000002984 001__ 2984
000002984 005__ 20250424232807.0
000002984 0247_ $$2DOI$$a10.6083/M4C24VG2
000002984 037__ $$aETD
000002984 245__ $$aThe role of assembly-activating protein in the capsid assembly of different adeno-associated virus serotypes
000002984 269__ $$a2016-04-01
000002984 336__ $$aDissertation
000002984 502__ $$bPh.D.
000002984 520__ $$aAdeno-associated  virus  (AAV)  based  in  vivo  gene  therapy  has  made  great progress in recent years including an approved treatment (Glybera) in Europe and several successful  clinical trials in the United States for hemophilia B and  Leber congenital  amaurosis.  One  drawback  of  the  vector  is  the  large  quantity  of  virus needed  to  induce  therapeutic  levels  of  transgene  expression  and  thus,  production of  the  vector  must  be  as  efficient  as  possible.  Fundamental  aspects  of  AAV’s capsid assembly remain poorly characterized, but the recent discovery of assembly-activating  protein  (AAP)  may  be  the  key  to  finally  understanding  this crucial part of recombinant AAV (rAAV) vector production and AAV biology.
000002984 542__ $$fIn copyright - single owner
000002984 650__ $$aLeber Congenital Amaurosis$$038851
000002984 650__ $$aHemophilia B$$016644
000002984 650__ $$aGenetic Therapy$$028314
000002984 650__ $$aDependovirus$$014224
000002984 691__ $$aSchool of Medicine$$041369
000002984 692__ $$aDepartment of Molecular Microbiology and Immunology$$041429
000002984 7001_ $$aEarley, Lauriel$$uOregon Health and Science University$$041354$$10000-0002-9429-9685
000002984 7201_ $$aNakai, Hiroyuki$$uOregon Health and Science University$$041354$$7Personal$$eMentor
000002984 8564_ $$96aa26b66-9803-4b9b-bd7c-e800b9e7ad1e$$s4709566$$uhttps://digitalcollections.ohsu.edu/record/2984/files/3773_etd.pdf$$ePublic$$22cd9bf14cb866a05b2930f2b1801de18$$31
000002984 905__ $$a/rest/prod/z8/90/rt/43/z890rt430
000002984 909CO $$ooai:digitalcollections.ohsu.edu:2984$$pstudent-work
000002984 980__ $$aTheses and Dissertations