Adeno-associated vectors and virions for treatment of galactosemia and methods of use and manufacture

Adeno-associated vectors and virions for treatment of galactosemia and methods of use and manufacture

In one embodiment, a recombinant adeno-associated virus (rAAV) vector is provided, the rAAV vector comprising a transgene expressing galactose-1-phosphate uridinetransferase (GALT); a virion (rAAV virion) comprising the vector; methods for their production; methods of use thereof, including methods...

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Bibliographische Detailangaben
Hauptverfasser: HUGHES, MARTIN, RASMUSSEN, SCOTT, A, LEHR, KLAUS, FOSTER KEITH, DAHAL SANJAY, DAENZER, JUDITH, M., I, FRIDWICH-KOHL JONATHAN L
Format: Patent
Sprache:chi ; eng
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BIOCHEMISTRY
CHEMISTRY
COMPOSITIONS THEREOF
CULTURE MEDIA
ENZYMOLOGY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
MICROBIOLOGY
MICROORGANISMS OR ENZYMES
MUTATION OR GENETIC ENGINEERING
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS
SPIRITS
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WINE
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Zusammenfassung:In one embodiment, a recombinant adeno-associated virus (rAAV) vector is provided, the rAAV vector comprising a transgene expressing galactose-1-phosphate uridinetransferase (GALT); a virion (rAAV virion) comprising the vector; methods for their production; methods of use thereof, including methods of treating galactosemia, GALT-deficiency, symptoms thereof; and a kit. 提供了重组腺相关病毒(rAAV)载体，所述rAAV载体包含表达半乳糖-1-磷酸尿苷酰转移酶(GALT)的转基因；包含所述载体的病毒体(rAAV病毒体)；它们的产生方法；它们的使用方法，包括治疗半乳糖血症、GALT-缺乏症、它们的症状的方法；以及试剂盒。