Wordt geladen...
A pilot study of neonatal GALT gene replacement using AAV9 dramatically lowers galactose metabolites in blood, liver, and brain and minimizes cataracts in GALT-null rat pups
Classic galactosemia (CG) is a rare metabolic disorder that results from profound deficiency of galactose-1-P uridylyltransferase (GALT). Despite early detection by newborn screening and rapid and lifelong dietary restriction of galactose, which is the current standard of care, most patients grow to...
Bewaard in:
| Gepubliceerd in: | J Inherit Metab Dis |
|---|---|
| Hoofdauteurs: | , , |
| Formaat: | Artigo |
| Taal: | Inglês |
| Gepubliceerd in: |
2020
|
| Onderwerpen: | |
| Online toegang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7855732/ https://ncbi.nlm.nih.gov/pubmed/32882063 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jimd.12311 |
| Tags: |
Voeg label toe
Geen labels, Wees de eerste die dit record labelt!
|