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Innovative therapy for Classic Galactosemia - tale of two HTS.

published in: Molecular Genetics and Metabolism

date of publication: 2011-10-01

main subject: galactosemia

Cites articles 125

Date

Title

Authors

2011-09-01

Structure-activity analysis and cell-based optimization of human galactokinase inhibitors
2011-08-16

Identification of novel small molecule inhibitors of 4-diphosphocytidyl-2-C-methyl-D-erythritol (CDP-ME) kinase of Gram-negative bacteria
2011-07-25

The structural and molecular biology of type I galactosemia: Enzymology of galactose 1-phosphate uridylyltransferase

David J. Timson
2011-07-21

The adult galactosemic phenotype

Robert C. Green, Nancy L Potter, Corrine K. Welt, Gerard Berry, David T. Miller
2011-01-01

Gene therapy for lysosomal storage diseases: progress, challenges and future prospects
2011-05-31

Allogeneic hematopoietic stem cell transplantation for sickle cell disease: the time is now.
2011-03-23

Systemic administration of PRO051 in Duchenne's muscular dystrophy

Annemieke Aartsma-Rus, Gert-Jan van Ommen, Jan J Verschuuren
2011-01-07

New developments in the treatment of hyperammonemia: emerging use of carglumic acid

Giancarlo la Marca, Luca Filippi
2011-02-01

Identification of a NBD1-binding pharmacological chaperone that corrects the trafficking defect of F508del-CFTR

David Y. Thomas
2011-01-19

Is prenatal myo-inositol deficiency a mechanism of CNS injury in galactosemia?

Gerard Berry
2010-12-01

Enzyme replacement therapy of lysosomal storage diseases

Dominique P Germain
2010-12-05

Molecular and biochemical characterization of human galactokinase and its small molecule inhibitors

Klaas J. Wierenga
2010-01-01

Career moves: induced pluripotent cells from human aortic smooth muscle cells can efficiently redifferentiate into parental phenotype
2010

Roles of inositol phosphates and inositol pyrophosphates in development, cell signaling and nuclear processes
2010-01-01

Converting an injectable protein therapeutic into an oral form: phenylalanine ammonia lyase for phenylketonuria

Charles R. Scriver
2010-11-30

Introduction to the Maastricht workshop: lessons from the past and new directions in galactosemia

Gerard Berry
2010-12-15

Immunoglobulin abnormalities and effects of enzyme replacement therapy in children with Gaucher disease
2009-11-01

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

Veronique Kiermer, Alain Fischer, Marina Cavazzana-Calvo, Salima Hacein-Bey-Abina, Laure Caccavelli, Emmanuel Payen, Nizar Mahlaoui, Christof Von Kalle, Philippe Leboulch, Pierre Bougnères, Manfred Schmidt, Patrick Aubourg, Cynthia C Bartholomae, Stéphane Blanche, Ulrich Abel, Najiba Lahlou, Nathalie Cartier
2009-11

Galactose toxicity in animals

Klaas J. Wierenga
2009-10-23

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Katherine A. High, Alberto Auricchio, Eric A Pierce, Francesco Testa, Sandro Banfi, Frauke Coppieters, Elfride De Baere, Enrico M. Surace, T. Michael Redmond, Edwin M. Stone, Francesca Simonelli, Albert M Maguire, Kathleen Marshall, Settimio Rossi, Federico Mingozzi
2009-04-12

Stop codon read-through of a methylmalonic aciduria mutation
2009-01-01

Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease

Anthony Donsante, Christine R. Kaneski
2009-10-01

Knockout mice in understanding the mechanism of action of lithium

Gerard Berry, Robert H. Belmaker, Galila Agam
2008-01-01

ARHI: A new target of galactose toxicity in Classic Galactosemia

Klaas J. Wierenga
2008-11-21

The mevalonate pathway of Staphylococcus aureus
2008-12-01

The unexplored potential of the pentose phosphate pathway in health and disease

Gerard Berry
2008-09-18

PI3K pathway alterations in cancer: variations on a theme
2008-09-14

The Mycobacterium tuberculosis MEP (2C-methyl-d-erythritol 4-phosphate) pathway as a new drug target
2008-05-13

Virtual screening for novel COX-2 inhibitors using the ZINC database
2008-06-29

Pluripotent stem cells induced from adult neural stem cells by reprogramming with two factors

Guangming Wu, Marcos J Araúzo-Bravo, Hans Schöler, Luca Gentile
2008-01-01

Galactosemia and amenorrhea in the adolescent

Gerard Berry
2008-05-19

High-throughput screening for human galactokinase inhibitors

Peter Buchwald, Klaas J. Wierenga

Article - wd:Q34054239

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