UB Paderborn / Katalog / Suche / in CDI

Sortiert nach

Einfacher Volltextzugriff von (fast) überall

Installieren Sie die LibKey Nomad-Browsererweiterung und erhalten Sie von vielen Webseiten einfachen Zugriff auf lizenzierte Volltexte oder Open Access Publikationen. Mehr erfahren...

Gene therapy for metachromatic leukodystrophy

Journal of neuroscience research, 2016-11, Vol.94 (11), p.1169-1179

2016

Volltextzugriff (PDF)

Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema

Allergy (Copenhagen), 2019-06, Vol.74 (6), p.1081-1089

2019

Volltextzugriff (PDF)

Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Human gene therapy, 2021-06, Vol.32 (11-12), p.563-580

2021

Volltextzugriff (PDF)

Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy

Journal of inherited metabolic disease, 2021-11, Vol.44 (6), p.1382-1392

2021

Volltextzugriff (PDF)

Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy

Journal of allergy and clinical immunology, 2016-12, Vol.138 (6), p.1652-1662.e7

2016

Volltextzugriff (PDF)

Expression and processing of mature human frataxin after gene therapy in mice

Scientific reports, 2024-04, Vol.14 (1), p.8391-8391, Article 8391

2024

Volltextzugriff (PDF)

Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use

PloS one, 2020-11, Vol.15 (11), p.e0239780

2020

Volltextzugriff (PDF)

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

Human gene therapy, 2017-01, Vol.28 (1), p.3-84

2017

Volltextzugriff (PDF)

Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway

Human molecular genetics, 2014-04, Vol.23 (8), p.2005-2022

2014

Volltextzugriff (PDF)

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA

Human gene therapy, 2008-05, Vol.19 (5), p.463-474

2008

Volltextzugriff (PDF)

Biology of the Adrenal Gland Cortex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders

Human gene therapy, 2018-04, Vol.29 (4), p.403-412

2018

Volltextzugriff (PDF)

Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease)

Scientific reports, 2018-10, Vol.8 (1), p.15229-12, Article 15229

2018

Volltextzugriff (PDF)

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

Human gene therapy. Clinical development, 2018-03, Vol.29 (1), p.24-47

2018

Volltextzugriff (PDF)

Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice

Human gene therapy, 2012-08, Vol.23 (8), p.903-914

2012

Volltextzugriff (PDF)

Expressed Protein Ligation: A General Method for Protein Engineering

Proceedings of the National Academy of Sciences - PNAS, 1998-06, Vol.95 (12), p.6705-6710

1998

Volltextzugriff (PDF)

Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector

Molecular therapy, 2007-03, Vol.15 (3), p.481-491

2007

Volltextzugriff (PDF)

Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma

PloS one, 2016-10, Vol.11 (10), p.e0162978-e0162978

2016

Volltextzugriff (PDF)

Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function

PloS one, 2013-08, Vol.8 (8), p.e73128-e73128

2013

Volltextzugriff (PDF)

Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates

Human gene therapy. Clinical development, 2014-09, Vol.25 (3), p.164-177

2014

Volltextzugriff (PDF)

Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice

The Journal of neuroscience, 2016-12, Vol.36 (49), p.12425-12435

2016

Volltextzugriff (PDF)

Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells

Human gene therapy, 2023-08, Vol.34 (15-16), p.697-704

2023

Volltextzugriff (PDF)

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2

Science translational medicine, 2020-12, Vol.12 (572)

2020

Volltextzugriff (PDF)

Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models

Neurobiology of aging, 2016-08, Vol.44, p.159-172

2016

Volltextzugriff (PDF)

Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates

Human gene therapy, 2020-12, Vol.31 (23-24), p.1237-1259

2020

Volltextzugriff (PDF)

Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy

Human gene therapy, 2020-08, Vol.31 (15-16), p.819-827

2020

Volltextzugriff (PDF)

1 – 25 von 97

Suche als RSS-Feed

Suchergebnisse filtern

Filter anzeigen

Nur zeigen

Geprüft (Peer-Review)
(87)
Mit Volltextzugriff
(64)

Publikationsform

Artikel
(95)
Buchkapitel
(2)

Sprache

Englisch
(97)

Erscheinungsjahr

n.n

Thema

Science & Technology
(57)
Life Sciences & Biomedicine
(51)
Humans
(39)
Animals
(37)
Gene Therapy
(37)
Medicine, Research & Experimental
(29)
Research & Experimental Medicine
(29)
Biotechnology & Applied Microbiology
(27)
Genetics & Heredity
(25)
Mice
(22)
Dependovirus - Genetics
(21)
Genetic Vectors - Genetics
(16)
Male
(16)
Adeno-Associated Virus
(14)
Female
(13)
Genetic Therapy
(13)
Genetic Therapy - Methods
(12)
Child
(11)
Neuronal Ceroid-Lipofuscinoses - Genetics
(11)
Neuronal Ceroid-Lipofuscinoses - Therapy
(11)

15 weitere anzeigen...

Weniger anzeigen...

Menü

Weitere Dienste

Einstellungen

Gene therapy for metachromatic leukodystrophy

Gene therapy for C1 esterase inhibitor deficiency in a Murine Model of Hereditary angioedema

Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy

Anti-hIgE gene therapy of peanut-induced anaphylaxis in a humanized murine model of peanut allergy

Expression and processing of mature human frataxin after gene therapy in mice

Cocaine vaccine dAd5GNE protects against moderate daily and high-dose "binge" cocaine use

Genetic Modification of the Lung Directed Toward Treatment of Human Disease

Human iPSC models of neuronal ceroid lipofuscinosis capture distinct effects of TPP1 and CLN3 mutations on the endocytic pathway

Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA

Biology of the Adrenal Gland Cortex Obviates Effective Use of Adeno-Associated Virus Vectors to Treat Hereditary Adrenal Disorders

Untargeted Metabolite Profiling of Cerebrospinal Fluid Uncovers Biomarkers for Severity of Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2, Batten Disease)

AAVrh.10-Mediated APOE2 Central Nervous System Gene Therapy for APOE4-Associated Alzheimer's Disease

Correction of brain oligodendrocytes by AAVrh.10 intracerebral gene therapy in metachromatic leukodystrophy mice

Expressed Protein Ligation: A General Method for Protein Engineering

Enhanced Survival of the LINCL Mouse Following CLN2 Gene Transfer Using the rh.10 Rhesus Macaque-derived Adeno-associated Virus Vector

Anti-Epidermal Growth Factor Receptor Gene Therapy for Glioblastoma

Spectrum of ocular manifestations in CLN2-associated batten (Jansky-Bielschowsky) disease correlate with advancing age and deteriorating neurological function

Comparative efficacy and safety of multiple routes of direct CNS administration of adeno-associated virus gene transfer vector serotype rh.10 expressing the human arylsulfatase A cDNA to nonhuman primates

Vectored Intracerebral Immunization with the Anti-Tau Monoclonal Antibody PHF1 Markedly Reduces Tau Pathology in Mutant Tau Transgenic Mice

Assessment of Residual Full-Length SV40 Large T Antigen in Clinical-Grade Adeno-Associated Virus Vectors Produced in 293T Cells

Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2

Intracerebral adeno-associated virus gene delivery of apolipoprotein E2 markedly reduces brain amyloid pathology in Alzheimer's disease mouse models

Quantitative Whole-Body Imaging of I-124-Labeled Adeno-Associated Viral Vector Biodistribution in Nonhuman Primates

Stress-Induced Mouse Model of the Cardiac Manifestations of Friedreich's Ataxia Corrected by AAV-mediated Gene Therapy

Suchergebnisse filtern