(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

0 references

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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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Stem cell biology and neurodegenerative disease

31 March 2020

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Systematic determination of the packaging limit of lentiviral vectors

31 March 2020

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31 March 2020

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Gene therapy: theoretical and bioethical concepts

31 March 2020

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Regulatory issues for clinical gene therapy trials

31 March 2020

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31 March 2020

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31 March 2020

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Correction of a genetic defect by nuclear transplantation and combined cell and gene therapy

31 March 2020

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Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

31 March 2020

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Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

31 March 2020

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31 March 2020

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Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis

31 March 2020

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Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo

31 March 2020

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Safety and biological efficacy of a lipid-CFTR complex for gene transfer in the nasal epithelium of adult patients with cystic fibrosis

31 March 2020

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Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: a phase I trial of safety and efficacy in the nasal airway

31 March 2020

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Transfection of nasal mucosa with a normal alpha1-antitrypsin gene in alpha1-antitrypsin-deficient subjects: comparison with protein therapy

31 March 2020

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Aspartoacylase gene transfer to the mammalian central nervous system with therapeutic implications for Canavan disease

31 March 2020

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Phase I study of dystrophin plasmid-based gene therapy in Duchenne/Becker muscular dystrophy.

31 March 2020

1 reference

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

31 March 2020

1 reference

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

1 reference

Jak3 and the pathogenesis of severe combined immunodeficiency

31 March 2020

1 reference

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

1 reference

Repeated adeno-associated virus serotype 2 aerosol-mediated cystic fibrosis transmembrane regulator gene transfer to the lungs of patients with cystic fibrosis: a multicenter, double-blind, placebo-controlled trial

31 March 2020

1 reference

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

31 March 2020

1 reference

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

31 March 2020

1 reference

Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

31 March 2020

1 reference

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

1 reference

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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