Genetic medicines: treatment strategies for hereditary disorders (Q36423907)

14 August 2017

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title

Genetic medicines: treatment strategies for hereditary disorders (English)

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14 August 2017

Timothy P O'Connor

1

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PubMed ID

16543931

14 August 2017

Ronald G Crystal

series ordinal

2

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14 August 2017

1 April 2006

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14 August 2017

Nature Reviews Genetics

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14 August 2017

7

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14 August 2017

4

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14 August 2017

261-276

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https://scigraph.springernature.com/pub.10.1038/nrg1829

14 August 2017

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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31 March 2020

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Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

31 March 2020

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Safety and feasibility of liver-directed ex vivo gene therapy for homozygous familial hypercholesterolemia.

31 March 2020

1 reference

Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC)

31 March 2020

1 reference

Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease

31 March 2020

1 reference

Gene therapy of X-linked severe combined immunodeficiency

31 March 2020

1 reference

Leukocyte adhesion deficiency in children and Irish setter dogs

31 March 2020

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Jak3 and the pathogenesis of severe combined immunodeficiency

31 March 2020

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Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion

31 March 2020

1 reference

Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector

31 March 2020

1 reference

A pilot study of in vivo liver-directed gene transfer with an adenoviral vector in partial ornithine transcarbamylase deficiency

31 March 2020

1 reference

Correlation between DNA transfer and cystic fibrosis airway epithelial cell correction after recombinant adeno-associated virus serotype 2 gene therapy

31 March 2020

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31 March 2020

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31 March 2020

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Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

31 March 2020

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31 March 2020

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An adenovirus vector with genetically modified fibers demonstrates expanded tropism via utilization of a coxsackievirus and adenovirus receptor-independent cell entry mechanism

31 March 2020

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7 January 2021

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Identifiers

DOI

10.1038/NRG1829

1 reference

PubMed ID

16543931

Dimensions Publication ID

14 August 2017

0 references

1 reference

PubMed ID

16543931