UB Paderborn / Katalog / Suche / in CDI

1 – 25 von 311

Sortiert nach

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Annual review of virology, 2019-09, Vol.6 (1), p.601-621

2019

Link zum Volltext

Verfügbarkeit prüfen

Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders

International journal of molecular sciences, 2024-01, Vol.25 (2), p.1050

2024

Link zum Volltext

Verfügbarkeit prüfen

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

Nature communications, 2021-04, Vol.12 (1), p.2121-13, Article 2121

2021

Link zum Volltext

Verfügbarkeit prüfen

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

Ophthalmology (Rochester, Minn.), 2016-07, Vol.123 (7), p.1606-1620

2016

Link zum Volltext

Verfügbarkeit prüfen

Gene therapy: The first two decades and the current state-of-the-art

Journal of cellular physiology, 2007-11, Vol.213 (2), p.301-305

Flotte, Terence R.

2007

Link zum Volltext

Verfügbarkeit prüfen

Women’s Representation Among Members and Leaders of National Medical Specialty Societies

Academic medicine, 2020-07, Vol.95 (7), p.1043-1049

2020

Link zum Volltext

Verfügbarkeit prüfen

Common pathways to Dean of Medicine at U.S. medical schools

PloS one, 2021-03, Vol.16 (3), p.e0249078-e0249078

2021

Link zum Volltext

Verfügbarkeit prüfen

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Proceedings of the National Academy of Sciences - PNAS, 2008-09, Vol.105 (39), p.15112-15117

2008

Link zum Volltext

Verfügbarkeit prüfen

Long-term, efficient inhibition of microRNA function in mice using rAAV vectors

Nature methods, 2012-03, Vol.9 (4), p.403

2012

Link zum Volltext

Verfügbarkeit prüfen

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Proceedings of the National Academy of Sciences - PNAS, 2009-09, Vol.106 (38), p.16363-16368

2009

Link zum Volltext

Verfügbarkeit prüfen

Intrathecal gene therapy for neurologic disease in humans

Molecular therapy, 2024-05, Vol.32 (5), p.1185-1186

Flotte, Terence R.

2024

Link zum Volltext

Verfügbarkeit prüfen

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

Human gene therapy, 2015-12, Vol.26 (12), p.779

2015

Link zum Volltext

Verfügbarkeit prüfen

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

Molecular therapy, 2011-03, Vol.19 (3), p.526-535

2011

Link zum Volltext

Verfügbarkeit prüfen

Precision of Exon Skipping with U7 Constructs

Human gene therapy, 2021-11, Vol.32 (21-22), p.1315

Flotte, Terence R

2021

Link zum Volltext

Verfügbarkeit prüfen

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. Reply

The New England journal of medicine, 2023-12, Vol.389 (23), p.2211-2211

2023

Link zum Volltext

Verfügbarkeit prüfen

Base Editing to the Rescue

Human gene therapy, 2021-07, Vol.32 (13-14), p.647

Flotte, Terence R

2021

Link zum Volltext

Verfügbarkeit prüfen

CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing

Human gene therapy, 2021-03, Vol.32 (5-6), p.235

Flotte, Terence R

2021

Link zum Volltext

Verfügbarkeit prüfen

Liver targeting with rAAV7: balancing tropism with immune profiles

Gene therapy, 2021-04, Vol.28 (3-4), p.115-116

Flotte, Terence R

2021

Link zum Volltext

Verfügbarkeit prüfen

Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia

Human gene therapy, 2021-05, Vol.32 (9-10), p.449

Flotte, Terence R

2021

Link zum Volltext

Verfügbarkeit prüfen

Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

Molecular therapy. Methods & clinical development, 2021-12, Vol.23, p.490-506

2021

Link zum Volltext

Verfügbarkeit prüfen

Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies

Molecular therapy, 2023-04, Vol.31 (4), p.913-914

2023

Link zum Volltext

Verfügbarkeit prüfen

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

Molecular therapy. Methods & clinical development, 2022-06, Vol.25, p.425-438

2022

Link zum Volltext

Verfügbarkeit prüfen

Epigenome Editing Strategies for Low Back Pain

Human gene therapy, 2019-09, Vol.30 (9), p.1037

Flotte, Terence R

2019

Link zum Volltext

Verfügbarkeit prüfen

In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis

Molecular therapy, 2021-09, Vol.29 (9), p.2633-2634

2021

Link zum Volltext

Verfügbarkeit prüfen

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

Molecular therapy. Methods & clinical development, 2018-12, Vol.11, p.65-72

2018

Link zum Volltext

Verfügbarkeit prüfen

1 – 25 von 311

Suche als RSS-Feed

Suchergebnisse filtern

Filter anzeigen

Nur zeigen

Geprüft (Peer-Review)
(295)
Mit Volltextzugriff
(222)

Publikationsform

Sprache

Englisch
(311)
Japanisch
(43)

Erscheinungsjahr

n.n

Thema

Science & Technology
(211)
Life Sciences & Biomedicine
(198)
Humans
(167)
Animals
(128)
Medicine, Research & Experimental
(128)
Research & Experimental Medicine
(128)
Biotechnology & Applied Microbiology
(116)
Genetics & Heredity
(116)
Gene Therapy
(101)
Dependovirus - Genetics
(96)
Mice
(70)
Adeno-Associated Virus
(65)
Genetic Vectors
(65)
Genetic Therapy
(63)
Biological And Medical Sciences
(51)
Genetic Therapy - Methods
(49)
Medical Sciences
(39)
Genetic Vectors - Genetics
(37)
Male
(37)
Fundamental And Applied Biological Sciences. Psychology
(36)

15 weitere anzeigen...

Weniger anzeigen...

Neu hinzugefügt

... seit 90 Tagen
(2)

Menü

Weitere Dienste

Einstellungen

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

Delivery of Adeno-Associated Virus Vectors to the Central Nervous System for Correction of Single Gene Disorders

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood–Onset Retinal Dystrophy

Gene therapy: The first two decades and the current state-of-the-art

Women’s Representation Among Members and Leaders of National Medical Specialty Societies

Common pathways to Dean of Medicine at U.S. medical schools

Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

Long-term, efficient inhibition of microRNA function in mice using rAAV vectors

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

Intrathecal gene therapy for neurologic disease in humans

Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?

MicroRNA-regulated, Systemically Delivered rAAV9: A Step Closer to CNS-restricted Transgene Expression

Precision of Exon Skipping with U7 Constructs

Death after High-Dose rAAV9 Gene Therapy in a Patient with Duchenne's Muscular Dystrophy. Reply

Base Editing to the Rescue

CRISPR Keeps Things Fresh: Next-Generation Tools for Gene Editing

Liver targeting with rAAV7: balancing tropism with immune profiles

Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia

Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

Analyzing clinical observations to better understand and manage immune responses to AAV gene therapies

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

Epigenome Editing Strategies for Low Back Pain

In vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis

Adeno-Associated Virus Neutralizing Antibodies in Large Animals and Their Impact on Brain Intraparenchymal Gene Transfer

Suchergebnisse filtern