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Biosimilar Gene Therapy: Investigational Assessment of Secukinumab Gene Therapy

Ali Fallah, Hajar Estiri, Elizabeth Parrish, Mansoureh Soleimani, Sirous Zeinali, Azita Zadeh-Vakili
2019 Cell journal  
With the current progress in stem cells and gene therapy technologies, there is the promise of replacing costly mAbs production in bioreactors with a more direct and cost-effective production method inside  ...  The engineered stem cells and recombinant viruses were applied for ex vivo and in vivo gene therapy, respectively, in different groups of rat models.  ...  Acknowledgements This study was conducted as part of the thesis for obtaining a Ph.D. degree in molecular medicine at the School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical  ... 
doi:10.22074/cellj.2020.6309 pmid:31376325 pmcid:PMC6722441 fatcat:3xmsopujcnfythfd7u7j6zdrle

Gene Therapy [chapter]

2008 Viruses and Human Disease  
If the foreign gene replaces the structural genes, it will be translated from the 5′ end of the genome.  ...  A retrovirus vector that expressed such a gene could also be useful for conventional gene therapy, because it would allow the infected cells to be killed if the infection process threatened to get out  ... 
doi:10.1016/b978-0-12-373741-0.50014-3 fatcat:eokvl5jjizd75njhtgo7bztmf4

Gene Therapy Progress and Prospects: Reprograming gene expression by trans-splicing

L G Mitchell, G J McGarrity
2005 Gene Therapy  
High-capacity screens will see wider application to improve the specificity and efficiency of trans-splicing in the complex environment of human cells.  ...  or elimination of undesirable ectopic expression; the ability to use smaller constructs that transsplice only a portion of the gene to be replaced; and the conversion of dominant-negative mutations to  ...  It represents an important step towards the universal imaging of an endogenous pre-mRNA target, with potential applications in research and diagnostics.  ... 
doi:10.1038/ pmid:16121205 fatcat:dhrwst6aubczbbjoorkcm2tp74

Fetal Gene Therapy [chapter]

Christopher Porada, Graca Almeida-Por
2011 Gene Therapy Applications  
clinically applicable FVIII-encoding gene therapy vectors would be to use an animal model that both closely resembles the disease process of HA and closely parallels normal human physiology.  ...  The fetus as a gene therapy recipient In addition to the clinical and financial advantages of performing gene therapy prior to birth, numerous aspects of the fetus make it a more suitable gene therapy  ... 
doi:10.5772/18722 fatcat:zxdgu5bqkvetpflpuewfpqblsm

Gene Therapy for Blindness

José-Alain Sahel, Botond Roska
2013 Annual Review of Neuroscience  
Our review discusses traditional approaches such as gene replacement and neuroprotection and also new avenues such as optogenetic therapies.  ...  Here we discuss the concepts and recent advances in gene therapy in the retina.  ...  ACKNOWLEDGMENTS We thank Katia Marazova, Dasha Nelidova, Deniz Dalkara, Serge Picaud, and Sara Oakeley for advice on and corrections to the manuscript.  ... 
doi:10.1146/annurev-neuro-062012-170304 pmid:23724995 fatcat:ixom4o62aff2lhy2w4gubtr6tu

Promising Role of Engineered Gene Circuits in Gene Therapy [chapter]

Wei-Dong Wang, Jinyi Lang
2011 Gene Therapy - Developments and Future Perspectives  
This system would be particularly useful in gene silencing applications, as it was shown to yield > 99% repression of a target gene.  ...  An original example for new applications that derives from the interface of engineering and life sciences, which came out of the iGEM student competition, is the image-processing E.coli.  ...  Gene Therapy -Developments and Future Perspectives The aim of this book is to cover key aspects of existing problems in the field of development and future perspectives in gene therapy.  ... 
doi:10.5772/17400 fatcat:d2pfmhudjzavrk4rd743l4esvq

Molecular imaging and gene therapy

C Nichol, E E Kim
2001 Journal of Nuclear Medicine  
The merging fields of molecular biology, molecular medicine, and imaging modalities may provide the means to screen active drugs in vivo, image molecular processes, and diagnose disease at a presymptomatic  ...  Recent advances in this field show promise, particularly in the imaging of gene expression.  ...  For these reasons, it holds promise for imaging the emerging clinical applications in gene therapy by monitoring gene delivery and identifying protein expression.  ... 
pmid:11535727 fatcat:bvbkj4wginaepaknrcaxkiywxu

Gene therapy for cardiovascular disease

D W Muller
1994 Heart  
Clinical applications of gene transfer Although originally conceived as a means of curing inherited genetic diseases by replacing mutated genes in germ cells, gene therapy will have its greatest clinical  ...  It is used to receive, replicate, and allow the selection of the genomic DNA or cDNA of interest after introduction into the host. IMAGES Figure 2 2 Retrovirus-mediated gene transfer.  ... 
doi:10.1136/hrt.72.4.309 pmid:7833184 pmcid:PMC1025535 fatcat:gv5crintnnfevdzavgbijfebf4

Transductional targeting of adenovirus vectors for gene therapy

J N Glasgow, M Everts, D T Curiel
2006 Cancer Gene Therapy  
We wish to thank Dr Long P Le for assistance with graphic images.  ...  Acknowledgements This work was supported by grants from the National Institutes of Health: R01 CA083821, R01 CA094084 and R01 CA111569-01A1, and grant W81XWH-05-1-0035 from the US Department of Defense  ...  Future applications Despite the advances in tumor-targeted gene therapy as described in this article, several obstacles remain.  ... 
doi:10.1038/sj.cgt.7700928 pmid:16439993 pmcid:PMC1781516 fatcat:42cf5mk2svec5fffyl4lfgfpoi

Gene Therapy for Cardiomyopathies [chapter]

Yves Fromes, Caroline Roques
2019 In Vivo and Ex Vivo Gene Therapy for Inherited and Non-Inherited Disorders  
as delivery of genes to replace defective or deficient components and thereby restore structure or function in a diseased heart.  ...  Cardiac gene therapy will be integrated into the therapeutic armamentarium for CM and heart failure.  ...  Acknowledgments The authors are grateful to the late Ketty Schwartz and Marc Fiszman for their great mentorship and for developing a cardiac gene therapy programme at the very early stage of the field.  ... 
doi:10.5772/intechopen.80478 fatcat:bv2xqaducvftpn5szfmiz6b6ta

Gene therapy for leukodystrophies

A. Biffi, P. Aubourg, N. Cartier
2011 Human Molecular Genetics  
Leukodystrophies (LDs) refer to a group on inherited diseases in which molecular abnormalities of glial cells are responsible for exclusive or predominant defects in myelin formation and/or maintenance  ...  For three of them [X-linked adrenoleukodystrophy (X-ALD), metachromatic (MLD) and globoid cell LDs], a gene therapy strategy aiming at transferring the disease gene into autologous hematopoietic stem cells  ...  It must be balanced with the benefit of HSC gene therapy.  ... 
doi:10.1093/hmg/ddr142 pmid:21459776 fatcat:ydhx6aenpnaszmelozsttk7mry

Cancer Gene Therapy: Targeted Genomedicines [chapter]

Yadollah Omidi, Jaleh Barar, George Coukos
2013 Novel Gene Therapy Approaches  
The MSC-based gene therapy is still in its infancy era and need much more investigation prior to its clinical applications even though the MSCs themselves are under clinical trials [117] .  ...  DNAzymes, similar to aptamers, can be isolated through a combinatorial in vitro selection process.  ...  Author details Yadollah Omidi 1,2 , Jaleh Barar 1,2 and George Coukos 1,3  ... 
doi:10.5772/54739 fatcat:h6lugupoyjhs3jax2pjqccbcfy

Conditioning and Scaffolding of Chondrocytes: Smart Steps Towards Osteoarthritis Gene Therapy [chapter]

Muhammad Farooq, Annemarie Lang, Matthias Sieber, Michael F.G.
2011 Gene Therapy Applications  
The negative impact of gene therapy can be overcome if it is suitably improved in each of its components. The first and foremost component of gene therapy is the vector.  ...  Obstacles and challenges in clinical application of gene therapy Gene therapy for OA has been described extensively for the last twenty years and a large body of impressive preclinical safety and efficacy  ... 
doi:10.5772/18489 fatcat:meltejbxzrg7zofuk2vzief2hu

Impacts of DNA Microarray Technology in Gene Therapy [chapter]

Jaleh Barar, Amir Ata, Yadollah Omidi
2011 Gene Therapy - Developments and Future Perspectives  
Data Mining is all about automating the process of searching for patterns in the data -it is an iterative process of discovery.  ...  in Gene Therapy Fig. 4 .  ...  Impacts of DNA Microarray Technology in Gene Therapy, Gene Therapy -Developments and Future Perspectives, Prof.  ... 
doi:10.5772/18979 fatcat:alzlo5wyjfe4to3ehgmoqveyei

13. The Liver as a Target for Gene Therapy [chapter]

1997 Concepts in Gene Therapy  
There is only very little experience with in vivo selection systems so far but the recent findings will strongly stimulate research in this field of gene therapy and repopulation strategies could become  ...  Application of HSV vectors in vivo via the portal vein has been performed in mice. It leads to reporter gene expression for about four weeks (30) .  ... 
doi:10.1515/9783110808636-014 fatcat:zvvvbu2okvcehdnykw2akcwdge
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