The Gene Therapy of Glioblastoma: New Targeted Approaches

A special issue of Cancers (ISSN 2072-6694). This special issue belongs to the section "Cancer Therapy".

Deadline for manuscript submissions: 30 September 2024 | Viewed by 487

Special Issue Editors


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Guest Editor
Fondazione Pisana per la Scienza, Via Ferruccio Giovannini 13, 56017 San Giuliano Terme, Italy
Interests: glioblastoma; ex vivo models; personalized medicine
Special Issues, Collections and Topics in MDPI journals

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Guest Editor
Laboratory of Hematology, Department of Clinical and Experimental Medicine, University of Pisa, 56126 Pisa, Italy
Interests: experimental medicine

Special Issue Information

Dear Colleagues,

Glioblastoma is the most aggressive form of primary brain tumor in adults, with a median overall survival of less than 15 months. The standard of care consists of maximal safe surgical resection in combination with radio- and temozolomide (TMZ) chemotherapy. Tumors invariably recur, and there is no standard of care upon disease progression. Although the initial treatment leads to some improvement in survival, the effect is limited and more lasting therapeutic options are needed. GB is currently incurable, due to its resistance to conventional therapies and invasive nature. Despite advances in therapeutic options, the prognosis remains very poor due to the lack of safe and effective carriers that are able to specifically target tumor cells and penetrate the tumor. There is a clear need for better therapeutic options, and substantial efforts have been made to explore immunotherapy and precision oncology approaches. In contrast to other solid tumors, however, biological factors, such as the blood–brain barrier and the unique tumor and immune microenvironment, represent significant challenges in the development of novel therapies. Innovative clinical trial designs with biomarker-enrichment strategies are needed to ultimately improve the outcome of patients with glioblastoma. In dealing with the major challenges of GBM, namely intratumoral heterogeneity and invasive growth patterns, an important concept of targeted therapy emerges as a possible future direction, which is the focus on the personal aspect over the  universal aspect. Since glioblastoma occurs through the sequential acquisition of genetic alterations, gene therapy, which enables the modification of the genetic make-up of target cells, appears to be a promising approach to overcome the obstacles encountered by the current therapeutic strategies. Gene therapy is a rapidly evolving field with the ultimate goal of achieving the specific delivery of therapeutic molecules using either viral or non-viral delivery vehicles. A specific molecular profile of each individual tumor could eventually be beneficial in designing new tailored therapeutic approaches, with the ultimate quest of searching for targetable tumor markers that reflect the fundamental differences between tumor and normal cells.

Dr. Chiara Maria Mazzanti
Dr. Serena Barachini
Guest Editors

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Keywords

  • glioblastoma
  • prognostic markers
  • clinical approaches
  • gene therapy
  • gene delivery

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