Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats

Result code in IS VaVaI

<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00216208%3A11110%2F13%3A10192305" target="_blank" >RIV/00216208:11110/13:10192305 - isvavai.cz</a>

Result on the web

<a href="http://dx.doi.org/10.1089/hgtb.2013.108" target="_blank" >http://dx.doi.org/10.1089/hgtb.2013.108</a>

DOI - Digital Object Identifier

<a href="http://dx.doi.org/10.1089/hgtb.2013.108" target="_blank" >10.1089/hgtb.2013.108</a>

Result language

angličtina

Original language name

Improved efficacy and reduced toxicity by ultrasound-guided intrahepatic injections of helper-dependent adenoviral vector in Gunn rats

Original language description

Crigler-Najjar syndrome type I is caused by mutations of the uridine diphospho-glucuronosy] transferase lAI (UGT1A1) gene resulting in life-threatening increase of serum bilirubin. Life-long correction of hyperbilirubinemia was previously shown with intravenous injection of high doses of a helper-dependent adenoviral (HDAd) vector cxpressing UGT1A1 in the Gunn rat, the animal model of Crigler-Najjar syndrome. I lowever, such high vector doses can activate an acute and potentially lethal inflammatory response with elevated serum interleukin-6 (IL-6). To overcome this obstacle, we investigated safety and efficacy of direct injections of low HDAd doses delivered directly into the liver parenchyma of Gunn rats. Direct hepatic injections performed by eitherlaparotomy or ultrasound-guided percutaneous injections were compared with the same doses given by intravenous injections. A greater reduction of hyperbilirubinemia and inereased conjugated bilirubin in hile were achieved with 1 x 1011 v

Czech name

—

Czech description

—

Type

J_x - Unclassified - Peer-reviewed scientific article (Jimp, Jsc and Jost)

CEP classification

CE - Biochemistry

OECD FORD branch

—

Project

—

Continuities

I - Institucionalni podpora na dlouhodoby koncepcni rozvoj vyzkumne organizace

Publication year

2013

Confidentiality

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Name of the periodical

Human gene therapy methods

ISSN

1946-6536

e-ISSN

—

Volume of the periodical

24

Issue of the periodical within the volume

5

Country of publishing house

US - UNITED STATES

Number of pages

7

Pages from-to

321-327

UT code for WoS article

—

EID of the result in the Scopus database

—