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Updated: Jul 6, 2026

Effective and Safe Gene Delivery to the Mouse Kidney via Slow Retrograde Renal Pelvis Injection of Adeno-Associated Virus Vectors
Published on: August 1, 2025
Gene therapy for renal cancer
Yosef S Haviv1, David T Curiel
1Program in Kidney Gene Therapy, Division of Nephrology, Department of Medicine, Hadassah-Hebrew University Medical Center, Jerusalem, Israel.
Abstract:
Recent advances in understanding the molecular events associated with renal cell carcinoma (RCC) are revolutionizing the therapeutic options offered for patients with advanced-stage RCC. These targeted approaches for RCC are based primarily on antiangiogenesis and/or specific kinase inhibitors targeting the vascular-endothelial growth factor and platelet-derived growth factor receptors, Raf and mammalian target of rapamycin inhibitor. In this context, characterization of the molecular events unique to RCC is also of critical significance for gene therapy endeavors. The attributes of gene therapy for RCC may include true targeting to cancer cells, transfer of immunomodulatory or antiangiogenic genes and novel nonapoptotic cancer cell killing mechanisms. Gene therapy may thus become a promising new adjuvant modality for RCC and expand the therapeutic armamentarium against RCC. Beyond the current stage of preclinical proof of principle and toxicological analysis in animal models, the utility of RCC gene therapy will depend on safety and efficacy trials in human subjects. These trials will determine whether targeted therapy for RCC employing genome-based strategies will broaden the current therapeutic spectrum for RCC comprising kinome-based, immunomodulatory and antiangiogenesis strategies.
Insights
Molecular insights into renal cell carcinoma (RCC) are driving new targeted therapies. Gene therapy offers potential for enhanced cancer cell targeting and novel killing mechanisms, expanding treatment options for advanced RCC.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Recent molecular discoveries are transforming advanced renal cell carcinoma (RCC) treatment.
- Current targeted therapies focus on antiangiogenesis and kinase inhibitors (VEGF, PDGF, Raf, mTOR).
Purpose of the Study:
- To explore the significance of characterizing unique molecular events in RCC for gene therapy.
- To evaluate the potential of gene therapy as an adjuvant treatment for RCC.
Main Methods:
- Review of current targeted therapies and molecular understanding of RCC.
- Discussion of potential gene therapy strategies including targeting, gene transfer, and cell killing mechanisms.
Main Results:
- Gene therapy may offer true cancer cell targeting, immunomodulatory or antiangiogenic gene transfer, and novel non-apoptotic cell killing.
- Preclinical data suggests gene therapy's potential, but human trials are needed for safety and efficacy.
Conclusions:
- Gene therapy presents a promising new adjuvant modality for RCC, potentially expanding the therapeutic armamentarium.
- Future clinical trials will determine if genome-based strategies broaden the therapeutic spectrum for RCC.
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