Recent gene therapy advancements for neurological diseases. (Q38085469)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

title

Recent gene therapy advancements for neurological diseases (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

0 references

Sahana Nagabhushan Kalburgi

series ordinal

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

Nadia N Khan

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

Steven J Gray

series ordinal

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

publication date

1 February 2013

1 reference

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24 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

volume

15

1 reference

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24 February 2020

issue

81

1 reference

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24 February 2020

page(s)

111-119

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

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

24 February 2020

cites work

1 reference

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Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease

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6 September 2017

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6 September 2017

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6 September 2017

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Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.

6 September 2017

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Effect of genome size on AAV vector packaging

6 September 2017

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Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

6 September 2017

1 reference

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

6 September 2017

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6 September 2017

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Phase Ib trial of mutant herpes simplex virus G207 inoculated pre-and post-tumor resection for recurrent GBM

6 September 2017

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Insertional gene activation by lentiviral and gammaretroviral vectors.

6 September 2017

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6 September 2017

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Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy.

6 September 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.

6 September 2017

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New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy

6 September 2017

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Use of nonintegrating lentiviral vectors for gene therapy

6 September 2017

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Targeted transgene expression in muller glia of normal and diseased retinas using lentiviral vectors

6 September 2017

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A meta-analysis of unrelated donor umbilical cord blood transplantation versus unrelated donor bone marrow transplantation in adult and pediatric patients

6 September 2017

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6 September 2017

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6 September 2017

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Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo

6 September 2017

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6 September 2017

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6 September 2017

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6 September 2017

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6 September 2017

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

6 September 2017

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Lentivirus-mediated gene transfer to the central nervous system: therapeutic and research applications

6 September 2017

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Lentiviral vectors for use in the central nervous system.

6 September 2017

1 reference

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New recombinant serotypes of AAV vectors.

6 September 2017

1 reference

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Adeno-associated virus: from defective virus to effective vector

6 September 2017

1 reference

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Integration of adeno-associated virus (AAV) and recombinant AAV vectors

6 September 2017

1 reference

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Human CD34+ cells differentiate into microglia and express recombinant therapeutic protein

6 September 2017

1 reference

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Oncolytic herpes viruses as a potential mechanism for cancer therapy.

6 September 2017

1 reference

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ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES

6 September 2017

1 reference

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Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

6 September 2017

1 reference

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Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

6 September 2017

1 reference

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Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

6 September 2017

1 reference

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Global CNS gene transfer for a childhood neurogenetic enzyme deficiency: Canavan disease

6 September 2017

1 reference

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Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

6 September 2017

1 reference

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Retrograde axonal transport of herpes simplex virus: evidence for a single mechanism and a role for tegument

6 September 2017

1 reference

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6 September 2017

1 reference

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Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery

6 September 2017

1 reference

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Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue

6 September 2017

1 reference

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Development of a self-inactivating lentivirus vector.

6 September 2017

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New insight on the role of extrachromosomal retroviral DNA

6 September 2017

1 reference

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The function of herpes simplex virus genes: a primer for genetic engineering of novel vectors

6 September 2017

1 reference

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Prolonged gene expression and cell survival after infection by a herpes simplex virus mutant defective in the immediate-early genes encoding ICP4, ICP27, and ICP22.

6 September 2017

1 reference

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A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase

6 September 2017

1 reference

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Rapid evolution of a protein in vitro by DNA shuffling

6 September 2017

1 reference

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DNA shuffling by random fragmentation and reassembly: in vitro recombination for molecular evolution

6 September 2017

1 reference

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Attenuated multi–mutated herpes simplex virus–1 for the treatment of malignant gliomas

6 September 2017

1 reference

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Lysosomal storage diseases

6 September 2017

1 reference

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Cell surface receptors for herpes simplex virus are heparan sulfate proteoglycans

6 September 2017

1 reference

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Persistence of high-capacity adenoviral vectors as replication-defective monomeric genomes in vitro and in murine liver

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Stable gene transfer to muscle using non-integrating lentiviral vectors

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Efficient transduction of neurons using Ross River glycoprotein-pseudotyped lentiviral vectors.

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Circularization of the herpes simplex virus type 1 genome upon lytic infection

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

A combination of mutations enhances the neurotropism of AAV-2.

14 September 2017

1 reference

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Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus

14 September 2017

1 reference

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Standardized and simplified nomenclature for proteins common to all retroviruses

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Production and neurotropism of lentivirus vectors pseudotyped with lyssavirus envelope glycoproteins.

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Gene therapy for neurologic disease

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

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

28 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Gene therapy of x-linked adrenoleukodystrophy using hematopoietic stem cells and a lentiviral vector

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Quantitative analysis of the packaging capacity of recombinant adeno-associated virus

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Demonstration of circularization of herpes simplex virus DNA following infection using pulsed field gel electrophoresis

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Immune responses to AAV in a phase I study for Canavan disease

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

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

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Outcome of 27 patients with Hurler's syndrome transplanted from either related or unrelated haematopoietic stem cell sources

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Functional amelioration of murine galactosialidosis by genetically modified bone marrow hematopoietic progenitor cells

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Toxicity of a first-generation adenoviral vector in rhesus macaques

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Targeting gene-modified hematopoietic cells to the central nervous system: use of green fluorescent protein uncovers microglial engraftment.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

Evaluation of genetically engineered herpes simplex viruses as oncolytic agents for human malignant brain tumors.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5554939

[Gene therapy of adrenoleukodystrophy]

31 August 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23449113

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

The controversial and changing role of haematopoietic cell transplantation for lysosomal storage disorders: an update

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23449113

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Gene therapy for lysosomal storage disorders

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/23449113

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:23449113%20AND%20SRC:MED&resulttype=core&format=json

24 February 2020

PubMed publication ID

23449113

1 reference