AAV liver gene therapy-mediated inhibition of FGF23 signaling as a therapeutic strategy for X-linked hypophosphatemia

2021 ◽  
Author(s):  
Volha Zhukouskaya ◽  
Louisa Jauze ◽  
Séverine Charles ◽  
Christian Leborgne ◽  
Stéphane Hilliquin ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Therapeutic Strategy ◽  
Liver Gene

Inhibition of glioma by adenovirus KGHV500 encoding anti-p21Ras scFv and carried by cytokine-induced killer cells

2021 ◽  
pp. 153537022098676
Author(s):  
Jing Qian ◽  
Mo Yang ◽  
Qiang Feng ◽  
Xin-Yan Pan ◽  
Li-Lin Yang ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Glioma Cell ◽  
Therapeutic Strategy ◽  
Recombinant Adenovirus ◽  
Mapk Pathway ◽  
Control Treatment ◽  
Proliferation Index ◽  
Ras Gene ◽  
Cik Cells ◽  
Anti Tumor Activity

Ras gene mutation or overexpression can lead to tumorigenesis in multiple kinds of cancer, including glioma. However, no drugs targeting Ras or its expression products have been approved for clinical application thus far. Adenoviral gene therapy is a promising method for the treatment of glioma. In this study, the human glioma cell line U251 was co-cultured with recombinant adenovirus KGHV500, and the anti-tumor effects of KGHV500 were determined by MTT, scratch test, Transwell invasion, and apoptosis assays. Then, KGHV500 was delivered via the intravenous injection of CIK cells into glioma xenografts. Tumor volume, ki67 proliferation index, apoptosis levels, and anti-p21Ras scFv expression were tested to evaluate targeting ability, anti-tumor efficacy, and safety. We found that the KGHV500 exhibited anti-tumor activity in U251 cells and increased the intracellular expression of anti-p21Ras scFv compared with that in the control groups. CIK cells delivered KGHV500 to U251 glioma cell xenografts and enhanced anti-tumor activity against glioma xenografts compared to that produced by the control treatment. In conclusion, targeting Ras is a useful therapeutic strategy for gliomas and other Ras-driven cancers, and the delivery of anti-p21Ras scFv by recombinant adenovirus and CIK cells may play an essential role in the therapy of Ras-driven cancers. Impact statement For glioma treatment, gene therapy/virotherapy approach is a promising candidate. The Ras gene is reported to play a vital role in the RAS/RAF/mitogen-activated protein kinase (MAPK) pathway in gliomas. Thus, targeting the Ras gene should be a reasonable potential therapeutic method for glioma. In the present study, we used cytokine-induced killer (CIK) cells as secondary vectors to systemically deliver recombinant adenovirus KGHV500 to glioma xenografts and investigated the anti-tumor efficiency of recombinant adenovirus KGHV500 in vitro and in vivo. Our results expand evidence that targeting Ras is a useful and potential therapeutic strategy for gliomas. We believe that anti-p21Ras scFv delivered by recombinant adenovirus and CIK cells may play an important role in the therapy of Ras-driven cancers.

Liver Gene Therapy: Will Gene Therapy Deliver to the Liver Patient?

2008 ◽  
pp. 441-449
Author(s):  
N. Levicar ◽  
L. Jiao ◽  
Ph. Bachellier ◽  
D. Zaharoulis ◽  
D. Jaeck ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Liver Gene

scholarly journals Adenovirus and Immunotherapy: Advancing Cancer Treatment by Combination

Cancers ◽  
2020 ◽  
Vol 12 (5) ◽  
pp. 1295 ◽  
Author(s):  
Mizuho Sato-Dahlman ◽  
Christopher J. LaRocca ◽  
Chikako Yanagiba ◽  
Masato Yamamoto
Keyword(s):  
Gene Therapy ◽  
Cancer Treatment ◽  
Viral Vectors ◽  
Therapeutic Strategy ◽  
Tumor Antigens ◽  
Oncolytic Viruses ◽  
The Past ◽  
Oncolytic Adenoviruses ◽  
Gene Therapy Vectors ◽  
Preclinical And Clinical Studies

Gene therapy with viral vectors has significantly advanced in the past few decades, with adenovirus being one of the most commonly employed vectors for cancer gene therapy. Adenovirus vectors can be divided into 2 groups: (1) replication-deficient viruses; and (2) replication-competent, oncolytic (OVs) viruses. Replication-deficient adenoviruses have been explored as vaccine carriers and gene therapy vectors. Oncolytic adenoviruses are designed to selectively target, replicate, and directly destroy cancer cells. Additionally, virus-mediated cell lysis releases tumor antigens and induces local inflammation (e.g., immunogenic cell death), which contributes significantly to the reversal of local immune suppression and development of antitumor immune responses (“cold” tumor into “hot” tumor). There is a growing body of evidence suggesting that the host immune response may provide a critical boost for the efficacy of oncolytic virotherapy. Additionally, genetic engineering of oncolytic viruses allows local expression of immune therapeutics, thereby reducing related toxicities. Therefore, the combination of oncolytic virus and immunotherapy is an attractive therapeutic strategy for cancer treatment. In this review, we focus on adenovirus-based vectors and discuss recent progress in combination therapy of adenoviruses with immunotherapy in preclinical and clinical studies.

scholarly journals Liver Gene Therapy: Reliable and Durable?

2019 ◽  
Vol 27 (11) ◽  
pp. 1863-1864 ◽  
Author(s):  
Roland W. Herzog ◽  
Glenn F. Pierce
Keyword(s):  
Gene Therapy ◽  
Liver Gene

scholarly journals Helper-dependent adenoviral liver gene therapy protects against induced attacks and corrects protein folding stress in acute intermittent porphyria mice

2013 ◽  
Vol 22 (14) ◽  
pp. 2929-2940 ◽  
Author(s):  
Carmen Unzu ◽  
Ana Sampedro ◽  
Itsaso Mauleón ◽  
Manuela González-Aparicio ◽  
Rafael Enríquez de Salamanca ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Protein Folding ◽  
Acute Intermittent Porphyria ◽  
Liver Gene

Round two for liver gene therapy

1996 ◽  
Vol 12 (3) ◽  
pp. 232-233 ◽  
Author(s):  
James M. Wilson
Keyword(s):  
Gene Therapy ◽  
Liver Gene

scholarly journals Novel Interventional Approaches for ALI/ARDS: Cell-Based Gene Therapy

2011 ◽  
Vol 2011 ◽  
pp. 1-7 ◽  
Author(s):  
Ying-Gang Zhu ◽  
Jie-Ming Qu ◽  
Jing Zhang ◽  
Hong-Ni Jiang ◽  
Jin-Fu Xu
Keyword(s):  
Gene Therapy ◽  
Therapeutic Strategy ◽  
Distress Syndrome ◽  
Therapeutic Strategies ◽  
Severe Form ◽  
Pharmacological Treatments ◽  
Ventilator Support ◽  
Cell Lineages ◽  
Multiple Cell ◽  
Cell And Gene Therapy

Acute lung injury (ALI) and its more severe form, acute respiratory distress syndrome (ARDS), continue to be a major cause of morbidity and mortality in critically ill patients. The present therapeutic strategies for ALI/ARDS including supportive care, pharmacological treatments, and ventilator support are still controversial. More scientists are focusing on therapies involving stem cells, which have self-renewing capabilities and differentiate into multiple cell lineages, and, genomics therapy which has the potential to upregulate expression of anti-inflammatory mediators. Recently, the combination of cell and gene therapy which has been demonstrated to provide additive benefit has opened up a new chapter in therapeutic strategy and provides a basis for the development of an innovative approach for the prevention and treatment of ALI/ARDS.

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