In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy (Q35999976)

11 August 2017

In Vivo Selection Yields AAV-B1 Capsid for Central Nervous System and Muscle Gene Therapy (English)

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11 August 2017

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Sourav R Choudhury

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Zachary Fitzpatrick

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Anne F Harris

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Stacy A Maitland

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Jennifer S Ferreira

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Yuanfan Zhang

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Shan Ma

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Rohit B Sharma

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Heather L Gray-Edwards

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Jacob A Johnson

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Aime K Johnson

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Laura C Alonso

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Claudio Punzo

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Kathryn R Wagner

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Casey A Maguire

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Robert M Kotin

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Douglas R Martin

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Miguel Sena-Esteves

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27 April 2016

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1247-1257

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describes a project that uses

11 August 2017

ImageJ

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11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC5088762/fullTextXML

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Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

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2 October 2017

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2 October 2017

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2 October 2017

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Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

2 October 2017

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2 October 2017

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2 October 2017

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2 October 2017

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2 October 2017

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Sustained normalization of neurological disease after intracranial gene therapy in a feline model

2 October 2017

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2 October 2017

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2 October 2017

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Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS

2 October 2017

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Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome

2 October 2017

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A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.

2 October 2017

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Lessons learned from the clinical development and market authorization of Glybera.

2 October 2017

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Therapeutic response in feline sandhoff disease despite immunity to intracranial gene therapy.

2 October 2017

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A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina

2 October 2017

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Long-term follow-up after gene therapy for canavan disease.

2 October 2017

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Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer

2 October 2017

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Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

2 October 2017

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Structural Insight into the Unique Properties of Adeno-Associated Virus Serotype 9

2 October 2017

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Directed evolution of novel adeno-associated viruses for therapeutic gene delivery.

2 October 2017

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Adenovirus-associated virus vector-mediated gene transfer in hemophilia B

2 October 2017

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Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders

2 October 2017

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The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice

2 October 2017

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Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates

2 October 2017

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2 October 2017

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2 October 2017

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A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease

2 October 2017

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RETRACTED ARTICLE: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

2 October 2017

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2 October 2017

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Vision 1 year after gene therapy for Leber's congenital amaurosis

2 October 2017

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Neural deficits contribute to respiratory insufficiency in Pompe disease

2 October 2017

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Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons

2 October 2017

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2 October 2017

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A myocardium tropic adeno-associated virus (AAV) evolved by DNA shuffling and in vivo selection

2 October 2017

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Efficient gene therapy-based method for the delivery of therapeutics to primate cortex

2 October 2017

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Engineering and selection of shuffled AAV genomes: a new strategy for producing targeted biological nanoparticles

2 October 2017

1 reference

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Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA.

2 October 2017

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Results from a phase I safety trial of hAADC gene therapy for Parkinson disease

2 October 2017

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5088762

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

2 October 2017

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Structure of Adeno-Associated Virus Serotype 8, a Gene Therapy Vector

2 October 2017

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Alpha2,3 and alpha2,6 N-linked sialic acids facilitate efficient binding and transduction by adeno-associated virus types 1 and 6

2 October 2017

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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

2 October 2017

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2 October 2017

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Clades of Adeno-associated viruses are widely disseminated in human tissues.

2 October 2017

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Adeno-associated viruses undergo substantial evolution in primates during natural infections

2 October 2017

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

2 October 2017

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The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

2 October 2017

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Adeno-associated virus serotype 4 (AAV4) and AAV5 both require sialic acid binding for hemagglutination and efficient transduction but differ in sialic acid linkage specificity

2 October 2017

1 reference

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Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach

2 October 2017

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Axonal transport of adeno-associated viral vectors is serotype-dependent

2 October 2017

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Several rAAV vectors efficiently cross the blood-brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system

27 July 2018

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Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

27 July 2018

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Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants.

27 July 2018

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Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I/II trial

23 September 2018

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In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous

23 September 2018

1 reference

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Gene therapy for aromatic L-amino acid decarboxylase deficiency

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5088762

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5088762

Abnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts

23 September 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5088762

A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease

23 September 2018

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

23 September 2018

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Case 15-2009: a man with coma after cardiac arrest

23 September 2018

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PubMed

https://pubmed.ncbi.nlm.nih.gov/27117222

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Engineering and Selection of Shuffled AAV Genomes: A New Strategy for Producing Targeted Biological Nanoparticles

1 reference

Crossref

https://api.crossref.org/works/10.1038%2FMT.2016.84

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/MT.2016.84

1 reference

11 August 2017

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11 August 2017

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