scholarly journals Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy

2012 ◽  
pp. 303 ◽  
Author(s):  
Aritz Perez Ruiz de Garibay ◽  
Delgado ◽  
Ana del Pozo ◽  
Mª Angeles Solinís ◽  
Alicia Rodriguez Gascón
Keyword(s):  
Gene Therapy ◽  
Fabry Disease ◽  
Nonviral Vectors

scholarly journals α-Galactosidase A Augmentation by Non-Viral Gene Therapy: Evaluation in Fabry Disease Mice

Pharmaceutics ◽  
2021 ◽  
Vol 13 (6) ◽  
pp. 771
Author(s):  
Julen Rodríguez-Castejón ◽  
Ana Alarcia-Lacalle ◽  
Itziar Gómez-Aguado ◽  
Mónica Vicente-Pascual ◽  
María Ángeles Solinís Aspiazu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Fabry Disease ◽  
Viral Vector ◽  
Viral Gene ◽  
Lysosomal Storage ◽  
Wild Type ◽  
Dose Administration ◽  
Viral Gene Therapy ◽  
Liver Transaminases ◽  
Low Levels

Fabry disease (FD) is a monogenic X-linked lysosomal storage disorder caused by a deficiency in the lysosomal enzyme α-Galactosidase A (α-Gal A). It is a good candidate to be treated with gene therapy, in which moderately low levels of enzyme activity should be sufficient for clinical efficacy. In the present work we have evaluated the efficacy of a non-viral vector based on solid lipid nanoparticles (SLN) to increase α-Gal A activity in an FD mouse model after intravenous administration. The SLN-based vector incremented α-Gal A activity to about 10%, 15%, 20% and 14% of the levels of the wild-type in liver, spleen, heart and kidney, respectively. In addition, the SLN-based vector significantly increased α-Gal A activity with respect to the naked pDNA used as a control in plasma, heart and kidney. The administration of a dose per week for three weeks was more effective than a single-dose administration. Administration of the SLN-based vector did not increase liver transaminases, indicative of a lack of toxicity. Additional studies are necessary to optimize the efficacy of the system; however, these results reinforce the potential of lipid-based nanocarriers to treat FD by gene therapy.

scholarly journals Gene Therapy Delivery Systems for Enhancing Viral and Nonviral Vectors for Cardiac Diseases: Current Concepts and Future Applications

2013 ◽  
Vol 24 (11) ◽  
pp. 914-927 ◽  
Author(s):  
Michael G. Katz ◽  
Anthony S. Fargnoli ◽  
Richard D. Williams ◽  
Charles R. Bridges
Keyword(s):  
Gene Therapy ◽  
Delivery Systems ◽  
Cardiac Diseases ◽  
Nonviral Vectors ◽  
Therapy Delivery

A phase I/II multicenter gene therapy clinical study for Fabry disease

2020 ◽  
Vol 129 (2) ◽  
pp. S77
Author(s):  
Derralynn Hughes ◽  
Ozlem Goker-Alpan ◽  
Jaya Ganesh ◽  
Heather Lau ◽  
Cheryl Wong Po Foo ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Clinical Study ◽  
Phase I ◽  
Fabry Disease

Correction of the Nonlinear Dose Response Improves the Viability of Adenoviral Vectors for Gene Therapy of Fabry Disease

2002 ◽  
Vol 13 (8) ◽  
pp. 935-945 ◽  
Author(s):  
Robin J. Ziegler ◽  
Chester Li ◽  
Maribeth Cherry ◽  
Yunxiang Zhu ◽  
Donna Hempel ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Fabry Disease ◽  
Dose Response ◽  
Adenoviral Vectors

Current Status of Nonviral Vectors for Gene Therapy in China

2018 ◽  
Vol 29 (2) ◽  
pp. 110-120 ◽  
Author(s):  
Li Liu ◽  
Jingyun Yang ◽  
Ke Men ◽  
Zhiyao He ◽  
Min Luo ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Current Status ◽  
Nonviral Vectors

Functional evaluation of an AAV9 based vector expressing alpha-galactosidase A for potential gene therapy of Fabry disease

2019 ◽  
Vol 126 (2) ◽  
pp. S113-S114 ◽  
Author(s):  
Saida Ortolano ◽  
Maria G. Biferi ◽  
Beatriz San Millán ◽  
Olga Souto ◽  
Irene Vieitez ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Fabry Disease ◽  
Functional Evaluation ◽  
Alpha Galactosidase
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