scholarly journals Understanding and Prevention of “Therapy-” Induced Dyskinesias

2012 ◽  
Vol 2012 ◽  
pp. 1-9 ◽  
Author(s):  
Iciar Aviles-Olmos ◽  
Zinovia Kefalopoulou ◽  
Thomas Foltynie
Keyword(s):  
Gene Therapy ◽  
Synaptic Plasticity ◽  
Basal Ganglia ◽  
Cell Therapy ◽  
Dopamine Release ◽  
The Subject ◽  
Cell And Gene Therapy ◽  
Further Development ◽  
Gene And Cell Therapy

L-dopa is the most effective, currently available treatment for Parkinson’s disease (PD), but it leads to the development of involuntary movements known as L-dopa-induced dyskinesia (LID) in the majority of patients after long-term use. Both gene and cell therapy approaches are the subject of multiple ongoing studies as potential ways of relieving symptoms of PD without the complication of dyskinesia. However, the spectre of dyskinesia in the absence of L-dopa, the so-called “off-phase” or graft-induced dyskinesia (GID), remains a major obstacle particularly in the further development of cell therapy in PD, but it is also a concern for proponents of gene therapy approaches. LID results from nonphysiological dopamine release, supersensitivity of dopamine receptors, and consequent abnormal signalling through mechanisms of synaptic plasticity. Restoration of physiological circuitry within the basal ganglia loops is ultimately the aim of all cell and gene therapy approaches but each using distinctive strategies and accompanied by risks of exacerbation of LID or development of “off-phase”/GID. In this paper we discuss the details of what is understood regarding the development of dyskinesias with relevance to cell and gene therapy and potential strategies to minimize their occurrence.

scholarly journals Autologous Gene and Cell Therapy Provides Safe and Long-Term Curative Therapy in A Large Pig Model of Hereditary Tyrosinemia Type 1

2018 ◽  
Vol 28 (1) ◽  
pp. 79-88 ◽  
Author(s):  
Raymond D. Hickey ◽  
Clara T. Nicolas ◽  
Kari Allen ◽  
Shennen Mao ◽  
Faysal Elgilani ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Cell Therapy ◽  
Ex Vivo ◽  
Curative Therapy ◽  
Ex Vivo Gene Therapy ◽  
Tyrosinemia Type 1 ◽  
Hereditary Tyrosinemia ◽  
Gene And Cell Therapy

Orthotopic liver transplantation remains the only curative therapy for inborn errors of metabolism. Given the tremendous success for primary immunodeficiencies using ex-vivo gene therapy with lentiviral vectors, there is great interest in developing similar curative therapies for metabolic liver diseases. We have previously generated a pig model of hereditary tyrosinemia type 1 (HT1), an autosomal recessive disorder caused by deficiency of fumarylacetoacetate hydrolase (FAH). Using this model, we have demonstrated curative ex-vivo gene and cell therapy using a lentiviral vector to express FAH in autologous hepatocytes. To further evaluate the long-term clinical outcomes of this therapeutic approach, we continued to monitor one of these pigs over the course of three years. The animal continued to thrive off the protective drug NTBC, gaining weight appropriately, and maintaining sexual fecundity for the course of his life. The animal was euthanized 31 months after transplantation to perform a thorough biochemical and histological analysis. Biochemically, liver enzymes and alpha-fetoprotein levels remained normal and abhorrent metabolites specific to HT1 remained corrected. Liver histology showed no evidence of tumorigenicity and Masson’s trichrome staining revealed minimal fibrosis and no evidence of cirrhosis. FAH-immunohistochemistry revealed complete repopulation of the liver by transplanted FAH-positive cells. A complete histopathological report on other organs, including kidney, revealed no abnormalities. This study is the first to demonstrate long-term safety and efficacy of hepatocyte-directed gene therapy in a large animal model. We conclude that hepatocyte-directed ex-vivo gene therapy is a rational choice for further exploration as an alternative therapeutic approach to whole organ transplantation for metabolic liver disease, including HT1.

Chinese Medicine Protein and Peptide in Gene and Cell Therapy

2019 ◽  
Vol 20 (3) ◽  
pp. 251-264 ◽  
Author(s):  
Yinlu Feng ◽  
Zifei Yin ◽  
Daniel Zhang ◽  
Arun Srivastava ◽  
Chen Ling
Keyword(s):  
Chinese Medicine ◽  
Cell Therapy ◽  
Future Research ◽  
The Past ◽  
Promising Strategy ◽  
Wide Range ◽  
New Directions ◽  
Gene And Cell Therapy ◽  
Proteins And Peptides

The success of gene and cell therapy in clinic during the past two decades as well as our expanding ability to manipulate these biomaterials are leading to new therapeutic options for a wide range of inherited and acquired diseases. Combining conventional therapies with this emerging field is a promising strategy to treat those previously-thought untreatable diseases. Traditional Chinese medicine (TCM) has evolved for thousands of years in China and still plays an important role in human health. As part of the active ingredients of TCM, proteins and peptides have attracted long-term enthusiasm of researchers. More recently, they have been utilized in gene and cell therapy, resulting in promising novel strategies to treat both cancer and non-cancer diseases. This manuscript presents a critical review on this field, accompanied with perspectives on the challenges and new directions for future research in this emerging frontier.

scholarly journals Equitable Access to Gene Therapy: A Call to Action for the American Society of Gene and Cell Therapy

2018 ◽  
Vol 26 (12) ◽  
pp. 2715-2716 ◽  
Author(s):  
Kenneth Cornetta ◽  
Kirtika Patel ◽  
Christopher Mwaniki Wanjiku ◽  
Naftali Busakhala
Keyword(s):  
Gene Therapy ◽  
Cell Therapy ◽  
Equitable Access ◽  
Call To Action ◽  
American Society ◽  
Gene And Cell Therapy

scholarly journals A few ethical issues in translational research for gene and cell therapy

2019 ◽  
Vol 17 (1) ◽  
Author(s):  
Luciana Riva ◽  
Carlo Petrini
Keyword(s):  
Gene Therapy ◽  
Cell Therapy ◽  
Translational Research ◽  
Early Phase ◽  
Ethical Issues ◽  
The United States ◽  
Cell Therapies ◽  
Biomedical Technologies ◽  
Regulatory Approach ◽  
Gene And Cell Therapy

Abstract Background Although translational research for drug development can provide patients with valuable therapeutic resources it is not without risk, especially in the early-phase trials that present the highest degree of uncertainty. With the extraordinary evolution of biomedical technologies, a growing number of innovative products based on human cells and gene therapy are being tested and used as drugs. Their use on humans poses several challenges. Methods In this work, we discuss some ethical issues related to gene and cell therapies translational research. We focus on early-phase studies analysing the regulatory approach of Europe and the United States. We report the current recommendations and guidelines of international scientific societies and European and American regulatory authorities. Results The peculiarity of human cell- or tissue-based products and gene therapy has required the development of specific regulatory tools that must be continually updated in line with the progress of the research. The ethics of translational research for these products also requires further considerations, particularly with respect to the specificity of the associated risk profiles. Conclusions An integrated ethical approach that aims for transparency and regulation of development processes, the support of independent judgment in clinical trials and the elimination of unregulated and uncontrolled grey areas of action are necessary to move gene and cell therapy forward.

A new cell therapy sector arising from the convergence of cell and gene therapy

Cytotherapy ◽  
2013 ◽  
Vol 15 (4) ◽  
pp. S52 ◽  
Author(s):  
E.J. Culme-Seymour ◽  
S. Edwards-Parton ◽  
J. Carmen ◽  
W. Folkerts ◽  
D. Smith ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Cell Therapy ◽  
Cell And Gene Therapy

scholarly journals Shipbuilding of the Azov Fleet in 1695-1712 in the Russian historiography

2020 ◽  
pp. 84-106
Author(s):  
Timofei Vladimirovich Alekseev
Keyword(s):  
Military Industry ◽  
Shipbuilding Industry ◽  
Voronezh Region ◽  
Key Issues ◽  
The Subject ◽  
Russian Military ◽  
Further Development ◽  
Military Production

The subject of this article is the assessments of national researchers on various aspects of shipbuilding of the Azov Fleet in 1695-1712. The goal consists in revealing the key issues in development of shipbuilding industry as one of the branches of domestic military production by means of critical analysis and generalization of results of research conducted by the Russian scholars. The author examines the factors  and prerequisites for selecting Voronezh as the center of construction of the Azov fleet, the key stages and evolution of shipbuilding industry and activity in the Voronezh Region, establishment of administrative branches and role of foreign experts thereof,  results of shipbuilding of the Azov Fleet and its impact upon further development of shipbuilding industry in Russia. The novelty of this study consists in analysis of the works of national researchers dedicated to shipbuilding of the Azov Fleet for covering the problem of establishment of shipbuilding sectors of the Russian military industry, clarifying and generalizing debating points on the subject matter. The article describes the influence of the conditions of shipbuilding of the Azov Fleet upon the course of shipbuilding works and the image of forming shipbuilding industry. The factors of institutionalization of shipbuilding industry, the course of development of the first shipbuilding cluster in Russia, as well as the results of short-term and long-term shipbuilding activity of are revealed.

scholarly journals Accelerating Innovation in the Creation of Biovalue

2017 ◽  
Vol 42 (5) ◽  
pp. 925-946 ◽  
Author(s):  
John Gardner ◽  
Andrew Webster
Keyword(s):  
Gene Therapy ◽  
Regenerative Medicine ◽  
Cell Therapy ◽  
Sociotechnical Imaginaries ◽  
The Creation ◽  
Cell And Gene Therapy

The field of regenerative medicine (RM) has considerable therapeutic promise that is proving difficult to realize. As a result, governments have supported the establishment of intermediary agencies to “accelerate” innovation. This article examines in detail one such agency, the United Kingdom’s Cell and Gene Therapy Catapult (CGTC). We describe CGTC’s role as an accelerator agency and its value narrative, which combines both “health and wealth.” Drawing on the notion of sociotechnical imaginaries, we unpack the tensions within this narrative and its instantiation as the CGTC cell therapy infrastructure is built and engages with other agencies, some of which have different priorities and roles to play within the RM field.

The European Society of Gene and Cell Therapy: A Nearly 30-Year Endeavor to Make Gene Therapy a Clinical Reality

2021 ◽  
Vol 32 (19-20) ◽  
pp. 979-982
Author(s):  
Hildegard Büning ◽  
Elizabeth Wilson ◽  
Juan Bueren ◽  
Axel Schambach ◽  
Alberto Auricchio
Keyword(s):  
Gene Therapy ◽  
Cell Therapy ◽  
European Society ◽  
Clinical Reality ◽  
Gene And Cell Therapy
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