TY  - GEN
AB  - Adeno-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.
AD  - Oregon Health and Science University
AU  - Earley, Lauriel
DA  - 2016-04-01
DO  - 10.6083/M4C24VG2
DO  - DOI
ED  - Nakai, Hiroyuki
ED  - Mentor
ID  - 2984
KW  - Leber Congenital Amaurosis
KW  - Hemophilia B
KW  - Genetic Therapy
KW  - Dependovirus
L1  - https://digitalcollections.ohsu.edu/record/2984/files/3773_etd.pdf
L2  - https://digitalcollections.ohsu.edu/record/2984/files/3773_etd.pdf
L4  - https://digitalcollections.ohsu.edu/record/2984/files/3773_etd.pdf
LK  - https://digitalcollections.ohsu.edu/record/2984/files/3773_etd.pdf
N2  - Adeno-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.
PY  - 2016-04-01
T1  - The role of assembly-activating protein in the capsid assembly of different adeno-associated virus serotypes
TI  - The role of assembly-activating protein in the capsid assembly of different adeno-associated virus serotypes
UR  - https://digitalcollections.ohsu.edu/record/2984/files/3773_etd.pdf
Y1  - 2016-04-01
ER  -