Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer
about
Recent advances in the pharmacology of neurological gene therapy.Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients.Enzyme replacement therapy for Fabry disease: some answers but more questionsSex differences of urinary and kidney globotriaosylceramide and lyso-globotriaosylceramide in Fabry mice.AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities.The expanding clinical spectrum of Anderson-Fabry disease: a challenge to diagnosis in the novel era of enzyme replacement therapy.Gene therapy for renal disorders.Gene therapy of liver diseases.Angiogenic and antiangiogenic gene therapy.Gene therapy progress and prospects--vectorology: design and production of expression cassettes in AAV vectors.Novel therapeutic targets for the treatment of Fabry disease.Prediction of response of mutated alpha-galactosidase A to a pharmacological chaperone.Anti-metastatic effects of viral and non-viral mediated Nk4 delivery to tumours.Screening for pharmacological chaperones in Fabry diseaseNeurological features of Fabry disease: clinical, pathophysiological aspects and therapy.Gene therapy for fabry disease: a review of the literature.Promise of adeno-associated virus as a gene therapy vector for cardiovascular diseases.Multicomponent nanoparticles as nonviral vectors for the treatment of Fabry disease by gene therapy.New treatments for denervating diseases.Fabry disease
P2860
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P2860
Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer
description
2003 nî lūn-bûn
@nan
2003 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2003 թվականի մարտին հրատարակված գիտական հոդված
@hy
2003年の論文
@ja
2003年論文
@yue
2003年論文
@zh-hant
2003年論文
@zh-hk
2003年論文
@zh-mo
2003年論文
@zh-tw
2003年论文
@wuu
name
Long-term correction of globot ...... d virus-mediated gene transfer
@ast
Long-term correction of globot ...... d virus-mediated gene transfer
@en
Long-term correction of globot ...... d virus-mediated gene transfer
@nl
type
label
Long-term correction of globot ...... d virus-mediated gene transfer
@ast
Long-term correction of globot ...... d virus-mediated gene transfer
@en
Long-term correction of globot ...... d virus-mediated gene transfer
@nl
prefLabel
Long-term correction of globot ...... d virus-mediated gene transfer
@ast
Long-term correction of globot ...... d virus-mediated gene transfer
@en
Long-term correction of globot ...... d virus-mediated gene transfer
@nl
P2093
P2860
P356
P1476
Long-term correction of globot ...... d virus-mediated gene transfer
@en
P2093
Advait Limaye
Gary J Murray
Jane M Quirk
Jinhee Park
Monique P Gelderman
Pankaj Qasba
Roscoe O Brady
P2860
P304
P356
10.1073/PNAS.0537900100
P407
P577
2003-03-06T00:00:00Z