scholarly journals Long‐term effects of systemic gene therapy in a canine model of myotubular myopathy

2017 ◽  
Vol 56 (5) ◽  
pp. 943-953 ◽  
Author(s):  
Matthew Elverman ◽  
Melissa A. Goddard ◽  
David Mack ◽  
Jessica M. Snyder ◽  
Michael W. Lawlor ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Canine Model ◽  
Long Term Effects ◽  
Myotubular Myopathy

scholarly journals Long-term effects of hepatocyte growth factor gene therapy in rat myocardial infarct model

Gene Therapy ◽  
2011 ◽  
Vol 19 (8) ◽  
pp. 836-843 ◽  
Author(s):  
Y-N Jin ◽  
M Inubushi ◽  
K Masamoto ◽  
K Odaka ◽  
I Aoki ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Growth Factor ◽  
Hepatocyte Growth Factor ◽  
Myocardial Infarct ◽  
Long Term Effects ◽  
Factor Gene ◽  
Growth Factor Gene ◽  
Myocardial Infarct Model ◽  
Hepatocyte Growth

scholarly journals Long-Term Effects of Gene Therapy in a Novel Mouse Model of Human MFRP-Associated Retinopathy

2019 ◽  
Vol 30 (5) ◽  
pp. 632-650 ◽  
Author(s):  
Anil Chekuri ◽  
Bhubanananda Sahu ◽  
Venkata Ramana Murthy Chavali ◽  
Marina Voronchikhina ◽  
Angel Soto-Hermida ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mouse Model ◽  
Long Term Effects

scholarly journals Adenovirus-Mediated Gene Therapy for Mucopolysaccharidosis VII: Involvement of Cross-Correction in Wide-Spread Distribution of the Gene Products and Long-Term Effects of CTLA-4Ig Coexpression

2000 ◽  
Vol 1 (5) ◽  
pp. 406-413 ◽  
Author(s):  
Motomichi Kosuga ◽  
Satori Takahashi ◽  
Kyoko Sasaki ◽  
Xiao-Kang Li ◽  
Masayuki Fujino ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Products ◽  
Wide Spread ◽  
Long Term Effects

Abstract 5423: rAAV-Mediated Gene Therapy with Inducible Nitric Oxide Synthase (iNOS) Affords Permanent Cardioprotection (1 Year) without Adverse Functional Consequences

Circulation ◽  
2008 ◽  
Vol 118 (suppl_18) ◽  
Author(s):  
Qianhong Li ◽  
Yiru Guo ◽  
Wen-Jian Wu ◽  
Qinghui Ou ◽  
Santosh K Sanganalmath ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Gene Transfer ◽  
Viral Vector ◽  
Long Term Effects ◽  
Inos Gene ◽  
Inos Protein Expression ◽  
Functional Consequences ◽  
And Function ◽  
Oxide Synthase

The ultimate goal of prophylactic gene therapy is to confer permanent protection against ischemia. Although gene therapy with iNOS is known to protect against myocardial infarction at 3 days and up to 2 months, the long-term effects of iNOS gene therapy on myocardial ischemic injury and function are unknown. To address this issue, we created a recombinant adeno-associated viral vector carrying the iNOS gene (rAAV/iNOS) which enables long-lasting transgene expression. Mice received injections in anterior LV wall of rAAV/LacZ or rAAV/iNOS; 1 year later, they underwent a 30-min coronary occlusion (O) and 4 h of reperfusion (R). iNOS gene transfer resulted in elevated iNOS protein expression (+ 2.9-fold vs. LacZ group, n=6, P<0.05; Fig ) and iNOS activity (+ 3.3-fold vs. LacZ group, n=4, P<0.05) 1 year later. Infarct size (% of risk region) was dramatically reduced at 1 year after iNOS gene transfer (13.5+/−2.2%, n=12, vs. 42.9+/−2.6%, n=12, in LacZ group; Fig ). The infarct-sparing effects of iNOS gene therapy at 1 year were as powerful as those observed 24 h after ischemic PC (six 4-min O/4-min R cycles) (16.3+/−2.3%, n=8; Fig ). Importantly, compared with the LacZ group (n=11), iNOS gene transfer (n=10) had no effect on LV dimensions or function for up to 1 year (at 1 year: LVEDD 4.4+/−0.1 vs. 4.2+/−0.2 mm; LVESD 2.9+/−0.1 vs. 2.9+/−0.2 mm; FS 34+/−1.8 vs. 32+/−2.6%; EF 56+/−2.3 vs. 60+/−2.9%) (echocardiography). These data demonstrate, for the first time, that rAAV-mediated iNOS gene transfer affords long-term, probably permanent (1 year) cardioprotection without adverse functional consequences, providing a strong rationale for further preclinical testing of prophylactic gene therapy.

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