TY - JOUR
T1 - Advanced genetic engineering to achieve in vivo targeting of adenovirus utilizing camelid single domain antibody
AU - Lee, Myungeun
AU - Lu, Zhi Hong
AU - Shoemaker, Charles B.
AU - Tremblay, Jacqueline M.
AU - St. Croix, Bradley
AU - Seaman, Steven
AU - Gonzalez-Pastor, Rebeca
AU - Kashentseva, Elena A.
AU - Dmitriev, Igor P.
AU - Curiel, David T.
N1 - Publisher Copyright:
© 2021 The Author(s)
PY - 2021/6/10
Y1 - 2021/6/10
N2 - For the developing field of gene therapy the successful address of the basic requirement effective gene delivery has remained a critical barrier. In this regard, the “Holy Grail” vector envisioned by the field's pioneers embodied the ability to achieve efficient and specific in vivo gene delivery. Functional linkage of antibody selectivity with viral vector efficiency represented a logical strategy but has been elusive. Here we have addressed this key issue by developing the technical means to pair antibody-based targeting with adenoviral-mediated gene transfer. Our novel method allows efficient and specific gene delivery. Importantly, our studies validated the achievement of this key vectorology mandate in the context of in vivo gene delivery. Vectors capable of effective in vivo delivery embody the potential to dramatically expand the range of successful gene therapy cures.
AB - For the developing field of gene therapy the successful address of the basic requirement effective gene delivery has remained a critical barrier. In this regard, the “Holy Grail” vector envisioned by the field's pioneers embodied the ability to achieve efficient and specific in vivo gene delivery. Functional linkage of antibody selectivity with viral vector efficiency represented a logical strategy but has been elusive. Here we have addressed this key issue by developing the technical means to pair antibody-based targeting with adenoviral-mediated gene transfer. Our novel method allows efficient and specific gene delivery. Importantly, our studies validated the achievement of this key vectorology mandate in the context of in vivo gene delivery. Vectors capable of effective in vivo delivery embody the potential to dramatically expand the range of successful gene therapy cures.
KW - Adenoviral vectors (Ad)
KW - CD276 [B7-H3]
KW - Camelid single domain antibody (sdAb)
KW - Gene delivery
KW - Human epithelial ovarian cancer cell (SKOV3.ip1)
KW - Ovarian Cancer (OvCa) xenograft mouse model
UR - http://www.scopus.com/inward/record.url?scp=85104649278&partnerID=8YFLogxK
U2 - 10.1016/j.jconrel.2021.04.009
DO - 10.1016/j.jconrel.2021.04.009
M3 - Article
C2 - 33872627
AN - SCOPUS:85104649278
SN - 0168-3659
VL - 334
SP - 106
EP - 113
JO - Journal of Controlled Release
JF - Journal of Controlled Release
ER -