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

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Updated July 19, 2026

At a glance

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

20 papers ranked by 18-month citation count

#1 of 6,480

18918m citations

AAV1-hOTOF gene therapy for autosomal recessive deafness 9: a single-arm trial

Jun Lv et al.The Lancet202410.1016/s0140-6736(23)02874-x

Yilai ShuFudan University, China

AAV1 vectorhOTOF geneOTOFERLIN proteinDFNB9gene therapysingle-arm trialsensorineural hearing lossinner ear gene deliverycochlear hair cellssynaptic vesicle fusionauditory synapsehearing restoration

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#2 of 6,480

13818m citations

Risk of Second Tumors and T-Cell Lymphoma after CAR T-Cell Therapy

Mark P Hamilton et al.New England Journal of Medicine202410.1056/nejmoa2401361

Mark P HamiltonStanford University, United States

CAR T-cell therapySecondary malignanciesT-cell lymphomaVector Genome IntegrationAdoptive cell therapyretroviral integrationclonal hematopoiesisoncogenic integrationviral vector monitoringclonal relationshipsT-cell neoplasms

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#3 of 6,480

13618m citations

AAV gene therapy for Duchenne muscular dystrophy: the EMBARK phase 3 randomized trial

Jerry R Mendell, Francesco Muntoni et al.Nature Medicine202410.1038/s41591-024-03304-z

Jerry R. MendellNationwide Children's Hospital, United States

Duchenne Muscular DystrophyDMD geneDystrophinDelandistrogene moxeparvovecadeno-associated rh74 viral vectorAAV Gene Therapymicro-dystrophin expressionNorth Star Ambulatory AssessmentTime to Rise10-meter Walk/Runstride velocity 95th centile100-meter Walk/Run4-step ascentPROMIS MobilityUpper Extremityambulatory malesIntravenous AdministrationGenome Copies Per Kilogram

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#4 of 6,480

10618m citations

Engineered virus-like particles for transient delivery of prime editor ribonucleoprotein complexes in vivo

Meirui An et al.Nature Biotechnology202410.1038/s41587-023-02078-y

David R. LiuBroad Institute of MIT and Harvard, United States

prime editingengineered virus-like particlesPE-eVLPRibonucleoprotein complexesprime editing guide RNAsNicking sgRNAbase editor eVLPtransient deliverygenetic blindnessretinal editingvisual function rescueprotein expression restorationoff-target editing reductiontransgene integration avoidanceIn Vivo Deliverygenomic substitutionsinsertions and deletions

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#5 of 6,480

10618m citations

In vivo human T cell engineering with enveloped delivery vehicles

Jennifer R Hamilton et al.Nature Biotechnology202410.1038/s41587-023-02085-z

Jennifer A. DoudnaUniversity of California, Berkeley, United States

enveloped delivery vehiclesCas9-EDVsantibody-targeted deliveryCRISPR-Cas9 protein deliveryguide RNA deliverycell surface marker recognitionantibody fragmentsmembrane-derived particlesgenome editing in vivoCell type-specific deliveryAdeno-Associated Virusantibody-antigen interactionsHuman T cellsCAR T-cell therapyCAR-T cell generationHumanized miceMultiplexed Targetingtransient deliveryEx Vivo Gene Transfer

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#6 of 6,480

10018m citations

An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery

Qin Huang, Ken Y Chan et al.Science202410.1126/science.adm8386

Qin Huang, Ken Y. Chan, Benjamin E. DevermanBroad Institute of MIT and Harvard, United States

AAV capsidBI-hTFR1TfR1Blood-Brain Barrierbrain-wide gene deliverytranscytosishuman brain endothelial cellsAdeno-Associated Virus Serotype 9human TFRC knockin miceNeurotropismGBA1 geneGaucher diseaseParkinson's diseaseglucocerebrosidase activitycerebrospinal fluidCNS gene therapyreceptor-mediated transport

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#7 of 6,480

7518m citations

Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B

Adam Cuker et al.New England Journal of Medicine202410.1056/nejmoa2302982

Adam CukerUniversity of Pennsylvania, United States

fidanacogene elaparvovecHemophilia BAAV Gene TherapyFIX-R338Lfactor IX activityAnnualized Bleeding RateNeutralizing Antibodiesprophylactic factor IX concentrateOne-stage assayAminotransferase elevationglucocorticoid treatmentfactor IX inhibitorsGenome Copies Per Kilogram

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#8 of 6,480

5818m citations

Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study

Peter A Campochiaro et al.The Lancet202410.1016/s0140-6736(24)00310-6

Peter A. CampochiaroJohns Hopkins University School of Medicine, United States

RGX-314gene therapyWet age-related macular degenerationSubretinal Injectionphase 1/2a dose-escalation studyAnti-VEGFOcular Gene DeliveryAAV8 vectorchoroidal neovascularizationsustained VEGF inhibitionDose escalationretinal gene therapy

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#9 of 6,480

5318m citations

Directed evolution of engineered virus-like particles with improved production and transduction efficiencies

Aditya Raguram et al.Nature Biotechnology202410.1038/s41587-024-02467-x

Aditya Raguram, David R. LiuBroad Institute of MIT and Harvard, United States

engineered virus-like particleseVLPDirected evolutionbarcoded guide RNAsDNA-free eVLP-packaged cargoscapsid mutationstransduction efficiencyeVLP productionv4 eVLPsribonucleoprotein cargosgene editing agentsmammalian cell deliverycapsid structurelibrary selectionpackaging optimization

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#10 of 6,480

5118m citations

Lentiviral Gene Therapy with CD34+ Hematopoietic Cells for Hemophilia A

Alok Srivastava et al.New England Journal of Medicine202410.1056/nejmoa2410597

Alok SrivastavaChristian Medical College Vellore, India

hemophilia Alentiviral gene therapyCD34+ hematopoietic stem cellsfactor VIII expressionCD68 promotermyeloid-directed expressiontransduction enhancermyeloablative conditioningautologous HSC transplantationVector Copy NumberFactor VIII ActivityAnnualized Bleeding RateengraftmentneutropeniathrombocytopeniaOne-stage assay

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#11 of 6,480

5018m citations

Split intein-mediated protein trans-splicing to express large dystrophins

Hichem Tasfaout et al.Nature202410.1038/s41586-024-07710-8

Hichem Tasfaout, Jeffrey S. ChamberlainUniversity of Washington, United States

Split inteinprotein trans-splicingDystrophinintein-mediated protein ligationlarge protein expressionDuchenne Muscular DystrophyAAV vector deliveryN-terminal intein fragmentC-terminal intein fragmentDystrophin Restorationtherapeutic protein deliverydystrophin size limitationpost-translational protein assembly

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#12 of 6,480

4618m citations

Expression of tumor antigens within an oncolytic virus enhances the anti-tumor T cell response

Mason J Webb et al.Nature Communications202410.1038/s41467-024-49286-x

Richard G. VileMayo Clinic, United States

oncolytic vesicular stomatitis virusVSV-IFNßImmune checkpoint blockadeImmune checkpoint inhibitorsLiver cancertumor microenvironmentEffector T-Cell ResponseT cell responseCytometry by Time of Flighttumor antigen expressiondominant anti-viral T cell responsessub-dominant anti-tumor T cell responsesOncolytic virotherapyT cell recruitment and primingImmune checkpoint inhibitor resistancetumor antigen encoding

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#13 of 6,480

4518m citations

In vivo CAR T-cell generation in nonhuman primates using lentiviral vectors displaying a multidomain fusion ligand

Christopher J Nicolai et al.Blood202410.1182/blood.2024024523

Christopher J NicolaiUmoja Biopharma, United States

CAR T-cell therapyin vivo CAR T-cell generationVivoVec platformLentiviral Vectormultidomain fusion proteinT-cell activation signalscostimulatory signalsVivoVec particlesNon-human primatesanti-CD20 CARB-cell depletionin vivo transductionlymphodepleting chemotherapyB-cell malignancies

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#14 of 6,480

4418m citations

Combination therapy with oncolytic virus and T cells or mRNA vaccine amplifies antitumor effects

Rao Fu, Ruoyao Qi, Hualong Xiong, Xing Lei, Yao Jiang et al.Signal Transduction and Targeted Therapy202410.1038/s41392-024-01824-1

Quan Yuan, Tianying Zhang, Ningshao XiaXiamen University, China

Oncolytic virusrVSV-LCMVGAdoptive cell therapymRNA vaccineCombination therapytumor microenvironmentB16 tumor-bearing miceCD8+ T cellsNeutralizing Antibodiescytokine and chemokine levelsPD-1 down-regulationEffector T-Cell ResponseIntratumoral injectionIntravenous AdministrationTumor antigensT-cell recruitmentimmunosuppressive to immunosensitive transformationrefractory tumorsImmune cell infiltration

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#15 of 6,480

4418m citations

Chimeric Antigen Receptor T Cells Targeting CD19 and GCC in Metastatic Colorectal Cancer

Naifei Chen et al.JAMA Oncology202410.1001/jamaoncol.2024.3891

Lei Xiao, Jiuwei Cuithe First Hospital of Jilin University, China

CAR T-cell therapyCD19Guanylate cyclase-CGCC19 CARTmetastatic colorectal cancerautologous CAR T cellsLentiviral Vectorcytokine release syndromeobjective response rateprogression-free survivaloverall survivalpartial responserefractory colorectal cancersolid tumor immunotherapydual-target CAR T cellsDose escalation

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#16 of 6,480

4218m citations

An oncolytic virus–T cell chimera for cancer immunotherapy

Yuxuan Chen, Xiaohong Chen et al.Nature Biotechnology202410.1038/s41587-023-02118-7

Ping YuanZhejiang University, China

Oncolytic virusT cell chimeraCancer immunotherapyviral vector engineeringAdoptive cell therapytumor-infiltrating lymphocytesOncolytic virotherapyimmune cell deliverycombination immunotherapyengineered T cellsTherapeutic virusestumor microenvironment

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#17 of 6,480

4018m citations

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 et al.The Lancet Oncology202410.1016/s1470-2045(24)00079-2

Benjamin MusherBaylor College of Medicine, United States

LOAd703Oncolytic virusImmunostimulatory gene therapypancreatic cancerunresectable pancreatic cancermetastatic pancreatic cancerLOKON001phase 1/2 trialCombination therapychemotherapy combinationIntratumoral injectiontumor-directed therapyimmune activation

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#18 of 6,480

3818m citations

Systematic multi-trait AAV capsid engineering for efficient gene delivery

Fatma-Elzahraa Eid et al.Nature Communications202410.1038/s41467-024-50555-y

Fatma-Elzahraa Eid, Benjamin E. DevermanBroad Institute of MIT and Harvard, United States

Fit4FunctionCapsid Engineeringmachine learning modelssequence-to-function modelsPeptide-Modified Capsidliver-targeted capsidsmanufacturabilitycross-species predictionmacaque biodistributionhuman hepatocyte transductioncapsid library designuniform sequence space samplingmulti-trait optimizationAdeno-Associated Virus Serotype 9production yieldsmurine liver transductionin vivo screeningin vitro screeninggene therapy vectorsAdeno-Associated Virus

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#19 of 6,480

3618m citations

Carcinoembryonic antigen-expressing oncolytic measles virus derivative in recurrent glioblastoma: a phase 1 trial

Evanthia Galanis et al.Nature Communications202410.1038/s41467-023-43076-7

Evanthia GalanisMayo Clinic, United States

measles virusMV-CEAcarcinoembryonic antigenOncolytic virusGlioblastomaresection cavity administrationIntratumoral injectionviral replicationinterferon stimulated genesISG DLDA classification algorithmTumor microenvironment modulationCD8+ T cell infiltrationHumoral immune responseCellular immunityviremiaviral sheddingproinflammatory changestreatment personalization

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#20 of 6,480

3618m citations

Analytical characterization of full, intermediate, and empty AAV capsids

Aisleen McColl-Carboni et al.Gene Therapy202410.1038/s41434-024-00444-2

James B. McGivneyOxford Biomedica (US) LLC, United States

Adeno-Associated Virus Vectorfull capsidsintermediate capsidsempty capsidscapsid separation fractionationcellular transduction competitioncapsid loadvector genome titerinfectivity assaysgene expression assaysbiological activity assaysin vivo potency assaysAAV product efficacyAAV manufacturing impuritiespotency as function of capsid content

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Topic trends

Dominant research themes and year-over-year shifts in Virus-based gene therapy research

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.

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.

Methodology

PRI identifies high-impact research using a transparent, topic-agnostic framework applied consistently across scientific domains. Bibliographic records are drawn from OpenAlex, including publication dates, citation relationships, and document types.

This ranking covers the Class of 2026 cohort: journal articles published in 2024. Reviews and other non-article document types are excluded to ensure comparability.

Research impact is quantified with an 18-month post-publication citation window—the number of citing works published within 18 months of each paper's publication date. This metric captures early impact while controlling for publication age.

An LLM-based relevance classifier then reviews each candidate's title and abstract to confirm substantive alignment with the target domain. Only papers classified as relevant appear in the final ranking.

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

Full methodology GitHub bioRxiv preprint

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 2026-07-19.

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