Gene transfer studies in animals: what do they really tell us about the prospects for gene therapy in DMD? (Q34807072)

4 August 2017

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title

Gene transfer studies in animals: what do they really tell us about the prospects for gene therapy in DMD? (English)

1 reference

4 August 2017

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Dominic J Wells

1

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PubMed ID

12206790

4 August 2017

Kim E Wells

series ordinal

2

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4 August 2017

1 October 2002

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4 August 2017

Neuromuscular Disorders

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4 August 2017

12 Suppl 1

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4 August 2017

S11-22

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Outcome of paediatric domiciliary mask ventilation in neuromuscular and skeletal disease.

4 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin: the protein product of the Duchenne muscular dystrophy locus

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The limb-girdle muscular dystrophies-multiple genes, multiple mechanisms

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

X chromosome-linked muscular dystrophy (mdx) in the mouse

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The molecular basis of muscular dystrophy in the mdx mouse: a point mutation

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Phenotype of dystrophinopathy in old mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Recovery of induced mutations for X chromosome-linked muscular dystrophy in mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The frequency of revertants in mdx mouse genetic models for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dmd(mdx-beta geo): a new allele for the mouse dystrophin gene

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Postsynaptic abnormalities at the neuromuscular junctions of utrophin-deficient mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Subtle neuromuscular defects in utrophin-deficient mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Utrophin-dystrophin-deficient mice as a model for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Skeletal and cardiac myopathies in mice lacking utrophin and dystrophin: a model for Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Muscular dystrophy in a litter of golden retriever dogs

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

An error in dystrophin mRNA processing in golden retriever muscular dystrophy, an animal homologue of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Molecular analysis of a spontaneous dystrophin 'knockout' dog.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Regeneration of dystrophic muscle following multiple injections of bupivacaine.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dp71 can restore the dystrophin-associated glycoprotein complex in muscle but fails to prevent dystrophy.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Exogenous Dp71 restores the levels of dystrophin associated proteins but does not alleviate muscle damage in mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Visualization of dystrophic muscle fibers in mdx mouse by vital staining with Evans blue: evidence of apoptosis in dystrophin-deficient muscle.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Time course of changes in plasma membrane permeability in the dystrophin-deficient mdx mouse

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Understanding dystrophinopathies: an inventory of the structural and functional consequences of the absence of dystrophin in muscles of the mdx mouse

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Adeno-associated virus vector gene transfer and sarcolemmal expression of a 144 kDa micro-dystrophin effectively restores the dystrophin-associated protein complex and inhibits myofibre degeneration in nude/mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Utrophin may be a precursor of dystrophin during skeletal muscle development.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The dystrophin-related protein, utrophin, is expressed on the sarcolemma of regenerating human skeletal muscle fibres in dystrophies and inflammatory myopathies

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Human dystrophin expression corrects the myopathic phenotype in transgenic mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Mdx transgenic mouse: restoration of recombinant dystrophin to the dystrophic muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Prevention of dystrophic pathology in mdx mice by a truncated dystrophin isoform.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Expression of human full-length and minidystrophin in transgenic mdx mice: implications for gene therapy of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Forced expression of dystrophin deletion constructs reveals structure-function correlations.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a "mild Becker" phenotype

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Mdx mice inducibly expressing dystrophin provide insights into the potential of gene therapy for duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Expression of truncated utrophin leads to major functional improvements in dystrophin-deficient muscles of mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Expression of full-length utrophin prevents muscular dystrophy in mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Non-toxic ubiquitous over-expression of utrophin in the mdx mouse

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Enhanced expression of the alpha 7 beta 1 integrin reduces muscular dystrophy and restores viability in dystrophic mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

A nitric oxide synthase transgene ameliorates muscular dystrophy in mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Expression of a recombinant dystrophin in mdx mice using adenovirus vector

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Recombinant truncated dystrophin minigenes: construction, expression, and adenoviral delivery

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Generation, validation, and large scale production of adenoviral recombinants with large size inserts such as a 6.3 kb human dystrophin cDNA.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Study of adenovirus-mediated dystrophin minigene transfer to skeletal muscle by combined microscopic display of adenoviral DNA and dystrophin

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: prolongation of expression with immunosuppression.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Combinatorial blockade of calcineurin and CD28 signaling facilitates primary and secondary therapeutic gene transfer by adenovirus vectors in dystrophic (mdx) mouse muscles

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Effective restoration of dystrophin-associated proteins in vivo by adenovirus-mediated transfer of truncated dystrophin cDNAs

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Differential effects of dystrophin and utrophin gene transfer in immunocompetent muscular dystrophy (mdx) mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The spread of transgene expression at the site of gene construct injection.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Forced myofiber regeneration promotes dystrophin gene transfer and improved muscle function despite advanced disease in old dystrophic mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

DNA from both high-capacity and first-generation adenoviral vectors remains intact in skeletal muscle.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin expression in muscle following gene transfer with a fully deleted ("gutted") adenovirus is markedly improved by trans-acting adenoviral gene products.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Herpes simplex virus vector-mediated dystrophin gene transfer and expression in MDX mouse skeletal muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Direct retroviral-mediated transfer of a dystrophin minigene into mdx mouse muscle in vivo

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Genetic correction of dystrophin deficiency and skeletal muscle remodeling in adult MDX mouse via transplantation of retroviral producer cells.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Adenovirus-mediated utrophin gene transfer mitigates the dystrophic phenotype of mdx mouse muscles.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Prevention of the dystrophic phenotype in dystrophin/utrophin-deficient muscle following adenovirus-mediated transfer of a utrophin minigene

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Viral gene delivery to skeletal muscle: insights on maturation-dependent loss of fiber infectivity for adenovirus and herpes simplex type 1 viral vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Efficient infection of mature skeletal muscle with herpes simplex virus vectors by using dextran sulfate as a co-receptor.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Direct gene transfer into mouse muscle in vivo

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin expression improves myofiber survival in mdx muscle following intramuscular plasmid DNA injection.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Expression of deletion-containing dystrophins in mdx muscle: implications for gene therapy and dystrophin function

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Mini-dystrophin gene transfer in mdx4cv diaphragm muscle fibers increases sarcolemmal stability

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Mini- and full-length dystrophin gene transfer induces the recovery of nitric oxide synthase at the sarcolemma of mdx4cv skeletal muscle fibers

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Enhanced expression of recombinant dystrophin following intramuscular injection of Epstein-Barr virus (EBV)-based mini-chromosome vectors in mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Immune rejection of human dystrophin following intramuscular injections of naked DNA in mdx mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Immune responses to dystropin: implications for gene therapy of Duchenne muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Transfer of full-length Dmd to the diaphragm muscle of Dmd(mdx/mdx) mice through systemic administration of plasmid DNA

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Bacterial beta-galactosidase and human dystrophin genes are expressed in mouse skeletal muscle fibers after ballistic transfection.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Local and distant transfection of mdx muscle fibers with dystrophin and LacZ genes delivered in vivo by synthetic microspheres.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Gene transfer into muscle by electroporation in vivo.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

High-efficiency gene transfer into skeletal muscle mediated by electric pulses

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Electropermeabilization of skeletal muscle enhances gene transfer in vivo

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Efficient and regulated erythropoietin production by naked DNA injection and muscle electroporation

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Optimisation of electrotransfer of plasmid into skeletal muscle by pretreatment with hyaluronidase -- increased expression with reduced muscle damage

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Innate immune mechanisms dominate elimination of adenoviral vectors following in vivo administration.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Production and characterization of improved adenovirus vectors with the E1, E2b, and E3 genes deleted

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Rescue, propagation, and partial purification of a helper virus-dependent adenovirus vector

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Inflammatory responses following direct injection of plasmid DNA into skeletal muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Efficiency of in vivo gene transfer using murine retroviral vectors is strain-dependent in mice

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Factors influencing cross-presentation of non-self antigens expressed from recombinant adeno-associated virus vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Searching for the 1 in 2,400,000: a review of dystrophin gene point mutations

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Somatic reversion/suppression of the mouse mdx phenotype in vivo.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin gene transcripts skipping the mdx mutation

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Small-caliber skeletal muscle fibers do not suffer deleterious consequences of dystrophic gene expression

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The efficient expression of intravascularly delivered DNA in rat muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Efficient expression of naked dna delivered intraarterially to limb muscles of nonhuman primates

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

The abnormal expression of utrophin in Duchenne and Becker muscular dystrophy is age related

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin in skeletal muscle. II. Immunoreactivity in patients with Xp21 muscular dystrophy

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Dystrophin expression and somatic reversion in prednisone-treated and untreated Duchenne dystrophy. CIDD Study Group

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Cellular immune responses of healthy individuals to intradermal administration of an E1-E3- adenovirus gene transfer vector

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

Immune responses to adenovirus and adeno-associated virus in humans.

1 reference

https://api.crossref.org/works/10.1016%2FS0960-8966%2802%2900077-9

7 January 2021

10.1016/S0960-8966(02)00077-9

Identifiers

DOI

1 reference

4 August 2017

1 reference

PubMed ID

12206790