Biomedical Engineering Reference
In-Depth Information
viruses. J Virol 82(12):5887-5911. doi:
10.1128/
32. Allocca M, Doria M, Petrillo M, Colella P,
Garcia-Hoyos M, Gibbs D, Kim SR, Maguire
A, Rex TS, Di Vicino U, Cutillo L, Sparrow
JR, Williams DS, Bennett J, Auricchio A
(2008) Serotype-dependent packaging of
large genes in adeno-associated viral vectors
results in effective gene delivery in mice. J Clin
Invest 118(5):1955-1964. doi:
10.1172/
33. Wu Z, Yang H, Colosi P (2010) Effect of
genome size on AAV vector packaging. Mol
Ther 18(1):80-86. doi:
10.1038/mt.2009.255
34. Dong B, Nakai H, Xiao W (2010)
Characterization of genome integrity for over-
sized recombinant AAV vector. Mol Ther
18(1):87-92. doi:
10.1038/mt.2009.258
35. Lai Y, Yue Y, Duan D (2010) Evidence for the
failure of adeno-associated virus serotype 5 to
package a viral genome > or =8.2 kb. Mol Ther
18(1):75-79. doi:
10.1038/mt.2009.256
36. Grose WE, Clark KR, Griffin D, Malik V,
Shontz KM, Montgomery CL, Lewis S, Brown
RH Jr, Janssen PM, Mendell JR, Rodino-
Klapac LR (2012) Homologous recombina-
tion mediates functional recovery of dysferlin
deficiency following AAV5 gene transfer. PLoS
One 7(6):e39233. doi:
10.1371/journal.pone.
37. Nathwani AC, Gray JT, Ng CY, Zhou J,
Spence Y, Waddington SN, Tuddenham EG,
Kemball-Cook G, McIntosh J, Boon-Spijker
M, Mertens K, Davidoff AM (2006) Self-
complementary adeno-associated virus vectors
containing a novel liver-specific human factor
IX expression cassette enable highly efficient
transduction of murine and nonhuman pri-
mate liver. Blood 107(7):2653-2661.
38. Mattar CN, Nathwani AC, Waddington SN,
Dighe N, Kaeppel C, Nowrouzi A, McIntosh
J, Johana NB, Ogden B, Fisk NM, Davidoff
AM, David A, Peebles D, Valentine MB,
Appelt JU, von Kalle C, Schmidt M, Biswas A,
Choolani M, Chan JK (2011) Stable human
FIX expression after 0.9G intrauterine
gene transfer of self-complementary adeno-
associated viral vector 5 and 8 in macaques.
Mol Ther 19(11):1950-1960. doi:
10.1038/
39. Li H, Haurigot V, Doyon Y, Li T, Wong SY,
Bhagwat AS, Malani N, Anguela XM, Sharma R,
Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou
S, Paschon DE, Rebar EJ, Bushman FD, Gregory
PD, Holmes MC, High KA (2011) In vivo
genome editing restores haemostasis in a mouse
model of haemophilia. Nature 475(7355):217-
221. doi:
10.1038/nature10177
40. Wang Z, Lisowski L, Finegold MJ, Nakai H,
Kay MA, Grompe M (2012) AAV vectors con-
taining rDNA homology display increased
chromosomal integration and transgene per-
sistence. Mol Ther 20(10):1902-1911.
doi:
10.1038/mt.2012.157
41. Argyros O, Wong SP, Fedonidis C, Tolmachov
O, Waddington SN, Howe SJ, Niceta M,
Coutelle C, Harbottle RP (2011) Development
of S/MAR minicircles for enhanced and per-
sistent transgene expression in the mouse liver.
J Mol Med (Berl) 89(5):515-529.
42. Miller JC, Tan S, Qiao G, Barlow KA, Wang J,
Xia DF, Meng X, Paschon DE, Leung E,
Hinkley SJ, Dulay GP, Hua KL, Ankoudinova
I, Cost GJ, Urnov FD, Zhang HS, Holmes
MC, Zhang L, Gregory PD, Rebar EJ (2011)
A TALE nuclease architecture for efficient
genome editing. Nat Biotechnol 29(2):
143-148. doi:
10.1038/nbt.1755
43. Lock M, Alvira M, Vandenberghe LH,
Samanta A, Toelen J, Debyser Z, Wilson JM
(2010) Rapid, simple, and versatile manufac-
turing of recombinant adeno-associated viral
vectors at scale. Hum Gene Ther 21(10):
1259-1271. doi:
10.1089/hum.2010.055
44. Zolotukhin S, Byrne BJ, Mason E, Zolotukhin I,
Potter M, Chesnut K, Summerford C, Samulski
RJ, Muzyczka N (1999) Recombinant adeno-
associated virus purification using novel methods
improves infectious titer and yield. Gene Ther
6(6):973-985. doi:10.1038/sj.gt.3300938
Search WWH ::
Custom Search