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Open research questions in Virus-based gene therapy research

132 unresolved questions extracted from the limitations and future-work sections of 343 Virus-based gene therapy research papers in our library. Each links back to the study that raised it.

What the literature leaves open

  • The cold chain storage requirement - The need for thermostable vaccine products - The challenge of disseminating critical vaccines to hard-to-reach places and populations - The technical, logistical, and economic challenges of maintaining a cold chain pathway

    Development of shelf stable formulation for adenovirus vectored vaccines and therapeutics · 2026 · DOI

  • testing the in vivo functionality of AdV vaccines challenged at 45/55 o C, - characterization of the limitations of reconstitution time, volume, and stability post reconstitution, - clinical trials to elucidate the potential of PT120-D based formulation technology

    Development of shelf stable formulation for adenovirus vectored vaccines and therapeutics · 2026 · DOI

  • The development of effective oncolytic viruses that can selectively replicate within cancer cells and stimulate a systemic antitumor immune response. The need to overcome the counterproductive host response that fosters an immunosuppressive tumor microenvironment. The challenge of translating the findings from preclinical models to human patients.

    A PRK-armed oncolytic adenovirus drives calreticulin exposure for dendritic cell licensing to prime antitumor CD8⁺ T cells and synergizes with anti-PD-1 or CAR-T therapy in colorectal cancer · 2026 · DOI

  • The lack of effective therapeutic strategies to fundamentally reshape the immunosuppressive tumor landscape in colorectal cancer. The need for novel oncolytic viruses that can selectively replicate within cancer cells, directly lyse them, and stimulate a systemic antitumor immune response.

    A PRK-armed oncolytic adenovirus drives calreticulin exposure for dendritic cell licensing to prime antitumor CD8⁺ T cells and synergizes with anti-PD-1 or CAR-T therapy in colorectal cancer · 2026 · DOI

  • investigate the transmission dynamics of CPV-2c in domestic cats, - assess the impact of vaccination on CPV-2c prevalence, - study the role of other potential risk factors in CPV-2c detection

    Molecular Detection and Phylogenetic Characterization of CPV-2c in Apparently Healthy Domestic Cats in the Guadalajara Metropolitan Area, Mexico · 2026 · DOI

  • The presence of CPV-2c in domestic cats in the Guadalajara Metropolitan Area, Mexico, had not been investigated. The phylogenetic relationships of the detected variants in domestic cats were unknown. The risk factors associated with CPV-2c detection in domestic cats were not identified.

    Molecular Detection and Phylogenetic Characterization of CPV-2c in Apparently Healthy Domestic Cats in the Guadalajara Metropolitan Area, Mexico · 2026 · DOI

  • The gap in understanding the cancer-specific gene signatures and genetic factors that give rise to selective binding by HPV capsids. The lack of knowledge on the relationship between TGF-β-induced epithelial to mesenchymal transition and the binding specificity of HPV-based drug conjugates.

    TGF-β-induced epithelial to mesenchymal transition drives proteoglycan binding specificity of HPV-based drug conjugates for cancer cells · 2026 · DOI

  • One challenge is the limited clinical data available for oncolytic virotherapy in the context of spinal and spinal cord tumors. Another challenge is the need to balance the efficacy of oncolytic virotherapy with the potential risks of neurovirulence and toxicity. The paper also mentions the challenge of delivering oncolytic viruses to the spinal cord and spinal tumors, which requires dedicated preclinical and early-phase studies.

    Virotherapy for Spinal and Spinal Cord Tumors: Current Evidence and Future Perspectives · 2026 · DOI

  • Clinical data in the spine remain scarce - The alternative lengthening of telomeres (ALT) pathway limits activity against tumors that maintain telomeres - Poor T-cell trafficking, limited tumor infiltration, and an immunosuppressive microenvironment limit CAR T-cell therapy in solid tumors

    Virotherapy for Spinal and Spinal Cord Tumors: Current Evidence and Future Perspectives · 2026 · DOI

  • The lack of continued investments and robust regulatory pathways to support the field of gene therapy. The need for new technologies and academic-industry collaborations to address the challenges facing the field. The lack of standardized reporting of safety signals from gene therapies.

    Keep up the momentum for gene therapies · 2026 · DOI

  • The limited number of studies from Africa and Latin America precludes robust regional conclusions. The lack of standardized serological assays and region-specific seroprevalence data. The need to balance the benefits of AAV-based gene therapies with the risks associated with neutralizing antibodies.

    Global seroprevalence of neutralizing and total antibodies against AAV vectors and their impact on the clinical gene therapy landscape: a scoping review · 2026 · DOI

  • the limited number of studies from Africa and Latin America precludes robust regional conclusions, - trials registered exclusively in other databases may not be fully represented

    Global seroprevalence of neutralizing and total antibodies against AAV vectors and their impact on the clinical gene therapy landscape: a scoping review · 2026 · DOI

  • exploring modifications to producer cell lines to enhance AAV yields, - examining the effects of anti-inflammatory agents on AAV vector production, - investigating the use of CRISPR-based technologies for genome editing, - developing strategies to overcome the limitations of AAV-based gene therapy

    AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies · 2025 · DOI

  • The challenge of producing AAV in large quantities. Insufficient viral titers during production. The need for scaling up vector production.

    AAV-Based Gene Therapy: Opportunities, Risks, and Scale-Up Strategies · 2025 · DOI

  • Confirmation of the findings in independent cohorts is required, - Experimental validation of the results is needed

    Cross-Cohort Integration of Blood DNA Methylation and Sepsis Transcriptomes Prioritizes TP53INP1 as a Candidate Associated with B-Cell Transcriptomic Patterns in Pediatric HAdV-7-Associated Sepsis · 2026 · DOI

  • Reduced-intensity melphalan shows early promise but raises concerns regarding the durability of engraftment, whereas treosulfan offers a potentially safer profile, although data in the autologous setting are lacking.

    Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier · 2026 · DOI

  • Busulfan remains the standard conditioning agent, supported by consistent engraftment and clinical efficacy, but its use is limited by acute and long-term toxicities, including infertility and potential genotoxicity.

    Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier · 2026 · DOI

  • The mechanisms underlying AAV-related liver toxicity remain poorly understood, posing challenges for effective prevention and intervention.

    Contaminating plasmid sequences and disrupted vector genomes in the liver following adeno-associated virus gene therapy · 2026 · DOI

  • Although HAdVs are present as pathogens and vectors, the interaction between HAdVs and the human immune system remains insufficiently studied.

    One virus—many strategies: type-specific interactions between human adenoviruses and innate immunity · 2026 · DOI

  • Current methods for resolving LVV integration patterns are technically limited by the sequencing approach applied allowing for only limited characterization of LVV integration profiles and altered host gene regulation.

    Adaptation of lentiviral vectors for viral gene therapy and their impact on host cell biology · 2026 · DOI

  • Naturally occurring species B adenoviruses also remain relatively underexplored as potential starting backbones for oncolytic virotherapy.

    Biological characterization of a clinical human adenovirus type 3 isolate with oncolytic potential · 2026 · DOI

  • Despite its clinical importance, the biological characteristics and potential biomedical applications of contemporary circulating HAdV-3 strains remain incompletely understood.

    Biological characterization of a clinical human adenovirus type 3 isolate with oncolytic potential · 2026 · DOI

  • This review highlights current advancements while addressing critical gaps in the literature, including the need for optimized delivery methods, better biomarker-based patient stratification, and a deeper understanding of GBM’s immunosuppressive microenvironment.

    Oncolytic Therapies for Glioblastoma: Advances, Challenges, and Future Perspectives · 2025 · DOI

  • Non-viral oncolytic approaches, such as tumor-targeting bacteria and synthetic peptides, remain underexplored.

    Oncolytic Therapies for Glioblastoma: Advances, Challenges, and Future Perspectives · 2025 · DOI

  • Abstract Description Fibrosarcoma is a type of rare and poorly studied cancer associated with a ∼40% survival rate at five years after diagnosis.

    Oncolytic virotherapy and chemotherapeutic approaches against fibrosarcoma 2928 · 2025 · DOI

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132 open questions have been extracted from the limitations and future-work passages of 343 Virus-based gene therapy research papers in our library. Each one below links back to the study that raised it, so you can read the original claim in context.

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