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Establishment of two quantitative nested qPCR assays targeting the human EPO transgene

Gene therapy, 2016-04, Vol.23 (4), p.330-339

2016

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Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

Gene therapy, 2015-04, Vol.22 (4), p.316-324

2015

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Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats

Gene therapy, 2014-05, Vol.21 (5), p.522-528

2014

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Tolerogenic dendritic cells actively inhibit T cells through heme oxygenase-1 in rodents and in nonhuman primates

The FASEB journal, 2009-09, Vol.23 (9), p.3070-3077

2009

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Long-term expression of melanopsin and channelrhodopsin causes no gross alterations in the dystrophic dog retina

Gene therapy, 2017-11, Vol.24 (11), p.735-741

2017

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Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

Gene therapy, 2007-02, Vol.14 (4), p.292-303

2007

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Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping

Gene therapy, 2011-07, Vol.18 (7), p.709-718

2011

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G.P.41

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.807-807

2014

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Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)

Neuromuscular disorders : NMD, 2015, Vol.25, p.S315-S315

2015

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A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

Molecular therapy, 2011-11, Vol.19 (11), p.2084-2091

2011

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Oral administration of doxycycline allows tight control of transgene expression: a key step towards gene therapy of retinal diseases

Gene therapy, 2007-12, Vol.14 (23), p.1668-1673

2007

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Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells

Gene therapy, 2000-10, Vol.7 (19), p.1613-1623

2000

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P.20.13 Gene therapy of Duchenne Muscular Dystrophy using rAAV vectors: Exon skipping and microdystrophin approaches in GRMD dogs

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.842-843

2013

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T.I.3 Perspectives on microdystrophins and delivery

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.847-847

2013

Volltextzugriff (PDF)

P.20.12 Gene therapy of Duchenne Muscular Dystrophy using rAAV vectors: Patterns of dystrophin expression and histological improvements

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.842-842

2013

Volltextzugriff (PDF)

O.6 Restricting MTM1 transgene expression to skeletal muscle in AAV-mediated gene therapy for myotubular myopathy

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.741-741

2013

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G.P.92

Neuromuscular disorders : NMD, 2014-10, Vol.24 (9), p.822-822

2014

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Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors

The Journal of immunology (1950), 2012-06, Vol.188 (12), p.6418-6424

2012

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P.20.14 Non ambulant patients with deletion treatable by exon skipping 53 present a more severe phenotype than the general Duchenne population

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.843-843

2013

Volltextzugriff (PDF)

Design and construction of functional AAV vectors

Methods in molecular biology (Clifton, N.J.), 2011-01, Vol.807, p.25

2011

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P.4.3 Intravenous infusion of AAV8–MTM1 prolongs life and ameliorates severe muscle pathology in mouse and dog models of X-linked myotubular myopathy

Neuromuscular disorders : NMD, 2013-10, Vol.23 (9), p.760-760

2013

Volltextzugriff (PDF)

AAV-mediated liver-directed gene therapy

Methods in molecular biology (Clifton, N.J.), 2011, Vol.807, p.141

Sands, Mark S

2011

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O.4 Forelimb loco-regional injection of rAAV8-U7snRNA in GRMD dogs allows dose-dependant dystrophin expression and phenotypic correction

Neuromuscular disorders : NMD, 2011-10, Vol.21 (9), p.641-641

2011

Volltextzugriff (PDF)

Baculovirus vector requires electrostatic interactions including heparan sulfate for efficient gene transfer in mammalian cells

The journal of gene medicine, 1999-03, Vol.1 (2), p.93-102

1999

Volltextzugriff (PDF)

Novel cis-Acting Replication Element in the Adeno-Associated Virus Type 2 Genome Is Involved in Amplification of Integrated rep-cap Sequences

Journal of Virology, 2001-10, Vol.75 (20), p.9991-9994

2001

Volltextzugriff (PDF)

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Establishment of two quantitative nested qPCR assays targeting the human EPO transgene

Efficient central nervous system AAVrh10-mediated intrathecal gene transfer in adult and neonate rats

Intracisternal delivery of AAV9 results in oligodendrocyte and motor neuron transduction in the whole central nervous system of cats

Tolerogenic dendritic cells actively inhibit T cells through heme oxygenase-1 in rodents and in nonhuman primates

Long-term expression of melanopsin and channelrhodopsin causes no gross alterations in the dystrophic dog retina

Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping

G.P.41

Adeno-associated virus vector (AAV) microdystrophin gene therapy prolongs survival and restores muscle function in the canine model of Duchenne muscular dystrophy (DMD)

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

Oral administration of doxycycline allows tight control of transgene expression: a key step towards gene therapy of retinal diseases

Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells

P.20.13 Gene therapy of Duchenne Muscular Dystrophy using rAAV vectors: Exon skipping and microdystrophin approaches in GRMD dogs

T.I.3 Perspectives on microdystrophins and delivery

P.20.12 Gene therapy of Duchenne Muscular Dystrophy using rAAV vectors: Patterns of dystrophin expression and histological improvements

O.6 Restricting MTM1 transgene expression to skeletal muscle in AAV-mediated gene therapy for myotubular myopathy

G.P.92

Humoral and cellular capsid-specific immune responses to adeno-associated virus type 1 in randomized healthy donors

P.20.14 Non ambulant patients with deletion treatable by exon skipping 53 present a more severe phenotype than the general Duchenne population

Design and construction of functional AAV vectors

P.4.3 Intravenous infusion of AAV8–MTM1 prolongs life and ameliorates severe muscle pathology in mouse and dog models of X-linked myotubular myopathy

AAV-mediated liver-directed gene therapy

O.4 Forelimb loco-regional injection of rAAV8-U7snRNA in GRMD dogs allows dose-dependant dystrophin expression and phenotypic correction

Baculovirus vector requires electrostatic interactions including heparan sulfate for efficient gene transfer in mammalian cells

Novel cis-Acting Replication Element in the Adeno-Associated Virus Type 2 Genome Is Involved in Amplification of Integrated rep-cap Sequences

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