Gene therapy in regenerative medicine: latest achievements and actual directions of development

2020 ◽  
Vol XV (1) ◽  
Author(s):  
E. Slobodkina ◽  
M. Karagyaur ◽  
V. Balabanyan ◽  
P. Makarevich
Keyword(s):  
Gene Therapy ◽  
Regenerative Medicine

scholarly journals Decision Support Tools for Regenerative Medicine: Systematic Review (Preprint)

2018 ◽  
Author(s):  
Ching Lam ◽  
Edward Meinert ◽  
Abrar Alturkistani ◽  
Alison R. Carter ◽  
Jeffrey Karp ◽  
...  
Keyword(s):  
Tissue Engineering ◽  
Gene Therapy ◽  
Decision Support ◽  
Regenerative Medicine ◽  
Product Development ◽  
Cost Effective ◽  
Decision Support Tools ◽  
Cell Therapies ◽  
Allogeneic Cell Therapy ◽  
Support Tools

BACKGROUND Decisional tools have demonstrated their importance in informing manufacturing and commercial decisions in the monoclonal antibody domain. Recent approved therapies in regenerative medicine have shown great clinical benefits to patients. OBJECTIVE The objective of this review was to investigate what decisional tools are available and what issues and gaps have been raised for their use in regenerative medicine. METHODS We systematically searched MEDLINE to identify articles on decision support tools relevant to tissue engineering, and cell and gene therapy, with the aim of identifying gaps for future decisional tool development. We included published studies in English including a description of decisional tools in regenerative medicines. We extracted data using a predesigned Excel table and assessed the data both quantitatively and qualitatively. RESULTS We identified 9 articles addressing key decisions in manufacturing and product development challenges in cell therapies. The decision objectives, parameters, assumptions, and solution methods were analyzed in detail. We found that all decisional tools focused on cell therapies, and 6 of the 9 reviews focused on allogeneic cell therapy products. We identified no available tools on tissue-engineering and gene therapy products. These studies addressed key decisions in manufacturing and product development challenges in cell therapies, such as choice of technology, through modeling. CONCLUSIONS Our review identified a limited number of decisional tools. While the monoclonal antibodies and biologics decisional tool domain has been well developed and has shown great importance in driving more cost-effective manufacturing processes and better investment decisions, there is a lot to be learned in the regenerative medicine domain. There is ample space for expansion, especially with regard to autologous cell therapies, tissue engineering, and gene therapies. To consider the problem more comprehensively, the full needle-to-needle process should be modeled and evaluated.

Ethics of First-in-Human Transplantation Trials of Bioartificial Organs

2021 ◽  
Vol 66 (Special Issue) ◽  
pp. 62-63
Author(s):  
Dide de Jongh ◽  
◽  
Eline Bunnik ◽  
Emma Massey ◽  
◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Trials ◽  
Regenerative Medicine ◽  
Ethical Issues ◽  
Artificial Organs ◽  
Diabetic Patients ◽  
Bioartificial Organs ◽  
Deceased Donors ◽  
Cell And Gene Therapy

"The most effective treatment for type 1 diabetes is transplantation of either a whole pancreas from a deceased donor or islet cells derived from multiple deceased donors. However, transplantation has several limitations, including shortage of post-mortem donors and the need for post-transplant patients to use life-long immunosuppressive medication. In the last decade, the field of regenerative medicine has combined engineering and biological technologies in the attempt to regenerate organs. The European VANGUARD project aims to develop immune-protected bioartificial pancreases for transplantation into non-immunosuppressed type 1 diabetic patients. This project is creating a ‘combination product’ using cells and tissue from a variety of sources, including placentas and deceased donors. The clinical development of this complex product raises ethical questions for first-in-human (FIH) clinical trials. Under what conditions can bio-artificial organs safely are transplanted in humans for the first time? How can patients be selected, recruited and informed responsibly? In this presentation, we investigate the ethical conditions for clinical trials of bio-engineered organs, focusing inter alia on study design, subject selection, risk-benefit assessment, and informed consent. We present the results of a review of the literature on the ethics of clinical trials in regenerative medicine, cell and gene therapy and transplantation, and specify existing ethical guidance in the context of FIH transplantation trials of bioartificial organs. We conclude that this new and innovative area at the intersection of regenerative medicine, cell and gene therapy and transplantation requires adequate consideration of the ethical issues in order to guide responsible research and clinical implementation. "

scholarly journals Current Trends in Viral Gene Therapy for Human Orthopaedic Regenerative Medicine

2019 ◽  
Vol 16 (4) ◽  
pp. 345-355 ◽  
Author(s):  
Jagadeesh Kumar Venkatesan ◽  
Ana Rey-Rico ◽  
Magali Cucchiarini
Keyword(s):  
Gene Therapy ◽  
Regenerative Medicine ◽  
Viral Gene ◽  
Current Trends ◽  
Viral Gene Therapy

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.

Research on cell sources for brain cell replacement methods has gained major importance. Cell and gene therapy are potentially intriguing new domains of regenerative medicine

2021 ◽  
Author(s):  
Moataz Dowaidar
Keyword(s):  
Stem Cells ◽  
Gene Therapy ◽  
Regenerative Medicine ◽  
Brain Cell ◽  
Brain Diseases ◽  
Host Immune System ◽  
Differentiation Potential ◽  
Cell Replacement ◽  
Cell Sources ◽  
Cell And Gene Therapy

Great advances in neurodegenerative disease, cell and gene therapy have been made in recent decades. Following the recent advancement of stem cell-based neuronal therapies, including managing their differentiation potential, research on cell sources for brain cell replacement methods has gained major importance. The objective is to obtain a certain neuronal cell fate to repair and restore the injured cell function. Several cell-based therapeutic techniques that show promise in animal HD models have failed to attain a similar degree of success in human patients. Despite its poor prospects, fetal transplantation has opened the door to a potentially intriguing new domain of regenerative medicine. However, many obstacles need to be overcome before pre-differentiated stem cells can be used in clinical trials, and, in particular, ensuring that the source of stem cells has optimal differentiation potential with full integration and functional enhancement, has measurable clinical benefits with minimal impact on the host immune system, and is tumor-free. New cell, molecular, and pharmacological approaches may assist enhance neuronal survival of transplanted cells, and consequently therapy for many fatal brain diseases. Molecular approaches, on the other hand, have looked into the idea of entirely eliminating HTT utilizing RNAi in the hopes of preventing the mutant protein that produced it in the first place. In contrast, HTT's physiological significance requires the application of procedures that specifically interfere with MHTHTT. The CRISPR/Cas9 approach gives researchers the ability to inactivate the mHTT allele by deleting or editing particular regions, leading to increased knowledge of how to prevent mutation-induced toxicity. Overall, despite their appealing ability to reverse mHTT-induced toxicity, these therapies may face difficulties due to the need to modify their design for individuals in order to ensure therapeutic safety.As clinical investigations are planned, genome editing already shows promise as a potent treatment to overcome clinical HD features. While there is no certainty that HD symptomatology can be fully eased, researchers must continue to hunt for ways to diminish it because it has such profound and life-threatening effects on patients and their families. These new treatments are supposed to bring a brighter future for HD sufferers.

Cell & Gene Therapy, and Regenerative Medicine – Different Definitions

2016 ◽  
Vol 2 (2) ◽  
pp. 141-145 ◽  
Author(s):  
Chris Mason ◽  
Elisa Manzotti ◽  
Emily J Culme-Seymour
Keyword(s):  
Gene Therapy ◽  
Regenerative Medicine ◽  
Cell Gene
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