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594 delivery of recombinant adeno associated virus mediated human tissue kallikrein gene attenuates insulin resistance and prevents renal complications in diabetic rats

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594 Delivery of Recombinant Adeno Associated Virus Mediated Human Tissue Kallikrein Gene Attenuates Insulin Resistance and Prevents Renal Complications in Diabetic Rats Molecular Therapy �������� ���[.]

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Molecular Therapy Vol 7, No 5, May 2003, Part 2 of 2 Parts

CARDIOVASCULAR

Neovasculature Formation in Ischemic

Myocardium

Karen L Christman,1 Qizhi Fang,2 Richard E Sievers,3 Anne J

Kim,1 Hubert H Fok,3 Albert F Candia,4 Kenneth J Colley,4

Randall J Lee.2,3

1 Joint Bioengineering Graduate Group, University of California

Berkeley and San Francisco, San Francisco, CA, United States;

2 Cardiovascular Research Institute, University of California San

Francisco, San Francisco, CA, United States; 3 Medicine,

University of California San Francisco, San Francisco, CA,

United States; 4 Angiogenix, Inc., Burlingame, CA, United States.

Pleiotrophin (PTN) is a secreted heparin-binding cytokine with

diverse functions In vitro, PTN has been shown to induce

angiogenesis and stimulate endothelial cell proliferation We have

recently demonstrated that the PTN gene is upregulated during

ischemia in the heart, thus indicating a role in angiogenesis in the

myocardium In this study, we hypothesized that delivery of PTN

to ischemic myocardium will induce neovasculature formation We

first developed a pCMV plasmid encoding the PTN gene and

demonstrated via an ELISA that mammalian cells transfected with

this plasmid are capable of producing and secreting PTN The

produced PTN also induced proliferation of serum-starved SW13

cells, indicating that it is biologically active 250 μg of the

pCMV-PTN plasmid was injected directly into the infarcted myocardium

of female Sprague-Dawley rats (n=3) following occlusion of their

left anterior descending portion of the left coronary artery for 17

minutes Another set of rats (n=2) was injected with 250 μg of a

pCMV-β-gal plasmid to serve as a control The rats were sacrificed

after a minimum of five weeks The hearts were rapidly excised,

fresh frozen, and sectioned into 10 μm slices 5 sections from each

heart were stained for capillaries using a Griffonia simplicifolia lectin

which binds to a carbohydrate domain on endothelial cells The

average number of capillaries within the infarct scar was quantified

for each group using Fovea 2.0 plug-in for Adobe Photoshop

Another five sections were stained for arterioles using an anti-smooth

muscle actin antibody The average number and diameter of arterioles

within each infarct were also quantified for each group using SPOT

3.5.5 software Injection of PTN plasmid induced formation of

neovasculature compared to the β-gal plasmid control The capillary

density following delivery of PTN plasmid significantly increased

to 1258±157 capillaries/mm² compared to 782±166 capillaries/mm²

withβ-gal plasmid (P<0.05) The arteriole density also increased to

11±2 arterioles/mm² compared to 4±0 arterioles/mm² for the control

(P<0.03) (see Figure; A: β-gal plasmid, B: PTN plasmid) The

average arteriole diameter was not significantly different In addition,

there was no macroscopic or histological evidence of tumor formation

with the PTN plasmid PTN plasmid may therefore be a possible

treatment for ischemic heart disease

Figure 1 Anti-smooth muscle actin labeled arterioles within the

infarct scar (40×)

Adeno-Associated Virus-Mediated Human Tissue Kallikrein Gene Attenuates Insulin Resistance and Prevents Renal Complications in Diabetic Rats

Gang Yuan,1 Chunxia Zhao,1 Tao Wang,1 Xiao Xiao,1 Juan Li,1 Lee Chao,2 Julie Chao,2 Dao Wen Wang.1

1 Dept of Internal Medicine, Tongji Hospital/Medical College of Huazhong University of Science and Technology, Wuhan, China;

2 Dept of Biochemistry, Medical University of South Carolina, Charleston, SC.

Background: Insulin resistance plays a major role in type 2

diabetes Previous studies have shown that bradykinin potentiates insulin-stimulated glucose uptake and enhances insulin signal through B2 receptor In this study, we evaluated the potential of adeno-associated viral vector (AAV) expressing HK gene as a sole therapy

to correct insulin resistance and to prevent renal complications in

type 2 diabetic rats Methods: Male wistar rats received intravenous

injection of low dose streptozotocin (25mg/kg) and be fed with diets enriched in both glucose and fat to form type 2 diabetic model Single dose of rAAV(1x1010 pfu)expressing HK or LacZ was injected into diabetic rats through tail vein after 4 weeks The caudal arterial pressure was measured using tail cuff device once a week for 16 weeks and 24-hour urine and fasting blood were collected to determine levels of blood glucose, insulin and HK concentrations every 4 weeks until the animals were sacrificed The main organs

were obtained for histological analysis Results: (1) The 4-week

feeding with diets enriched in both glucose and fat induced an increase

in systolic pressure up to 20-25mmHg (95±5.1 up to 124.1±3.9mmHg) and hyperglycemia and hyperinsulinemia All the animals were randomly divided into two groups, receiving rAAV•HK and rAAV•LacZ injection, respectively (2) Delivery of rAAV•HK resulted in a reduction in blood pressure about 20mmHg in 2 weeks and the hypotensive effect lasted for the duration of the study, whereas the blood pressure in control group did not change (Fig 1) (3) The levels of the fast blood insulin were very significantly lower

in rAAV.HK group than control group (HK vs control: 7.72±3.31 mIu/L vs 13.06±4.22 mIu/L,P<0.01) though the levels of fasting blood glucose were not statistically different in both groups (HK vs control: 13.58±3.05 mmol/L vs 13.09±2.14mmol/L) at the end of the study, which suggests that HK enhance insulin sensitivity (4) The expression of HK was detected in blood and urine by ELISA in the HK group but undetectable in control group (5) Histological assessment of kidneys showed near normal morphology in rat kidneys in HK group, but glomerular hypertrophy, mild diffuse mesangial expansion, extracellular matrix accumulation and hyalinosis

in control group These indicated that rAAV•HK gene delivery prevented or reversed the renal injuries caused by diabetes

Conclusions: Our studies demonstrated that the rAAV-mediated

HK gene delivery can very efficiently attenuate insulin resistance and renal injuries as well as lower elevated blood pressure in type 2 diabetes These results suggest that rAAV-mediated HK gene administration may represent a useful tool for the therapy of insulin resistance and the prevention of chronic complications in patients with diabetes

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Molecular Therapy Vol 7, No 5, May 2003, Part 2 of 2 Parts

Copyright © The American Society of Gene Therapy

S232

CARDIOVASCULAR

The patent is pending

Improves the Angiogenic Potential of Myoblasts

Louis-Georges Guy,1 Anouk S Fortin,1 Guy Leclerc.1

1 Angiogene Inc., Montreal, QC, Canada.

Myocardial infarction caused by hypoxia results in cardiac tissue

damage Cardiac function can be improved by cell transplantation in

the scarred myocardium Furthermore, the implanted cells can also

be used as vectors for the production of angiogenic factors to improve

perfusion This study examined the angiogenic activity of the Hypoxia

Inducible Factor EPAS1 modified human myoblasts Methods:

Adenoviruses encoding EPAS1 were used to transduce cell lines or

primary myoblasts VEGF induction was used as a marker of

angiogenic activity and was quantified by ELISA and RNase

protection assays Angiogenesis was assayed in vivo by

subcutaneous implantation in mice of Matrigel pellets containing

unmodified or transduced myoblasts Microarrays were used to

document gene induction Results: In the cell types studied, wild

type EPAS1 was found to be a potent VEGF inducer, both in

normoxia and hypoxia In vivo, myoblasts transduced with EPAS1

showed a significantly increased angiogenesis activity in

sub-cutaneous implants Several genes implicated in the different steps

of the angiogenesis process were found to be induced in myoblasts

by EPAS1 Conclusions: EPAS1 is a potent inducer of angiogenic

genes in myoblasts, resulting in a strong in vivo angiogenesis It is

thus a good candidate for genetic manipulation of myoblasts to be

transplanted in the scarred myocardium

Employees of Angiogene Inc

Reendothelialization and Inhibition of In-Stent

Intimal Hyperplasia in Rabbit Model

Yaxue Shi,1 Zhengfeng Wang,1 Hao Zhang,1 Baigen Zhang.1

1 Vascular Surgery, RenJi Hospital of Shanghai Second Medical

University, Shanghai, China.

Objectives: Intact endothelial layer is important to maintain the

smooth muscle cell (SMC) phenotype and prevent the thrombosis

which is related to SMC proliferation.We transfer the

pSV-VEGF165 which stimulates the proliferation of endothelial cells to

the carotid artery by stents and assess its effects on the

reendothelialization and neointima.Methods:28 Cholesterol-fed New

Zealand White rabbits were balloon-denuded and implanted

pSV-VEGF165(800μg) coated stents in the carotid artery.Plasmid

pSV-β-gal was used as control RT-PCR, immunocytochemistry ,electron

microscope, histology were detected Results: After 1d, RT-PCR

showed the expression of VEGF165 mRNA in treatment group(0.3114±0.0493) Its expression increased after 1w (1.0467±0.0670) and persisted till 1m (fig1) Immunocytochemistry also confirmed the expression of VEGF protein, which was found after 3d(20.4950±2.8711%) in media(fig2) and reached the peak after 1w(43.5433±7.8592%) ,attenuated after 3m(9.5200±1.4127%).Scanning electron microscope showed that the time of reendothelialization in treatment group was 1 month (fig3)which was shorter than control group But transmission electron microscope showed that in treatment group there still were synthetic SMCs in media and intima after 1 month ,which was similar to the control group.The intima/media ratio was no difference

between two group(P>0.05)(tab1)(fig4) Conclusions:Plasmid

coated stent can locally deliver the pSV-VEGF165 gene to the stenting site and the vascular cells could uptake the gene,excrete the protein which can promote the endothelialization,but it can not prevent the in-stent intimal hyperplasia

Intimal/media ratio of treatment group and control group control group treatment group

1m 0.50±0.02 0.50±0.04 2m 0.90±0.11 0.87±0.08 3m 1.54±0.34 1.58±0.35

Growth Factor and Prostacyclin Synthase Gene Enhanced Therapeutic Angiogenesis in Rat and Rabbit Ischemia Model

Hiromi Koike,1 Naruya Tomita,2 Tadashi Tanabe,3 Yasufumi Kaneda,1 Ryuichi Morishita.1

1 Gene Therapy Science, Osaka University Graduate School of Medicine, Suita, Osaka, Japan; 2 General Medicine, Osaka University Hospital, Suita, Osaka, Japan; 3 Pharmacology, National Cardiovascular Center Research Institute, Suita, Osaka, Japan.

Background: Current therapeutic angiogenesis strategy to treat ischemic disease using angiogenic growth factors has been limited to use a single gene However, as vasodilative substances such as prostacyclin are widely used for the treatment of peripheral arterial disease, we are sure it might be useful to combine the angiogenesis with vasodilation of new vessels Thus, in this study, we examined the angiogenic activity of co-transfection of genes of an angiogenic growth factor, hepatocyte growth factor (HGF), and a vasodilative substance, prostacyclin, synthase

Methods and Results: Ten days after induction of hindlimb ischemia in mice, we intramuscularly injected naked plasmid of human HGF gene and/or prostacyclin synthase gene At 4 weeks after injections of plasmids, co-transfection of HGF gene with prostacyclin synthase gene demonstrated further increase in blood flow as compared to a single gene transfection, accompanied by a significant increase in capillary density, while blood flow was

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