Systematic multi-trait AAV capsid engineering for efficient gene delivery

Autor: Fatma-Elzahraa Eid, Albert T. Chen, Ken Y. Chan, Qin Huang, Qingxia Zheng, Isabelle G. Tobey, Simon Pacouret, Pamela P. Brauer, Casey Keyes, Megan Powell, Jencilin Johnston, Binhui Zhao, Kasper Lage, Alice F. Tarantal, Yujia A. Chan, Benjamin E. Deverman

Publikováno v: Nature Communications, Vol 15, Iss 1, Pp 1-14 (2024)

Abstract Broadening gene therapy applications requires manufacturable vectors that efficiently transduce target cells in humans and preclinical models. Conventional selections of adeno-associated virus (AAV) capsid libraries are inefficient at search

Externí odkaz: https://doaj.org/article/63fea91c4ddf435fb80d6986bfc4dbae

High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography

Autor: Michael Florea, Fotini Nicolaou, Simon Pacouret, Eric M. Zinn, Julio Sanmiguel, Eva Andres-Mateos, Carmen Unzu, Amy J. Wagers, Luk H. Vandenberghe

Publikováno v: Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 146-159 (2023)

The adeno-associated viral vector (AAV) provides a safe and efficient gene therapy platform with several approved products that have marked therapeutic impact for patients. However, a major bottleneck in the development and commercialization of AAV r

Externí odkaz: https://doaj.org/article/b21e4b6154cf46b4a7fae892eb16cad8

The Assembly-Activating Protein Promotes Stability and Interactions between AAV’s Viral Proteins to Nucleate Capsid Assembly

Autor: Anna C. Maurer, Simon Pacouret, Ana Karla Cepeda Diaz, Jessica Blake, Eva Andres-Mateos, Luk H. Vandenberghe

Publikováno v: Cell Reports, Vol 23, Iss 6, Pp 1817-1830 (2018)

Summary: The adeno-associated virus (AAV) vector is a preferred delivery platform for in vivo gene therapy. Natural and engineered variations of the AAV capsid affect a plurality of phenotypes relevant to gene therapy, including vector production and

Externí odkaz: https://doaj.org/article/d96a300cf0694e8cad6fb2c4ad63a814

A high-efficiency AAV for endothelial cell transduction throughout the central nervous system

Autor: Trevor Krolak, Ken Y. Chan, Luke Kaplan, Qin Huang, Jason Wu, Qingxia Zheng, Velina Kozareva, Thomas Beddow, Isabelle G. Tobey, Simon Pacouret, Albert T. Chen, Yujia A. Chan, Daniel Ryvkin, Chenghua Gu, Benjamin E. Deverman

Publikováno v: Nature Cardiovascular Research. 1:389-400

Endothelial cells have a crucial role in nervous system function, and mounting evidence points to endothelial impairment as a major contributor to a wide range of neurological diseases. However, tools to genetically interrogate these cells

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::71f567fa4f2622650f49ee7209776e84
https://doi.org/10.1038/s44161-022-00046-4

Chemical modification of the adeno-associated virus capsid to improve gene delivery

Autor: Mirja Hommel, Dimitri Alvarez-Dorta, Oumeya Adjali, Aurélien Leray, Eduard Ayuso, Gloria González-Aseguinolaza, Simon Pacouret, Mickaël Guilbaud, Mohammed Bouzelha, Philippe Moullier, Sébastien G. Gouin, Laurence Dubreil, Mathieu Mével, Véronique Blouin, Jean Philippe Combal, David Deniaud, Magalie Penaud-Budloo

Publikováno v: Chemical Science
Chemical Science, The Royal Society of Chemistry, 2020, 11 (4), pp.1122-1131. ⟨10.1039/C9SC04189C⟩
Chemical Science, 2020, 11 (4), pp.1122-1131. ⟨10.1039/C9SC04189C⟩

Gene delivery vectors based on adeno-associated virus (AAV) are highly promising due to several desirable features of this parent virus, including a lack of pathogenicity, efficient infection of dividing and non-dividing cells and sustained maintenan

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::87a9713574e18c368d03437b10a2f8f6
https://pubmed.ncbi.nlm.nih.gov/34084369