Gene therapy for eye as regenerative medicine? Lessons from RPE65 gene therapy for Leber's Congenital Amaurosis

2014 ◽  
Vol 56 ◽  
pp. 153-157 ◽  
Author(s):  
Elizabeth P. Rakoczy ◽  
Kristina Narfström
Keyword(s):  
Gene Therapy ◽  
Regenerative Medicine ◽  
Leber’S Congenital Amaurosis

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.

scholarly journals Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration

2010 ◽  
Vol 18 (3) ◽  
pp. 643-650 ◽  
Author(s):  
Francesca Simonelli ◽  
Albert M Maguire ◽  
Francesco Testa ◽  
Eric A Pierce ◽  
Federico Mingozzi ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Leber’S Congenital Amaurosis

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 Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

2015 ◽  
Vol 372 (20) ◽  
pp. 1887-1897 ◽  
Author(s):  
James W.B. Bainbridge ◽  
Manjit S. Mehat ◽  
Venki Sundaram ◽  
Scott J. Robbie ◽  
Susie E. Barker ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Term Effect ◽  
Long Term Effect ◽  
Leber’S Congenital Amaurosis
Sign in / Sign up

Export Citation Format

Share Document