Surface immobilization of hexa-histidine-tagged adeno-associated viral vectors for localized gene delivery

被引：19

作者：

Jang, J-H ^{[1
,2
]}

Koerber, J. T. ^{[1
]}

Gujraty, K. ^{[3
]}

Bethi, S. R. ^{[3
]}

Kane, R. S. ^{[3
]}

Schaffer, D. V. ^{[1
,4
]}

机构：

[1] Univ Calif Berkeley, Dept Chem Engn, Helen Wills Neurosci Inst, Berkeley, CA 94720 USA

[2] Yonsei Univ, Dept Chem & Biomol Engn, Seoul 120749, South Korea

[3] Rensselaer Polytech Inst, Dept Chem & Biol Engn, Troy, NY USA

[4] Univ Calif Berkeley, Helen Wills Neurosci Inst, Dept Bioengn, Berkeley, CA 94720 USA

来源：

GENE THERAPY | 2010年 / 17卷 / 11期

关键词：

AAV; localized gene delivery; substrate-mediated gene delivery; hexa-histidine; LEBERS CONGENITAL AMAUROSIS; DIRECTED EVOLUTION; VIRUS TYPE-2; THERAPY; PURIFICATION; EXPRESSION; ADENOVIRUS; SCAFFOLDS; ANTIBODY;

D O I：

10.1038/gt.2010.81

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

070307 [化学生物学]; 071010 [生物化学与分子生物学];

摘要：

Adeno-associated viral (AAV) vectors, which are undergoing broad exploration in clinical trials, have significant promise for therapeutic gene delivery because of their safety and delivery efficiency. Gene delivery technologies capable of mediating localized gene expression may further enhance the potential of AAV in a variety of therapeutic applications by reducing spread outside a target region, which may thereby reduce off-target side effects. We have genetically engineered an AAV variant capable of binding to surfaces with high affinity through a hexa-histidine metal-binding interaction. This immobilized AAV vector system mediates high-efficiency delivery to cells that contact the surface and thus may have promise for localized gene delivery, which may aid numerous applications of AAV delivery to gene therapy. Gene Therapy (2010) 17, 1384-1389; doi:10.1038/gt.2010.81; published online 27 May 2010

引用

页码：1384 / 1389

页数：6

共 23 条

[1]

Metabolic biotinylation provides a unique platform for the purification and targeting of multiple AAV vector serotypes [J].

Arnold, Gregory S. ;

Sasser, A. Kate ;

Stachler, Matthew D. ;

Bartlett, Jeffrey S. .

MOLECULAR THERAPY, 2006, 14 (01) :97-106

[2]

Effect of gene therapy on visual function in Leber's congenital amaurosis [J].

Bainbridge, James W. B. ;

Smith, Alexander J. ;

Barker, Susie S. ;

Robbie, Scott ;

Henderson, Robert ;

Balaggan, Kamaljit ;

Viswanathan, Ananth ;

Holder, Graham E. ;

Stockman, Andrew ;

Tyler, Nick ;

Petersen-Jones, Simon ;

Bhattacharya, Shomi S. ;

Thrasher, Adrian J. ;

Fitzke, Fred W. ;

Carter, Barrie J. ;

Rubin, Gary S. ;

Moore, Anthony T. ;

Ali, Robin R. .

NEW ENGLAND JOURNAL OF MEDICINE, 2008, 358 (21) :2231-2239

[3]

Targeted adenoviral vectors [J].

Barnett, BG ;

Crews, CJ ;

Douglas, JT .

BIOCHIMICA ET BIOPHYSICA ACTA-GENE STRUCTURE AND EXPRESSION, 2002, 1575 (1-3) :1-14

[4]

Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus [J].

Duan, DS ;

Yue, YP ;

Yan, ZY ;

Yang, JS ;

Engelhardt, JF .

JOURNAL OF CLINICAL INVESTIGATION, 2000, 105 (11) :1573-1587

[5]

Bisphosphonate-mediated gene vector delivery from the metal surfaces ot stents [J].

Fishbein, I ;

Alferiev, IS ;

Nyanguile, O ;

Gaster, R ;

Vohs, JM ;

Wong, GS ;

Felderman, H ;

Chen, IW ;

Choi, H ;

Wilensky, RL ;

Levy, RJ .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 2006, 103 (01) :159-164

[6]

Recombinant adeno-associated virus for muscle directed gene therapy [J].

Fisher, KJ ;

Jooss, K ;

Alston, J ;

Yang, YP ;

Haecker, SE ;

High, K ;

Pathak, R ;

Raper, SE ;

Wilson, JM .

NATURE MEDICINE, 1997, 3 (03) :306-312

[7]

Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus [J].

Flannery, JG ;

Zolotukhin, S ;

Vaquero, MI ;

LaVail, MM ;

Muzyczka, N ;

Hauswirth, WW .

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1997, 94 (13) :6916-6921

[8]

Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2 [J].

Girod, A ;

Ried, M ;

Wobus, C ;

Lahm, H ;

Leike, K ;

Kleinschmidt, J ;

Deléage, G ;

Hallek, M .

NATURE MEDICINE, 1999, 5 (09) :1052-1056

[9]

Localized viral vector delivery to enhance in situ regenerative gene therapy [J].

Hu, W-W ;

Wang, Z. ;

Hollister, S. J. ;

Krebsbach, P. H. .

GENE THERAPY, 2007, 14 (11) :891-901

[10]

Surface adsorption of DNA to tissue engineering scaffolds for efficient gene delivery [J].

Jang, JH ;

Bengali, Z ;

Houchin, TL ;

Shea, LD .

JOURNAL OF BIOMEDICAL MATERIALS RESEARCH PART A, 2006, 77A (01) :50-58

← 1 2 3 →