Literature DB >> 8636424

Animal models of human disease for gene therapy.

J M Wilson1.   

Abstract

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Year:  1996        PMID： 8636424      PMCID： PMC507165          DOI： 10.1172/JCI118527

Source DB:  PubMed          Journal:  J Clin Invest        ISSN： 0021-9738            Impact factor:   14.808


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  29 in total

1.  Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis.

Authors:  R G Crystal; N G McElvaney; M A Rosenfeld; C S Chu; A Mastrangeli; J G Hay; S L Brody; H A Jaffe; N T Eissa; C Danel
Journal:  Nat Genet       Date:  1994-09       Impact factor: 38.330

2.  Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency.

Authors:  D B Kohn; K I Weinberg; J A Nolta; L N Heiss; C Lenarsky; G M Crooks; M E Hanley; G Annett; J S Brooks; A el-Khoureiy
Journal:  Nat Med       Date:  1995-10       Impact factor: 53.440

3.  Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses.

Authors:  Y Yang; Q Li; H C Ertl; J M Wilson
Journal:  J Virol       Date:  1995-04       Impact factor: 5.103

4.  Generation and characterization of a delta F508 cystic fibrosis mouse model.

Authors:  W H Colledge; B S Abella; K W Southern; R Ratcliff; C Jiang; S H Cheng; L J MacVinish; J R Anderson; A W Cuthbert; M J Evans
Journal:  Nat Genet       Date:  1995-08       Impact factor: 38.330

5.  Lung disease in the cystic fibrosis mouse exposed to bacterial pathogens.

Authors:  D J Davidson; J R Dorin; G McLachlan; V Ranaldi; D Lamb; C Doherty; J Govan; D J Porteous
Journal:  Nat Genet       Date:  1995-04       Impact factor: 38.330

6.  T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

Authors:  R M Blaese; K W Culver; A D Miller; C S Carter; T Fleisher; M Clerici; G Shearer; L Chang; Y Chiang; P Tolstoshev; J J Greenblatt; S A Rosenberg; H Klein; M Berger; C A Mullen; W J Ramsey; L Muul; R A Morgan; W F Anderson
Journal:  Science       Date:  1995-10-20       Impact factor: 47.728

7.  A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia.

Authors:  M Grossman; D J Rader; D W Muller; D M Kolansky; K Kozarsky; B J Clark; E A Stein; P J Lupien; H B Brewer; S E Raper
Journal:  Nat Med       Date:  1995-11       Impact factor: 53.440

8.  MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses.

Authors:  Y Yang; H C Ertl; J M Wilson
Journal:  Immunity       Date:  1994-08       Impact factor: 31.745

9.  Adenosine-deaminase-deficient mice die perinatally and exhibit liver-cell degeneration, atelectasis and small intestinal cell death.

Authors:  A A Migchielsen; M L Breuer; M A van Roon; H te Riele; C Zurcher; F Ossendorp; S Toutain; M S Hershfield; A Berns; D Valerio
Journal:  Nat Genet       Date:  1995-07       Impact factor: 38.330

10.  Correction of lethal intestinal defect in a mouse model of cystic fibrosis by human CFTR.

Authors:  L Zhou; C R Dey; S E Wert; M D DuVall; R A Frizzell; J A Whitsett
Journal:  Science       Date:  1994-12-09       Impact factor: 47.728
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  4 in total

Review 1.  Demystified ... gene knockouts.

Authors:  J P Iredale
Journal:  Mol Pathol       Date:  1999-06

Review 2.  Diseases in a dish: modeling human genetic disorders using induced pluripotent cells.

Authors:  Gustavo Tiscornia; Erica Lorenzo Vivas; Juan Carlos Izpisúa Belmonte
Journal:  Nat Med       Date:  2011-12       Impact factor: 53.440

3.  Specificity and off-target effects of AAV8-TBG viral vectors for the manipulation of hepatocellular gene expression in mice.

Authors:  Christos Kiourtis; Ania Wilczynska; Colin Nixon; William Clark; Stephanie May; Thomas G Bird
Journal:  Biol Open       Date:  2021-09-22       Impact factor: 2.422

Review 4.  Animal models for target diseases in gene therapy--using DNA and siRNA delivery strategies.

Authors:  Ian S Blagbrough; Chiara Zara
Journal:  Pharm Res       Date:  2008-10-08       Impact factor: 4.200
  4 in total

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