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Virus Delivery of CRISPR Guides to the Murine Prostate for Gene Alteration
Published on: April 27, 2018
Considering the potential for gene-based therapy in prostate cancer
Justin R Gregg1, Timothy C Thompson2
1Department of Urology and Health Disparities Research, The University of Texas MD Anderson Cancer Center, Houston, TX, USA. JRGregg@mdanderson.org.
Abstract:
Therapeutic gene manipulation has been at the forefront of popular scientific discussion and basic and clinical research for decades. Basic and clinical research applications of CRISPR-Cas9-based technologies and ongoing clinical trials in this area have demonstrated the potential of genome editing to cure human disease. Evaluation of research and clinical trials in gene therapy reveals a concentration of activity in prostate cancer research and practice. Multiple aspects of prostate cancer care - including anatomical considerations that enable direct tumour injections and sampling, the availability of preclinical immune-competent models and the delineation of tumour-related antigens that might provide targets for an induced immune system - make gene therapy an appealing treatment option for this common malignancy. Vaccine-based therapies that induce an immune response and new technologies exploiting CRISPR-Cas9-assisted approaches, including chimeric antigen receptor (CAR) T cell therapies, are very promising and are currently under investigation both in the laboratory and in the clinic. Although laboratory and preclinical advances have, thus far, not led to oncologically relevant outcomes in the clinic, future studies offer great promise for gene therapy to become established in prostate cancer care.
Insights
Gene therapy, including CRISPR-Cas9, shows promise for treating prostate cancer by enabling genome editing. While clinical outcomes are pending, ongoing research explores vaccine-based and CAR T cell therapies for this malignancy.
Area of Science:
- Oncology
- Genetics
- Immunology
Background:
- Therapeutic gene manipulation, particularly genome editing with CRISPR-Cas9, has advanced significantly, demonstrating potential for curing diseases.
- Prostate cancer research has a notable focus on gene therapy due to unique anatomical and immunological factors.
- Preclinical models and identified tumor antigens support gene therapy's application in prostate cancer.
Purpose of the Study:
- To review the current state and future potential of gene therapy in prostate cancer treatment.
- To highlight the advantages of prostate cancer for gene therapy interventions.
- To discuss promising gene therapy approaches like vaccines and CRISPR-Cas9-based CAR T cells.
Main Methods:
- Review of basic and clinical research on gene therapy and CRISPR-Cas9 applications.
- Evaluation of ongoing clinical trials in gene therapy for various diseases, with a focus on prostate cancer.
- Analysis of factors contributing to gene therapy's appeal in prostate cancer care.
Main Results:
- Gene therapy research, especially CRISPR-Cas9, shows potential for treating human diseases.
- Prostate cancer is a key area for gene therapy research due to specific patient and disease characteristics.
- Promising gene therapy strategies include vaccine-based and CRISPR-Cas9-assisted chimeric antigen receptor (CAR) T cell therapies.
Conclusions:
- Despite current limitations in achieving oncologically relevant outcomes, gene therapy holds significant promise for prostate cancer.
- Future research and clinical investigations are expected to establish gene therapy as a viable treatment option for prostate cancer.
- The combination of anatomical suitability, preclinical models, and targeted antigens makes prostate cancer a strong candidate for advanced gene therapies.
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