Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes.

Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes.

Item
http://www.wikidata.org/entity/Q37351823
Perinatal gene transfer to the liver.Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome.An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene.Contrasting features of urea cycle disorders in human patients and knockout mouse models.Genetic manipulations of adenovirus type 5 fiber resulting in liver tropism attenuation.In vivo assessment of mutations in OTC for dominant-negative effects following rAAV2/8-mediated gene delivery to the mouse liver.Adenovirus-mediated in vivo gene transfer rapidly protects ornithine transcarbamylase-deficient mice from an ammonium challenge.'Late onset' ornithine transcarbamylase deficiency: function of three purified recombinant mutant enzymes.
P2860

Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes.

Item
http://www.wikidata.org/entity/Q37351823
description
article científic
@ca
article scientifique
@fr
articolo scientifico
@it
artigo científico
@pt
bilimsel makale
@tr
scientific article published on February 1996
@en
vedecký článok
@sk
vetenskaplig artikel
@sv
videnskabelig artikel
@da
vědecký článek
@cs
name
Patient selection may affect g ...... n mouse and human hepatocytes.
@en
Patient selection may affect g ...... n mouse and human hepatocytes.
@nl
type
label
Patient selection may affect g ...... n mouse and human hepatocytes.
@en
Patient selection may affect g ...... n mouse and human hepatocytes.
@nl
prefLabel
Patient selection may affect g ...... n mouse and human hepatocytes.
@en
Patient selection may affect g ...... n mouse and human hepatocytes.
@nl
P1433
P1476
P2093
P2860
P304
P31
P356
P407
P433
P478
P577
P698
P921
P932
P356
P1433
P1476
Patient selection may affect g ...... n mouse and human hepatocytes.
@en
P2093
P2860
P304
P31
P356
P407
P433
P478
P577
P698
P921
P932