Rational engineering of single-chain polypeptides into protein-only, BBB-targeted nanoparticles


Por: Serna, N, Cespedes, MV, Saccardo, P, Xu, ZK, Unzueta, U, Alamo, P, Pesarrodona, M, Sanchez-Chardi, A, Roldan, M, Mangues, R, Vazquez, E, Villaverde, A, Ferrer-Miralles, N

Publicada: 1 jul 2016
Resumen:
A single chain polypeptide containing the low density lipoprotein receptor (LDLR) ligand Seq-1 with blood-brain barrier (BBB) crossing activity has been successfully modified by conventional genetic engineering to self-assemble into stable protein-only nanoparticles of 30 nm. The nanoparticulate presentation dramatically enhances in vitro, LDLR-dependent cell penetrability compared to the parental monomeric version, but the assembled protein does not show any enhanced brain targeting upon systemic administration. While the presentation of protein drugs in form of nanoparticles is in general advantageous regarding correct biodistribution, this principle might not apply to brain targeting that is hampered by particular bio-physical barriers. Irrespective of this fact, which is highly relevant to the nanomedicine of central nervous system, engineering the cationic character of defined protein stretches is revealed here as a promising and generic approach to promote the controlled oligomerization of biologically active protein species as still functional, regular nanoparticles. (C) 2016 Elsevier Inc. All rights reserved.

Filiaciones:
Serna, N:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain

Cespedes, MV:
 CIBER BBN, Barcelona, Spain

 Hosp Santa Creu & Sant Pau, Biomed Res Inst St Pau IIB St Pau, Oncogenesis & Antitumor Drug Grp, Barcelona, Spain

Saccardo, P:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain

Xu, ZK:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain

Unzueta, U:
 CIBER BBN, Barcelona, Spain

 Hosp Santa Creu & Sant Pau, Biomed Res Inst St Pau IIB St Pau, Oncogenesis & Antitumor Drug Grp, Barcelona, Spain

Alamo, P:
 CIBER BBN, Barcelona, Spain

 Hosp Santa Creu & Sant Pau, Biomed Res Inst St Pau IIB St Pau, Oncogenesis & Antitumor Drug Grp, Barcelona, Spain

Pesarrodona, M:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain

Sanchez-Chardi, A:
 Univ Autonoma Barcelona, Serv Microscopia, E-08193 Barcelona, Spain

Roldan, M:
 Univ Autonoma Barcelona, Serv Microscopia, E-08193 Barcelona, Spain

Mangues, R:
 CIBER BBN, Barcelona, Spain

 Hosp Santa Creu & Sant Pau, Biomed Res Inst St Pau IIB St Pau, Oncogenesis & Antitumor Drug Grp, Barcelona, Spain

Vazquez, E:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain

Villaverde, A:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain

Ferrer-Miralles, N:
 Univ Autonoma Barcelona, Inst Biotecnol & Biomed, E-08193 Barcelona, Spain

 Univ Autonoma Barcelona, Dept Genet & Microbiol, E-08193 Barcelona, Spain

 CIBER BBN, Barcelona, Spain
ISSN: 15499634
Editorial
ELSEVIER, RADARWEG 29, 1043 NX AMSTERDAM, NETHERLANDS, Países Bajos
Tipo de documento: Article
Volumen: 12 Número: 5
Páginas: 1241-1251
WOS Id: 000377409100009
ID de PubMed: 26949165
imagen Green Accepted

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