanis | Mayo Clinic, United States | Nature Communications | 36 | 10.1038/s41467-023-43076-7 |
| 20 | Analytical characterization of full, intermediate, and empty AAV capsids | Aisleen McColl-Carboni, Serena Dollive, Sarah Laughlin, Rudenc Lushi, Michael MacArthur, Shanshan Zhou, Jeffrey Gagnon, Christopher A Smith, Brenda Burnham, Robert Horton, Dimpal Lata, Brianna Uga, Kalyani Natu, Emmanuela Michel, Celia Slater, Evan DaSilva, Robert Bruccoleri, Tim Kelly, James B McGivney | James B. McGivney | Oxford Biomedica (US) LLC, United States | Gene Therapy | 36 | 10.1038/s41434-024-00444-2 |

## Topic trends

### What Topics Define the Class of 2026?

The Class of 2026 for Virus-based gene therapy research is defined by a pronounced focus on oncology and the treatment of specific genetic disorders. The most dominant concept is 'Oncolytic virus', followed closely by 'Adeno-Associated Virus' (AAV) and 'gene therapy'. There is a clear clustering around cancer immunotherapy, evidenced by the prevalence of terms like 'CAR T-cell therapy', 'CD8+ T cell infiltration', 'Immune checkpoint inhibitors', 'Intratumoral injection', and the 'tumor microenvironment'. This indicates that researchers are intensely investigating how engineered viruses can be leveraged to stimulate targeted anti-tumor immune responses and overcome the immunosuppressive tumor microenvironment. Additionally, there is significant emphasis on specific clinical outcomes for monogenic diseases, highlighted by the prominence of 'Annualized Bleeding Rate', which strongly points to ongoing clinical evaluations of AAV-mediated gene therapies for hemophilia. Overall, the field is dominated by efforts to refine viral vectors for targeted delivery and to maximize therapeutic efficacy in both cancer and severe genetic bleeding disorders.

*Leading research themes*

### How Did Topics Shift from the Class of 2025 to the Class of 2026?

Between the Class of 2025 and the Class of 2026, the field experienced a dramatic shift towards advanced immuno-oncology applications and closer monitoring of clinical safety. The most explosive growth was seen in concepts related to 'CAR T-cell therapy' and 'CD8+ T cell infiltration', which surged from virtually no presence to becoming top concepts. This reflects a rapid pivot towards combining viral vectors with cell therapies and measuring precise immune responses within the tumor microenvironment. We also observe a sharp rise in clinical and safety endpoints, such as 'Hemostatic control', 'partial response', and 'Aminotransferase elevation', suggesting that many therapies have progressed into advanced clinical trials where detailed efficacy and liver toxicity are closely scrutinized. Conversely, foundational vector concepts like 'Adeno-Associated Virus Vector', 'Adeno-Associated Virus Serotype 9', and 'AAV serotypes' saw significant declines, alongside 'Systemic Administration' and 'Glioblastoma'. This pattern implies a maturation of the field: researchers are moving away from basic vector design and broad systemic delivery, focusing instead on targeted applications, precise immunological mechanisms, and rigorous clinical outcome reporting.

*How topics shifted year over year*

## Cite this ranking

```
Pepkio Research Index (PRI). Topics and Trends in Most Cited Virus-based gene therapy research Papers, Class of 2026. https://pri.pepkio.com/top-papers/virus-based-gene-therapy-research/2026. Accessed 2026-07-21.

Zheng Su, Tinsley Li, Thematic Shifts in Early-High-Impact Cancer Genomics and Diagnostics Research: A Bibliometric and Semantic Analysis. bioRxiv 2026.07.04.736459; doi: https://doi.org/10.64898/2026.07.04.736459
```