(Q35904452)

11 August 2017

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title

Fetal and neonatal gene therapy: benefits and pitfalls (English)

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11 August 2017

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Waddington SN

1

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11 August 2017

Kennea NL

2

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11 August 2017

Buckley SM

3

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11 August 2017

Gregory LG

4

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11 August 2017

Themis M

5

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11 August 2017

Coutelle C

6

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11 August 2017

1 October 2004

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11 August 2017

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11 August 2017

11 Suppl 1

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11 August 2017

S92-7

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https://scigraph.springernature.com/pub.10.1038/sj.gt.3302375

11 August 2017

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cites work

Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-defici

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Long-term correction of bilirubin UDPglucuronyltransferase deficiency in rats by in utero lentiviral gene transfer.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

In utero gene therapy rescues vision in a murine model of congenital blindness

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Genotype spectrum of ornithine transcarbamylase deficiency: correlation with the clinical and biochemical phenotype

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

State-of-the-art 2003 on PKU gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Perinatal management of newborns with haemophilia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Consensus perspectives on prophylactic therapy for haemophilia: summary statement

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Mucopolysaccharidosis type VII in the developing mouse fetus

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Muscle lesions associated with dystrophin deficiency in neonatal golden retriever puppies

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Non-physiological overexpression of the low density lipoprotein receptor (LDLr) gene in the liver induces pathological intracellular lipid and cholesterol storage

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Efficient lentiviral transduction of liver requires cell cycling in vivo

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Transcription start regions in the human genome are favored targets for MLV integration

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Murine Neonatal Lymphocytes Show Rapid Early Cell Cycle Entry and Cell Division

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

TNF-α-Dependent ICAM-1- and VCAM-1-Mediated Inflammatory Responses Are Delayed in Neonatal Mice Infected with Pneumocystis carinii

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Functional responses of human neonatal B lymphocytes to antigen receptor cross-linking and CpG DNA.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Blood plasmacytoid dendritic cell responses to CpG oligodeoxynucleotides are impaired in human newborns

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Phenotypic and functional analysis of human T lymphocytes in early second- and third-trimester fetuses

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Intrauterine T-cell activation and increased proinflammatory cytokine concentrations in preterm infants with cerebral lesions.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Hepatitis B immunisation induces higher antibody and memory Th2 responses in new-borns than in adults

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Infant and adult human B cell responses to rotavirus share common immunodominant variable gene repertoires

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Immature anti-inflammatory response in neonates

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Fetal membranes act as a barrier for adenoviruses: gene transfer into exocoelomic cavity of rat fetuses does not affect cells in the fetus

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Fetal wound healing: current biology

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Barrier function of the skin: "la raison d'être" of the epidermis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

The basal lamina is a physical barrier to herpes simplex virus-mediated gene delivery to mature muscle fibers.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Development of blood-brain barrier tight junctions in the rat cortex.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Developmental mucin gene expression in the human respiratory tract

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Lysozyme and RNases as anti-HIV components in beta-core preparations of human chorionic gonadotropin

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Contribution of human alpha-defensin 1, 2, and 3 to the anti-HIV-1 activity of CD8 antiviral factor.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Human alpha-defensin 1 (HNP-1) inhibits adenoviral infection in vitro

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Retrocyclin: a primate peptide that protects cells from infection by T- and M-tropic strains of HIV-1.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

The θ-Defensin, Retrocyclin, Inhibits HIV-1 Entry

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Neonatal skin in mice and humans expresses increased levels of antimicrobial peptides: innate immunity during development of the adaptive response

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

In vitro and in vivo antimicrobial activity of two alpha-helical cathelicidin peptides and of their synthetic analogs.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Human enteric defensins. Gene structure and developmental expression

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Differential short-term transduction efficiency of adult versus newborn mouse tissues by adenoviral recombinants.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Expression of the primary coxsackie and adenovirus receptor is downregulated during skeletal muscle maturation and limits the efficacy of adenovirus-mediated gene delivery to muscle cells

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Materno-foetal transfer of H. influenzae and pneumococcal antibodies is influenced by prematurity and low birth weight: implications for conjugate vaccine trials

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Postpartum maternal corticosterone decreases maternal and neonatal antibody levels and increases the susceptibility of newborn mice to herpes simplex virus-associated mortality

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Inhibition of herpes simplex virus infection by lactoferrin is dependent on interference with the virus binding to glycosaminoglycans

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Characterization of the anti-HIV effects of native lactoferrin and other milk proteins and protein-derived peptides

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Heparin-interacting sites of bovine lactoferrin are involved in anti-adenovirus activity

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Probiotics for preterm infants?

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Prophylactic Lactobacillus GG reduces antibiotic-associated diarrhea in children with respiratory infections: a randomized study

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302375

7 January 2021

Identifiers

DOI

10.1038/SJ.GT.3302375

1 reference

11 August 2017

1 reference