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Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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Updated: May 19, 2026

Stereotactic Adoptive Transfer of Cytotoxic Immune Cells in Murine Models of Orthotopic Human Glioblastoma Multiforme Xenografts

Stereotactic Adoptive Transfer of Cytotoxic Immune Cells in Murine Models of Orthotopic Human Glioblastoma Multiforme Xenografts

Published on: September 1, 2018

Gene therapy for brain tumors: basic developments and clinical implementation.

Hikmat Assi¹, Marianela Candolfi, Gregory Baker

¹Department of Neurosurgery, University of Michigan Medical School, MSRBII, Rm 4570, 1150 Medical Center Drive, Ann Arbor, MI 48109, USA.

Neuroscience Letters

|August 22, 2012

Summary

This summary is machine-generated.

Gene therapy offers new hope for glioblastoma multiforme (GBM). A novel combined approach using Herpes Simplex Type 1 Thymidine Kinase (TK) and Flt3L shows promise in pre-clinical models for treating this deadly brain tumor.

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Modeling Brain Tumors In Vivo Using Electroporation-Based Delivery of Plasmid DNA Representing Patient Mutation Signatures

Modeling Brain Tumors In Vivo Using Electroporation-Based Delivery of Plasmid DNA Representing Patient Mutation Signatures

Published on: June 23, 2023

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Last Updated: May 19, 2026

Stereotactic Adoptive Transfer of Cytotoxic Immune Cells in Murine Models of Orthotopic Human Glioblastoma Multiforme Xenografts

Stereotactic Adoptive Transfer of Cytotoxic Immune Cells in Murine Models of Orthotopic Human Glioblastoma Multiforme Xenografts

Published on: September 1, 2018

Modeling Brain Tumors In Vivo Using Electroporation-Based Delivery of Plasmid DNA Representing Patient Mutation Signatures

Modeling Brain Tumors In Vivo Using Electroporation-Based Delivery of Plasmid DNA Representing Patient Mutation Signatures

Published on: June 23, 2023

Area of Science:

Neuro-oncology
Gene Therapy
Immunotherapy

Background:

Glioblastoma multiforme (GBM) is an aggressive primary brain tumor with poor prognosis.
Surgical resection is limited by tumor invasiveness and location.
GBM exhibits resistance to conventional chemotherapy and radiotherapy, necessitating novel therapeutic strategies.

Purpose of the Study:

To review experimental therapies for GBM, focusing on cytotoxic and immune stimulatory approaches.
To present a novel combined gene therapy strategy for GBM treatment.
To evaluate the efficacy of a combined conditional cytotoxic and immune stimulatory therapy.

Main Methods:

Adenovirus-mediated gene delivery of Herpes Simplex Type 1 Thymidine Kinase (TK) and Flt3L.
Assessment of tumor cell death and anti-tumor immune response in pre-clinical models.
Investigation of the role of TLR2 activation in the therapeutic response.

Main Results:

Combined delivery of TK and Flt3L vectors induced glioblastoma cell death.
The therapy elicited a potent anti-tumor adaptive immune response.
TLR2 activation was identified as a requirement for the observed immune response.

Conclusions:

Combined cytotoxic and immunotherapeutic strategies represent effective approaches against GBM.
The developed gene therapy warrants further investigation in human clinical trials.
This novel approach holds promise for improving outcomes in GBM patients.