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Transfection of mammalian cells using linear polyethylenimine is a simple and effective means of producing recombinant adeno-associated virus vectors

Journal of virological methods, 2006-12, Vol.138 (1), p.85-98

2006

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Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA Splicing

Molecular therapy, 2007-08, Vol.15 (8), p.1471-1478

2007

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Immunological Barriers to Stem Cell Therapy in the Central Nervous System

Stem Cells International, 2014-01, Vol.2014 (2014), p.288-299

2014

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Phenotypic characterization of a mouse model of juvenile neuronal ceroid lipofuscinosis

Neurobiology of disease, 2008-02, Vol.29 (2), p.242-253

2008

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Characterization of the Transcription Profile of Adeno-Associated Virus Type 5 Reveals a Number of Unique Features Compared to Previously Characterized Adeno-Associated Viruses

Journal of Virology, 2002-12, Vol.76 (24), p.12435-12447

2002

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Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector

Molecular therapy, 2006-07, Vol.14 (1), p.54-62

2006

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Ocular phenotype in a mouse gene knockout model for infantile neuronal ceroid lipofuscinosis

Journal of neuroscience research, 2006-10, Vol.84 (5), p.1139-1149

2006

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Quantitation of encapsidated recombinant adeno-associated virus DNA in crude cell lysates and tissue culture medium by quantitative, real-time PCR

Journal of virological methods, 2006-11, Vol.137 (2), p.193-204

2006

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Efficient Replication of Adeno-Associated Virus Type 2 Vectors: a cis-Acting Element outside of the Terminal Repeats and a Minimal Size

Journal of Virology, 2000-12, Vol.74 (24), p.11511-11521

2000

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Neural precursors isolated from the developing cat brain show retinal integration following transplantation to the retina of the dystrophic cat

Veterinary ophthalmology, 2007-07, Vol.10 (4), p.245-253

2007

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Behavioral assessment in mouse models of neuronal ceroid lipofuscinosis using a light-cued T-maze

Behavioural brain research, 2005-06, Vol.161 (2), p.175-182

2005

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415. Stimulating Full-Length SMN2 Expression by Delivering Bi-Functional RNAs Via a Viral Vector

Molecular therapy, 2006-05, Vol.13 (S1), p.S159-S160

2006

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Nonsense mutations inhibit splicing of MVM RNA in cis when they interrupt the reading frame of either exon of the final spliced product

Genes & development, 1992-06, Vol.6 (6), p.1107-1119

1992

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The minor capsid protein VP1 of the autonomous parvovirus minute virus of mice is dispensable for encapsidation of progeny single-stranded DNA but is required for infectivity

Journal of Virology, 1993-01, Vol.67 (1), p.131-141

1993

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The trypsin-sensitive RVER domain in the capsid proteins of minute virus of mice is required for efficient cell binding and viral infection but not for proteolytic processing in Vivo

Virology (New York, N.Y.), 1992-12, Vol.191 (2), p.846-857

1992

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Post-Transcriptional Regulation of RNA Polymerase II Levels in Caenorhabditis elegans

Genetics (Austin), 1993-02, Vol.133 (2), p.237-245

1993

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Effective Treatment for the Canine RPE65 Null Mutation, a Hereditary Retinal Dystrophy Comparable to Human Leber’s Congenital Amaurosis

Retinal Degenerations, p.415-431

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Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

The New England journal of medicine, 2019-11, Vol.381 (19), p.1809-1819

2019

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HER2-HER3 Heterodimer Quantification by FRET-FLIM and Patient Subclass Analysis of the COIN Colorectal Trial

JNCI : Journal of the National Cancer Institute, 2020-09, Vol.112 (9), p.944-954

2020

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Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

The New England journal of medicine, 2015-07, Vol.373 (3), p.220-231

2015

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A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

The New England journal of medicine, 2011-11, Vol.365 (18), p.1663-1672

2011

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Imaging tumour heterogeneity of the consequences of a PKCα-substrate interaction in breast cancer patients

Biochemical Society transactions, 2014-12, Vol.42 (6), p.1498-1505

2014

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Airway Inflammation and Infection in Congenital Bilateral Absence of the Vas Deferens

American journal of respiratory and critical care medicine, 2004-01, Vol.169 (2), p.174-179

2004

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Adenovirus E1A proteins interact with the cellular YY1 transcription factor

Journal of Virology, 1995-03, Vol.69 (3), p.1628-1636

1995

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Neurosurgical Operative Atlas: Spine and Peripheral Nerves

Third Edition.

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Transfection of mammalian cells using linear polyethylenimine is a simple and effective means of producing recombinant adeno-associated virus vectors

Restoration of SMN Function: Delivery of a Trans-splicing RNA Re-directs SMN2 Pre-mRNA Splicing

Immunological Barriers to Stem Cell Therapy in the Central Nervous System

Phenotypic characterization of a mouse model of juvenile neuronal ceroid lipofuscinosis

Characterization of the Transcription Profile of Adeno-Associated Virus Type 5 Reveals a Number of Unique Features Compared to Previously Characterized Adeno-Associated Viruses

Stimulating Full-Length SMN2 Expression by Delivering Bifunctional RNAs via a Viral Vector

Ocular phenotype in a mouse gene knockout model for infantile neuronal ceroid lipofuscinosis

Quantitation of encapsidated recombinant adeno-associated virus DNA in crude cell lysates and tissue culture medium by quantitative, real-time PCR

Efficient Replication of Adeno-Associated Virus Type 2 Vectors: a cis-Acting Element outside of the Terminal Repeats and a Minimal Size

Neural precursors isolated from the developing cat brain show retinal integration following transplantation to the retina of the dystrophic cat

Behavioral assessment in mouse models of neuronal ceroid lipofuscinosis using a light-cued T-maze

415. Stimulating Full-Length SMN2 Expression by Delivering Bi-Functional RNAs Via a Viral Vector

Nonsense mutations inhibit splicing of MVM RNA in cis when they interrupt the reading frame of either exon of the final spliced product

The minor capsid protein VP1 of the autonomous parvovirus minute virus of mice is dispensable for encapsidation of progeny single-stranded DNA but is required for infectivity

The trypsin-sensitive RVER domain in the capsid proteins of minute virus of mice is required for efficient cell binding and viral infection but not for proteolytic processing in Vivo

Post-Transcriptional Regulation of RNA Polymerase II Levels in Caenorhabditis elegans

Effective Treatment for the Canine RPE65 Null Mutation, a Hereditary Retinal Dystrophy Comparable to Human Leber’s Congenital Amaurosis

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

HER2-HER3 Heterodimer Quantification by FRET-FLIM and Patient Subclass Analysis of the COIN Colorectal Trial

Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR

A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

Imaging tumour heterogeneity of the consequences of a PKCα-substrate interaction in breast cancer patients

Airway Inflammation and Infection in Congenital Bilateral Absence of the Vas Deferens

Adenovirus E1A proteins interact with the cellular YY1 transcription factor

Neurosurgical Operative Atlas: Spine and Peripheral Nerves

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