AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model (Q40277560)

19 September 2017

AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model (English)

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19 September 2017

1

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2 June 2020

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Nisha Nair

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2 June 2020

Tony Lahoutte

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2 June 2020

Wim Derave

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2 June 2020

author name string

Inge Everaert

3

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2 June 2020

Wouter Van Overbeke

4

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2 June 2020

Lynn Supply

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2 June 2020

Olivier Zwaenepoel

6

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2 June 2020

Cindy Peleman

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2 June 2020

Jo Van Dorpe

8

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2 June 2020

Nick Devoogdt

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2 June 2020

Marinee K Chuah

12

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2 June 2020

Jan Gettemans

14

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2 June 2020

Thierry VandenDriessche

13

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2 June 2020

publication date

21 February 2017

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19 September 2017

Human Molecular Genetics

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2 June 2020

volume

26

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2 June 2020

issue

7

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2 June 2020

page(s)

1353-1364

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Gelsolin variant (Asn-187) in familial amyloidosis, Finnish type

2 June 2020

cites work

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

The crystal structure of plasma gelsolin: implications for actin severing, capping, and nucleation

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Ca2+ binding by domain 2 plays a critical role in the activation and stabilization of gelsolin

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Structure of the N-terminal half of gelsolin bound to actin: roles in severing, apoptosis and FAF

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Furin initiates gelsolin familial amyloidosis in the Golgi through a defect in Ca(2+) stabilization

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Metalloendoprotease cleavage triggers gelsolin amyloidogenesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Hereditary gelsolin amyloidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Chaperone nanobodies protect gelsolin against MT1-MMP degradation and alleviate amyloid burden in the gelsolin amyloidosis mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

An ER-directed gelsolin nanobody targets the first step in amyloid formation in a gelsolin amyloidosis mouse model

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Systemic delivery of adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Directed evolution of protease beacons that enable sensitive detection of endogenous MT1-MMP activity in tumor cell lines

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Secretion of amyloidogenic gelsolin progressively compromises protein homeostasis leading to the intracellular aggregation of proteins

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Genome-wide computational analysis reveals cardiomyocyte-specific transcriptional Cis-regulatory motifs that enable efficient cardiac gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Adeno-associated virus (AAV) serotype 9 provides global cardiac gene transfer superior to AAV1, AAV6, AAV7, and AAV8 in the mouse and rat.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Analysis of AAV serotypes 1-9 mediated gene expression and tropism in mice after systemic injection

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Directed evolution of adeno-associated virus yields enhanced gene delivery vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Glycan binding avidity determines the systemic fate of adeno-associated virus type 9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

A llama-derived gelsolin single-domain antibody blocks gelsolin-G-actin interaction

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

AMIDE: a free software tool for multimodality medical image analysis

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Effect of beta-alanine and carnosine supplementation on muscle contractility in mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

Gelsolin–derived familial amyloidosis caused by asparagine or tyrosine substitution for aspartic acid at residue 187

21 January 2018

1 reference

https://api.crossref.org/works/10.1093%2FHMG%2FDDX056

21 January 2018

Identifiers

DOI

10.1093/HMG/DDX056

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28334940%20AND%20SRC:MED&resulttype=core&format=json

2 June 2020

1 reference