(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

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14 August 2017

Ronald G Crystal

series ordinal

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14 August 2017

publication date

1 April 2006

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14 August 2017

published in

Nature Reviews Genetics

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14 August 2017

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14 August 2017

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14 August 2017

page(s)

261-276

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Synthetic hammerhead ribozymes as therapeutic tools to control disease genes

14 August 2017

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Gene and stem cell therapies

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31 March 2020

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Adenovirus-mediated transfer of a minigene expressing multiple isoforms of VEGF is more effective at inducing angiogenesis than comparable vectors expressing individual VEGF cDNAs

31 March 2020

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Adenoviruses as gene-delivery vehicles

31 March 2020

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Gene therapy progress and prospects: viral trafficking during infection

31 March 2020

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Cell physiology as a variable in gene transfer to endothelium

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31 March 2020

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Non-viral vectors in cystic fibrosis gene therapy: progress and challenges

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Progress and problems with the use of viral vectors for gene therapy

31 March 2020

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

31 March 2020

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Modification to the capsid of the adenovirus vector that enhances dendritic cell infection and transgene-specific cellular immune responses

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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Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis.

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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Circumvention of anti-adenovirus neutralizing immunity by administration of an adenoviral vector of an alternate serotype

31 March 2020

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Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

31 March 2020

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Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

31 March 2020

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Gene transfer as an approach to treating hemophilia

31 March 2020

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Analysis of risk factors for local delivery of low- and intermediate-dose adenovirus gene transfer vectors to individuals with a spectrum of comorbid conditions

31 March 2020

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Safety of local delivery of low- and intermediate-dose adenovirus gene transfer vectors to individuals with a spectrum of morbid conditions

31 March 2020

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High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses

31 March 2020

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Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

31 March 2020

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Building a better vector: the manipulation of AAV virions

31 March 2020

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Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors

31 March 2020

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Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector

31 March 2020

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Adeno-associated virus type 5 (AAV5) but not AAV2 binds to the apical surfaces of airway epithelia and facilitates gene transfer

31 March 2020

1 reference

AAV-mediated gene transfer for treatment of hemophilia

31 March 2020

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Gene therapy progress and prospects: gene therapy of lysosomal storage disorders

31 March 2020

1 reference

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

31 March 2020

1 reference

AAV vector-mediated correction of brain pathology in a mouse model of Niemann-Pick A disease

31 March 2020

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Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

31 March 2020

1 reference

Adeno-associated virus vectors in clinical trials

31 March 2020

1 reference

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study

31 March 2020

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Gene therapy for hemophilia: an imperative to succeed

31 March 2020

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Expanding AAV packaging capacity with trans-splicing or overlapping vectors: a quantitative comparison

31 March 2020

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Genetic medicine at the RNA level: modifications of the genetic repertoire for therapeutic purposes by pre-mRNA trans-splicing

31 March 2020

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Gene therapy of storage disorders by retroviral and lentiviral vectors

31 March 2020

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Retroviral vectors: new applications for an old tool

31 March 2020

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Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia

31 March 2020

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Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

31 March 2020

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Protection and selection for gene therapy in the hematopoietic system

31 March 2020

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Gene therapy for severe combined immunodeficiency

31 March 2020

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Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.

31 March 2020

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Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics

31 March 2020

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Genome-wide analysis of retroviral DNA integration

31 March 2020

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

31 March 2020

1 reference

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

31 March 2020

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Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

31 March 2020

1 reference

In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

31 March 2020

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Progress toward the genetic treatment of the beta-thalassemias.

31 March 2020

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Progress in antisense technology.

31 March 2020

1 reference

Toxicology of antisense therapeutics

31 March 2020

1 reference

Antisense oligonucleotides in the treatment of Duchenne muscular dystrophy: Where are we now?

31 March 2020

1 reference

The silent revolution: RNA interference as basic biology, research tool, and therapeutic

31 March 2020

1 reference

Approaches for the sequence-specific knockdown of mRNA.

31 March 2020

1 reference

Adeno-associated virus vectors for short hairpin RNA expression

31 March 2020

1 reference

Expression profiling reveals off-target gene regulation by RNAi

31 March 2020

1 reference

RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

31 March 2020

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RNAi suppresses polyglutamine-induced neurodegeneration in a model of spinocerebellar ataxia.

31 March 2020

1 reference

Spliceosome-mediated RNA trans-splicing as a tool for gene therapy

31 March 2020

1 reference

Phenotype correction of hemophilia A mice by spliceosome-mediated RNA trans-splicing

31 March 2020

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Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency.

31 March 2020

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Partial correction of endogenous DeltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing

31 March 2020

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In vivo trans-splicing of 5' and 3' segments of pre-mRNA directed by corresponding DNA sequences delivered by gene transfer

31 March 2020

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Suppression of transthyretin expression by ribozymes: a possible therapy for familial amyloidotic polyneuropathy

31 March 2020

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Correction of factor IX deficiency in mice by embryonic stem cells differentiated in vitro

31 March 2020

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Human embryonic stem cell lines with genetic disorders

31 March 2020

1 reference

Embryonic stem cells and the challenge of transplantation tolerance

31 March 2020

1 reference

Human embryonic stem cells express an immunogenic nonhuman sialic acid

31 March 2020

1 reference

RETRACTED: Patient-specific embryonic stem cells derived from human SCNT blastocysts.

31 March 2020

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31 March 2020

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Neural subtype specification of fertilization and nuclear transfer embryonic stem cells and application in parkinsonian mice.

31 March 2020

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Dopamine neurons derived from embryonic stem cells function in an animal model of Parkinson's disease.

31 March 2020

1 reference

Embryonic stem cells develop into functional dopaminergic neurons after transplantation in a Parkinson rat model

31 March 2020

1 reference

Highly efficient endogenous human gene correction using designed zinc-finger nucleases

31 March 2020

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Gene therapy: the moving finger

31 March 2020

1 reference

Enzyme replacement and enhancement therapies for lysosomal diseases

31 March 2020

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Stem cells find their niche

31 March 2020

1 reference

Out of Eden: stem cells and their niches

31 March 2020

1 reference

The end of the beginning for pluripotent stem cells

31 March 2020

1 reference

Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa.

31 March 2020

1 reference

GLI3 zinc-finger gene interrupted by translocations in Greig syndrome families

31 March 2020

1 reference

Alpha 1-antitrypsin deficiency, emphysema, and liver disease. Genetic basis and strategies for therapy

31 March 2020

1 reference

The development of neural stem cells

31 March 2020

1 reference

Stem cell biology and neurodegenerative disease

31 March 2020

1 reference

Systematic determination of the packaging limit of lentiviral vectors

31 March 2020

1 reference

Confronting the issues of therapeutic misconception, enrollment decisions, and personal motives in genetic medicine-based clinical research studies for fatal disorders

31 March 2020

1 reference

Gene therapy: theoretical and bioethical concepts

31 March 2020

1 reference

Regulatory issues for clinical gene therapy trials

31 March 2020

1 reference

Embryonic and extraembryonic stem cell lines derived from single mouse blastomeres.

31 March 2020

1 reference

Generation of nuclear transfer-derived pluripotent ES cells from cloned Cdx2-deficient blastocysts

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

Cationic lipid-mediated CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial.

31 March 2020

1 reference

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

1 reference

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

31 March 2020

1 reference

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

1 reference

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

1 reference

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

31 March 2020

1 reference

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

31 March 2020

1 reference

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

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/NRG1829

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Dimensions Publication ID

14 August 2017

1018932463

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