Global history and clinical experiences of gene therapy: A brief review.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e17562-e17562
Author(s):  
Yi Li
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Cancer Patients ◽  
Treatment Protocol ◽  
Modern Medicine ◽  
Therapeutic Effects ◽  
Clinical Experiences ◽  
Clinical Issues ◽  
Dosage Calculation ◽  
Promising Treatment

e17562 Background: Based on the theoretic and clinical development of modern medicine, gene therapy has been a promising treatment strategy for cancer and other patients. Methods: In the present paper, we reviewed the history and clinical experiences of gene therapy from the aspects of treatment protocol, administration cycle, dosage calculation and evaluation of therapeutic effects, in order to provide more information for the further development of this promising treatment. Results: although the development of gene therapy has not gone smoothly since the beginning of the clinical trials, the results showed that this was a promising treatment for the cancer patients. There are still a lot of researches needed to be done to further solve the theoretic and clinical issues, and to improve the therapeutic effects. Conclusions: In conclusion, although the development of gene therapy has not gone smoothly since the beginning of the clinical trials, the results showed that this was a promising treatment for the cancer patients. There are still a lot of researches needed to be done to further solve the theoretic and clinical issues, and to improve the therapeutic effects. But the future of the gene therapy will be bright, and it will become a new basic strategy to help more patients.

Clinical experiences of gene therapy.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e17561-e17561
Author(s):  
Longjiang Li
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Cancer Patients ◽  
Treatment Protocol ◽  
Modern Medicine ◽  
Therapeutic Effects ◽  
Clinical Experiences ◽  
Clinical Issues ◽  
Dosage Calculation ◽  
Promising Treatment

e17561 Background: Based on the theoretic and clinical development of modern medicine, gene therapy has been a promising treatment strategy for cancer and other patients. Methods: In the present paper, we reviewed the history and clinical experiences of gene therapy from the aspects of treatment protocol, administration cycle, dosage calculation and evaluation of therapeutic effects, in order to provide more information for the further development of this promising treatment. Results: Although the development of gene therapy has not gone smoothly since the beginning of the clinical trials, the results showed that this was a promising treatment for the cancer patients. There are still a lot of researches needed to be done to further solve the theoretic and clinical issues, and to improve the therapeutic effects. Conclusions: : In conclusion, although the development of gene therapy has not gone smoothly since the beginning of the clinical trials, the results showed that this was a promising treatment for the cancer patients. There are still a lot of researches needed to be done to further solve the theoretic and clinical issues, and to improve the therapeutic effects. But the future of the gene therapy will be bright, and it will become a new basic strategy to help more patients.

scholarly journals Adenovirus Biology, Recombinant Adenovirus, and Adenovirus Usage in Gene Therapy

Viruses ◽  
2021 ◽  
Vol 13 (12) ◽  
pp. 2502
Author(s):  
Maki Watanabe ◽  
Yuya Nishikawaji ◽  
Hirotaka Kawakami ◽  
Ken-ichiro Kosai
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Regenerative Medicine ◽  
New Technologies ◽  
Recombinant Adenovirus ◽  
Methodological Principle ◽  
Therapeutic Effects ◽  
Patients With Cancer ◽  
Screening Study ◽  
Efficient Construction

Gene therapy is currently in the public spotlight. Several gene therapy products, including oncolytic virus (OV), which predominantly replicates in and kills cancer cells, and COVID-19 vaccines have recently been commercialized. Recombinant adenoviruses, including replication-defective adenoviral vector and conditionally replicating adenovirus (CRA; oncolytic adenovirus), have been extensively studied and used in clinical trials for cancer and vaccines. Here, we review the biology of wild-type adenoviruses, the methodological principle for constructing recombinant adenoviruses, therapeutic applications of recombinant adenoviruses, and new technologies in pluripotent stem cell (PSC)-based regenerative medicine. Moreover, this article describes the technology platform for efficient construction of diverse “CRAs that can specifically target tumors with multiple factors” (m-CRAs). This technology allows for modification of four parts in the adenoviral E1 region and the subsequent insertion of a therapeutic gene and promoter to enhance cancer-specific viral replication (i.e., safety) as well as therapeutic effects. The screening study using the m-CRA technology successfully identified survivin-responsive m-CRA (Surv.m-CRA) as among the best m-CRAs, and clinical trials of Surv.m-CRA are underway for patients with cancer. This article also describes new recombinant adenovirus-based technologies for solving issues in PSC-based regenerative medicine.

scholarly journals Engineered T cell therapies

2015 ◽  
Vol 17 ◽  
Author(s):  
Anne-Christine Field ◽  
Waseem Qasim
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
T Cell ◽  
Viral Vector ◽  
Ex Vivo ◽  
Immune Disorders ◽  
Clinical Experiences ◽  
Cell Therapies ◽  
Cell Gene

Alongside advancements in gene therapy for inherited immune disorders, the need for effective alternative therapeutic options for other conditions has resulted in an expansion in the field of research for T cell gene therapy. T cells are easily obtained and can be induced to divide robustly ex vivo, a characteristic that allows them to be highly permissible to viral vector-mediated introduction of transgenes. Pioneering clinical trials targeting cancers and infectious diseases have provided safety and feasibility data and important information about persistence of engineered cells in vivo. Here, we review clinical experiences with γ-retroviral and lentiviral vectors and consider the potential of integrating transposon-based vectors as well as specific genome editing with designer nucleases in engineered T cell therapies.

Treatment and secondary prevention of venous thromboembolism in cancer patients

2012 ◽  
Vol 03 (03) ◽  
pp. 121-125
Author(s):  
I. Pabinger ◽  
C. Ay
Keyword(s):  
Clinical Trials ◽  
Venous Thromboembolism ◽  
Cancer Patients ◽  
Oral Anticoagulants ◽  
Factor Xa ◽  
New Oral Anticoagulants ◽  
Phase Iii ◽  
Patients With Cancer ◽  
Potential Use

SummaryCancer is a major and independent risk factor of venous thromboembolism (VTE). In clinical practice, a high number of VTE events occurs in patients with cancer, and treatment of cancerassociated VTE differs in several aspects from treatment of VTE in the general population. However, treatment in cancer patients remains a major challenge, as the risk of recurrence of VTE as well as the risk of major bleeding during anticoagulation is substantially higher in patients with cancer than in those without cancer. In several clinical trials, different anticoagulants and regimens have been investigated for treatment of acute VTE and secondary prophylaxis in cancer patients to prevent recurrence. Based on the results of these trials, anticoagulant therapy with low-molecular-weight heparins (LMWH) has become the treatment of choice in cancer patients with acute VTE in the initial period and for extended and long-term anticoagulation for 3-6 months. New oral anticoagulants directly inhibiting thrombin or factor Xa, have been developed in the past decade and studied in large phase III clinical trials. Results from currently completed trials are promising and indicate their potential use for treatment of VTE. However, the role of the new oral thrombin and factor Xa inhibitors for VTE treatment in cancer patients still has to be clarified in further studies specifically focusing on cancer-associated VTE. This brief review will summarize the current strategies of initial and long-term VTE treatment in patients with cancer and discuss the potential use of the new oral anticoagulants.

The Novel Antipsychotic Drug Cariprazine and Cognition Enhancing Drugs: Indications for Their Use as an Add-on Therapy in Schizophrenia

2020 ◽  
Vol 26 ◽  
Author(s):  
Felix-Martin Werner ◽  
Rafael Coveñas
Keyword(s):  
Clinical Trials ◽  
Maintenance Therapy ◽  
Antipsychotic Drug ◽  
Cognitive Functions ◽  
Antipsychotic Drugs ◽  
Psychotic Disorders ◽  
Therapeutic Effects ◽  
Treatment Resistant ◽  
Long Acting ◽  
Novel Antipsychotic

Background: Schizophrenia and schizoaffective disorder are treated with antipsychotic drugs. Some patients show treatment-resistant forms of psychotic disorders and, in this case, they can be treated with clozapine. In these patients and based on previous reviews on novel antipsychotic drugs, it is important to know whether an add-on therapy with new drugs can ameliorate the positive and negative schizophrenic scale (PANSS) total score. Objective: The aim of this review is to suggest an appropriate treatment for patients with treatment-resistant forms of psychotic disorders. A combination of current available antipsychotic drugs with novel antipsychotic or modulating drugs might improve negative schizophrenic symptoms and cognitive function and thereby social functioning and quality of life. Results: The mechanisms of action, the therapeutic effects and the pharmacokinetic profiles of novel antipsychotic drugs such as cariprazine, brexipiprazole and lumateperone are up-dated. Published case reports of patients with treatmentresistant psychoses are also discussed. These patients were treated with clozapine but a high PANSS total score was observed. Only an add-on therapy with cariprazine improved the score and, above all, negative schizophrenic symptoms and cognitive functions. To ensure a constant antipsychotic drug concentration, long-acting injectable antipsychotic drugs may be a choice for a maintenance therapy in schizophrenia. New modulating drugs, such as receptor positive allosteric modulators (N-methyl-D-aspartate receptor; subtype 5 of the metabotropic glutamatergic receptor) and encenicline, an alpha7 nicotinic cholinergic receptor agonist, are being investigated in preclinical and clinical trials. Conclusion: In clinical trials, patients with treatment-resistant forms of psychosis should be examined to know whether a combination therapy with clozapine and a novel antipsychotic drug can ameliorate the PANSS total score. In schizophrenia, long-acting injectable antipsychotic drugs are a safe and tolerable maintenance therapy. In further clinical studies, it should be investigated whether patients with treatment-resistant forms of psychoses can improve negative schizophrenic symptoms and cognitive functions by an add-on therapy with cognition enhancing drugs.

Towards More Successful Gene Therapy Clinical Trials for β-Thalassemia

2013 ◽  
Vol 13 (8) ◽  
pp. 1314-1330 ◽  
Author(s):  
E. Drakopoulou ◽  
E. Papanikolaou ◽  
M. Georgomanoli ◽  
N. Anagnou
Keyword(s):  
Gene Therapy ◽  
Clinical Trials

Current RNA-based Therapeutics in Clinical Trials

2019 ◽  
Vol 19 (3) ◽  
pp. 172-196 ◽  
Author(s):  
Ling-Yan Zhou ◽  
Zhou Qin ◽  
Yang-Hui Zhu ◽  
Zhi-Yao He ◽  
Ting Xu
Keyword(s):  
Clinical Trials ◽  
Rapid Development ◽  
Genetic Diseases ◽  
Three Dimensional ◽  
Therapeutic Effects ◽  
Messenger Rnas ◽  
Small Interfering Rnas ◽  
Rna Modifications ◽  
Guide Rna ◽  
Endogenous Genes

Long-term research on various types of RNAs has led to further understanding of diverse mechanisms, which eventually resulted in the rapid development of RNA-based therapeutics as powerful tools in clinical disease treatment. Some of the developing RNA drugs obey the antisense mechanisms including antisense oligonucleotides, small interfering RNAs, microRNAs, small activating RNAs, and ribozymes. These types of RNAs could be utilized to inhibit/activate gene expression or change splicing to provide functional proteins. In the meantime, some others based on different mechanisms like modified messenger RNAs could replace the dysfunctional endogenous genes to manage some genetic diseases, and aptamers with special three-dimensional structures could bind to specific targets in a high-affinity manner. In addition, the recent most popular CRISPR-Cas technology, consisting of a crucial single guide RNA, could edit DNA directly to generate therapeutic effects. The desired results from recent clinical trials indicated the great potential of RNA-based drugs in the treatment of various diseases, but further studies on improving delivery materials and RNA modifications are required for the novel RNA-based drugs to translate to the clinic. This review focused on the advances and clinical studies of current RNA-based therapeutics, analyzed their challenges and prospects.

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