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Updated: Aug 19, 2026

Lumbar Intrathecal Injection of Gene Therapy Vectors for Central Nervous System Targeting in Mice and Rats
Published on: May 16, 2025
Intra-peritoneal administration of genetic therapies: promises and pitfalls
1Centre for Oncology and Applied Pharmacology, University of Glasgow, Cancer Research, UK Beatson Laboratories, Glasgow, UK. J.Evans@beatson.gla.ac.uk
Abstract:
Rapid advances in our understanding of the molecular basis of cancer development and progression over the past 3 decades have led to the design of new potential cancer therapies based on the expression of introduced genes into a tumor or its host. In an attempt to overcome the limitations of direct intratumoral administration of genetic therapies in patients with advanced malignant disease, loco-regional routes of delivery have been explored including the intraperitoneal route. This review highlights the potential of replication-competent oncolytic viruses, virus-mediated gene replacement, and gene-directed enzyme-prodrug strategies, as novel cancer therapies. The potential of exploiting the selectivity of the telomerase gene within cancer cells to develop gene therapy strategies is discussed and the promises and pitfalls in translating these novel therapeutics from the laboratory to the clinic are reviewed.
Insights
Novel gene therapies show promise for advanced cancers, utilizing oncolytic viruses and gene-directed enzyme-prodrug strategies. Researchers explore loco-regional delivery to overcome limitations and translate lab findings to clinical applications.
Area of Science:
- Oncology
- Molecular Biology
- Gene Therapy
Background:
- Significant progress in understanding cancer's molecular basis over 30 years.
- Development of novel cancer therapies based on gene expression.
- Limitations of direct intratumoral administration for advanced cancers.
Purpose of the Study:
- To review novel cancer therapeutics, including oncolytic viruses and gene-directed enzyme-prodrug strategies.
- To explore loco-regional delivery routes, such as intraperitoneal administration.
- To discuss the potential of telomerase gene selectivity for gene therapy and its clinical translation.
Main Methods:
- Review of replication-competent oncolytic viruses.
- Analysis of virus-mediated gene replacement strategies.
- Evaluation of gene-directed enzyme-prodrug (GDEP) therapies.
- Discussion of telomerase gene-targeted approaches.
Main Results:
- Loco-regional delivery routes, like intraperitoneal, offer alternatives to intratumoral administration.
- Oncolytic viruses, gene replacement, and GDEP strategies show therapeutic potential.
- Telomerase selectivity presents a promising avenue for cancer gene therapy.
Conclusions:
- Novel gene therapy strategies, including oncolytic viruses and GDEP, hold significant promise for advanced cancers.
- Loco-regional delivery enhances therapeutic efficacy and overcomes administration challenges.
- Further research is needed to address the challenges in translating these promising therapies from laboratory to clinic.
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