scholarly journals Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

2010 ◽  
Vol 18 (1) ◽  
pp. 11-16 ◽  
Author(s):  
Randy J Chandler ◽  
Charles P Venditti
Keyword(s):  
Gene Therapy ◽  
Murine Model ◽  
Methylmalonic Acidemia ◽  
Viral Gene ◽  
Viral Gene Therapy

Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy

2021 ◽  
Author(s):  
Hyung‐Ok Lee ◽  
Christiana O. Salami ◽  
Dolan Sondhi ◽  
Stephen M. Kaminsky ◽  
Ronald G. Crystal ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Viral Gene ◽  
Functional Correction ◽  
Viral Gene Therapy

scholarly journals Long-term control of neuropathic pain in a non-viral gene therapy paradigm

Gene Therapy ◽  
2009 ◽  
Vol 16 (4) ◽  
pp. 470-475 ◽  
Author(s):  
E M Sloane ◽  
R G Soderquist ◽  
S F Maier ◽  
M J Mahoney ◽  
L R Watkins ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Neuropathic Pain ◽  
Viral Gene ◽  
Viral Gene Therapy

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 Recognition of Virus Infection and Innate Host Responses to Viral Gene Therapy Vectors

2010 ◽  
Vol 18 (8) ◽  
pp. 1422-1429 ◽  
Author(s):  
Dmitry M Shayakhmetov ◽  
Nelson C Di Paolo ◽  
Karen L Mossman
Keyword(s):  
Gene Therapy ◽  
Virus Infection ◽  
Host Responses ◽  
Viral Gene ◽  
Viral Gene Therapy ◽  
Gene Therapy Vectors

scholarly journals Delivery of modified mRNA encoding vesicular stomatitis virus matrix protein for colon cancer gene therapy

RSC Advances ◽  
2018 ◽  
Vol 8 (22) ◽  
pp. 12104-12115 ◽  
Author(s):  
Ke Men ◽  
Rui Zhang ◽  
Xueyan Zhang ◽  
Rong Huang ◽  
Guonian Zhu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Colon Cancer ◽  
Colon Carcinoma ◽  
Vesicular Stomatitis Virus ◽  
Matrix Protein ◽  
Cancer Gene Therapy ◽  
Viral Gene ◽  
Cancer Gene ◽  
Viral Gene Therapy ◽  
Vesicular Stomatitis

Liposome–protamine complex delivered VSVMP mRNA efficiently inhibits C26 colon carcinoma with safety, providing an alternative strategy for non-viral gene therapy.

Viral gene therapy for haemophilia

2001 ◽  
Vol 6 (4) ◽  
pp. 165-166 ◽  
Author(s):  
Janet Fricker
Keyword(s):  
Gene Therapy ◽  
Viral Gene ◽  
Viral Gene Therapy
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