(Q35892058)

11 August 2017

Successful gene therapy in utero for lethal murine hypophosphatasia (English)

1 reference

11 August 2017

0 references

0 references

Hanako Sugano

1

1 reference

11 August 2017

Tae Matsumoto

2

1 reference

11 August 2017

Koichi Miyake

3

1 reference

11 August 2017

Atsushi Watanabe

4

1 reference

11 August 2017

Osamu Iijima

5

1 reference

11 August 2017

Makoto Migita

6

1 reference

11 August 2017

Sonoko Narisawa

7

1 reference

11 August 2017

José Luis Millán

8

1 reference

11 August 2017

Yoshitaka Fukunaga

9

1 reference

11 August 2017

Takashi Shimada

10

1 reference

11 August 2017

26 January 2012

1 reference

11 August 2017

1 reference

11 August 2017

23

1 reference

11 August 2017

4

1 reference

11 August 2017

399-406

1 reference

Molecular control of endothelial cell behaviour during blood vessel morphogenesis

11 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Early gestational gene transfer of IL-10 by systemic administration of lentiviral vector can prevent arthritis in a murine model

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Rescue of severe infantile hypophosphatasia mice by AAV-mediated sustained expression of soluble alkaline phosphatase

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Enzyme replacement therapy prevents dental defects in a model of hypophosphatasia

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Prolonged survival and phenotypic correction of Akp2(-/-) hypophosphatasia mice by lentiviral gene therapy

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Physiological role of alkaline phosphatase explored in hypophosphatasia

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Enzyme replacement therapy for murine hypophosphatasia

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Fetal gene transfer using lentiviral vectors and the potential for germ cell transduction in rhesus monkeys (Macaca mulatta).

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Evaluation of risks related to the use of adeno-associated virus-based vectors.

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Alkaline phosphatase knock-out mice recapitulate the metabolic and skeletal defects of infantile hypophosphatasia

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Temporally regulated expression patterns following in utero adenovirus-mediated gene transfer

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Factors influencing recombinant adeno-associated virus production

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Pyridoxine effect on synthesis rate of serotonin in the monkey brain measured with positron emission tomography

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Clinical characteristics of perinatal lethal hypophosphatasia: a report of 6 cases

27 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Dose response of bone-targeted enzyme replacement for murine hypophosphatasia

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Global gene transfer into the CNS across the BBB after neonatal systemic delivery of single-stranded AAV vectors

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Layer-specific activity of tissue non-specific alkaline phosphatase in the human neocortex.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Global diffuse distribution in the brain and efficient gene delivery to the dorsal root ganglia by intrathecal injection of adeno-associated viral vector serotype 1.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

AAV vector integration sites in mouse hepatocellular carcinoma.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Male germ-line cells are at risk following direct-injection retroviral-mediated gene transfer in utero

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

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

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Reexpression following readministration of an adenoviral vector in adult mice after initial in utero adenoviral administration

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Purification of recombinant adeno-associated virus by iodixanol gradient ultracentrifugation allows rapid and reproducible preparation of vector stocks for gene transfer in the nervous system

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Reversal of cystic fibrosis phenotype in mice by gene therapy in utero

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Inactivation of two mouse alkaline phosphatase genes and establishment of a model of infantile hypophosphatasia.

24 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3327603

Prevalence of c.1559delT in ALPL, a common mutation resulting in the perinatal (lethal) form of hypophosphatasia in Japanese and effects of the mutation on heterozygous carriers

24 September 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22133046

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Detection of minor immunological differences among human "universal-type" alkaline phosphatases

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22133046

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Bone formation following transplantation of genetically modified primary bone marrow stromal cells

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/22133046

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.1089/HUM.2011.148

1 reference

11 August 2017

1 reference

11 August 2017

1 reference