scholarly journals France plans national gene therapy strategy

Nature ◽  
1994 ◽  
Vol 372 (6505) ◽  
pp. 397-397
Author(s):  
Declan Butler
Keyword(s):  
Gene Therapy ◽  
Therapy Strategy ◽  
Gene Therapy Strategy

scholarly journals Gene therapy strategy of glomerulosclerosis.

Ensho ◽  
1998 ◽  
Vol 18 (4) ◽  
pp. 265-269
Author(s):  
Yasufumi Kaneda ◽  
Yoshitaka Isaka ◽  
Enyu Imai
Keyword(s):  
Gene Therapy ◽  
Therapy Strategy ◽  
Gene Therapy Strategy

scholarly journals Peritoneal Dialysis in the 21st Century: The Potential of Gene Therapy

2002 ◽  
Vol 13 (suppl 1) ◽  
pp. S117-S124
Author(s):  
Catherine M. Hoff ◽  
Ty R. Shockley
Keyword(s):  
Gene Therapy ◽  
Peritoneal Dialysis ◽  
Genetic Engineering ◽  
Genetic Manipulation ◽  
Membrane Damage ◽  
Peritoneal Membrane ◽  
Functional Changes ◽  
Therapy Strategy ◽  
Transfer Strategies ◽  
Gene Therapy Strategy

ABSTRACT. One of the greatest biotechnologic advances of the last 25 yr is genetic engineering—the ability to identify and isolate individual genes and transfer genetic elements between cells. Genetic engineering forms the basis of a unique biotechnology platform called gene therapy: an approach to treating disease through genetic manipulation. It is becoming clear that during peritoneal dialysis, the peritoneal membrane undergoes various structural and functional changes that compromise the dialyzing efficiency of the membrane and eventually lead to membrane failure. A gene therapy strategy based on genetic modification of the peritoneal membrane could improve the practice of peritoneal dialysis through the production of proteins that would be of therapeutic value in preventing membrane damage and preserving its dialyzing capacity. The peritoneal membrane can be genetically modified by either ex vivo or in vivo gene transfer strategies with a variety of potentially therapeutic genes, including those for anti-inflammatory cytokines, fibrinolytic factors, and antifibrotic molecules. These genes could be administered either on an acute basis, such as in response to peritonitis, or on an intermittent basis to maintain physiologic homeostasis and perhaps to prevent the adverse changes in the membrane that occur over time. The anticipated effect of a gene therapy strategy could be measured in maintenance of desired transport characteristics and in patients being able to remain on the therapy for longer periods of time without the negative outcomes. In summary, the use of a gene therapy strategy to enhance peritoneal dialysis is an innovative and exciting concept with the potential to provide new treatment platforms for patients with end-stage renal disease.

scholarly journals A Lentiviral Gene Therapy Strategy for the In Vitro Production of Feline Erythropoietin

PLoS ONE ◽  
2012 ◽  
Vol 7 (9) ◽  
pp. e45099 ◽  
Author(s):  
Natalia Vapniarsky ◽  
Michael Lame ◽  
Samantha McDonnel ◽  
Brian Murphy
Keyword(s):  
Gene Therapy ◽  
In Vitro Production ◽  
Therapy Strategy ◽  
Gene Therapy Strategy ◽  
Lentiviral Gene Therapy ◽  
Vitro Production

scholarly journals Deletion of M-opsin prevents “M cone” degeneration in a mouse model of Leber congenital amaurosis

2019 ◽  
Author(s):  
Hui Xu ◽  
Nduka Enemchukwu ◽  
Xiaoyue Zhong ◽  
Olivia Zhang ◽  
Yingbin Fu
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
General Mechanism ◽  
Mouse Line ◽  
Photoreceptor Degeneration ◽  
Multiple Forms ◽  
Leber Congenital Amaurosis ◽  
Reporter Mouse ◽  
Therapy Strategy ◽  
Gene Therapy Strategy

AbstractMutations in RPE65 or lecithin-retinol acyltransferase (LRAT) disrupt 11-cis-retinal synthesis and cause Leber congenital amaurosis (LCA). In Lrat−/− mouse model, mislocalized medium (M)-wavelength sensitive opsin was degraded whereas mislocalized short (S)-wavelength sensitive opsin accumulated before the onset of cone degeneration. The mechanism for the foveal medium (M)/long (L)-wavelength-sensitive cone degeneration in LCA is unknown. By crossing Lrat−/− mice with a proteasome reporter mouse line, we showed that M-opsin enriched dorsal cones in Lrat−/− mice exhibit proteasome stress due to the degradation of large amounts of M-opsin. Deletion of M-opsin relieves the proteasome stress and completely prevents “M cone” degeneration in Lrat−/−Opn1sw−/− mice (a pure “M cone” LCA model, Opn1sw−/− encoding S-opsin) for at least 12 months. Our results suggest that M-opsin degradation associated proteasome stress plays a major role in “M cone” degeneration in Lrat−/− model. This finding may represent a general mechanism for “M cone” degeneration for multiple forms of cone degeneration due to M-opsin mislocalization and degradation. Our results have important implications for the current gene therapy strategy for LCA that emphasizes the need for a combinatorial therapy to both improve vision and slow photoreceptor degeneration.

scholarly journals Lentiviral CRISPR Epigenome Editing of Inflammatory Receptors as a Gene Therapy Strategy for Disc Degeneration

2019 ◽  
Vol 30 (9) ◽  
pp. 1161-1175 ◽  
Author(s):  
Niloofar Farhang ◽  
Matt Ginley-Hidinger ◽  
Kristofer C. Berrett ◽  
Jason Gertz ◽  
Brandon Lawrence ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Disc Degeneration ◽  
Epigenome Editing ◽  
Therapy Strategy ◽  
Gene Therapy Strategy
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