Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex. - Wikidata - awgldk - TriplyDB

Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex.

Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex.

http://www.wikidata.org/entity/Q35575697

about

Non-specific accumulation of glycosphingolipids in GNE myopathy Dissecting darwinism Sialylation of Thomsen-Friedenreich antigen is a noninvasive blood-based biomarker for GNE myopathy.UDP-GlcNAc 2-Epimerase/ManNAc Kinase (GNE): A Master Regulator of Sialic Acid Synthesis.Mutation update for GNE gene variants associated with GNE myopathy.Neurology of inherited glycosylation disorders.GNE myopathy: current update and future therapy.Optimization of Non-Viral Gene Therapeutics Using Bilamellar Invaginated Vesicles.Oral monosaccharide therapies to reverse renal and muscle hyposialylation in a mouse model of GNE myopathy.Murine isoforms of UDP-GlcNAc 2-epimerase/ManNAc kinase: Secondary structures, expression profiles, and response to ManNAc therapy Preclinical Justification of pbi-shRNA EWS/FLI1 Lipoplex (LPX) Treatment for Ewing's Sarcoma.The nutritional role of free sialic acid, a human milk monosaccharide, and its application as a functional food ingredient.[GNE myopathy].Reversible masking using low-molecular-weight neutral lipids to achieve optimal-targeted delivery.Prevalence of GNE p.M712T and hereditary inclusion body myopathy (HIBM) in Sangesar population of Northern Iran.Phenotypic stratification and genotype-phenotype correlation in a heterogeneous, international cohort of GNE myopathy patients: First report from the GNE myopathy Disease Monitoring Program, registry portion.[GNE myopathy: proven failure of sialic acid supplementation… what's next?]CDG Therapies: From Bench to Bedside.GNE myopathy: from clinics and genetics to pathology and research strategies.GNE Myopathy (Hereditary Inclusion-Body Myopathy/Distal Myopathy with Rimmed Vacuoles): Clinical Features and Epidemiology

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P2860

Hereditary inclusion body myopathy: single patient response to intravenous dosing of GNE gene lipoplex.

http://www.wikidata.org/entity/Q35575697

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2011 nî lūn-bûn

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Human Gene Therapy

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Hereditary inclusion body myop ...... s dosing of GNE gene lipoplex.

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Alex W Tong

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Anagha P Phadke

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Beena O Pappen

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Cathy Hernandez

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Chris M Jay

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Cynthia Bedell

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Gregory Nemunaitis

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Henry Allen

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John Nemunaitis

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Joseph Kuhn

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P2860

UDP-GlcNAc 2-epimerase: a regulator of cell surface sialylation

A bifunctional enzyme catalyzes the first two steps in N-acetylneuraminic acid biosynthesis of rat liver. Purification and characterization of UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase

Hereditary inclusion body myopathy: a decade of progress

Standardization of Spirometry, 1994 Update. American Thoracic Society

Preclinical assessment of wt GNE gene plasmid for management of hereditary inclusion body myopathy 2 (HIBM2)

Hand-held myometry.

Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy.

Sialic acids in human health and disease.

Safety and in vivo expression of a GNE-transgene: a novel treatment approach for hereditary inclusion body myopathy-2.

Hereditary Inclusion Body Myopathy (HIBM2)

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1331-1341

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10.1089/HUM.2010.192

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11

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22

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