(Q28595868)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

author name string

Charles P Venditti

series ordinal

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

Translational Science of Rare Diseases

5 June 2020

language of work or name

English

0 references

publication date

2016

0 references

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

volume

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

issue

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

page(s)

73-89

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

Creative Commons Attribution-NonCommercial 4.0 International

5 June 2020

copyright license

start time

26 August 2016

1 reference

April 2022 Public Data File from Crossref

copyright status

copyrighted

0 references

cites work

A universal system to select gene-modified hepatocytes in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Efficacy of gene therapy for X-linked severe combined immunodeficiency

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Gene therapy for severe combined immunodeficiency: are we there yet?

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Gene transfer in humans using a conditionally replicating lentiviral vector

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Infectious hepatitis C virus pseudo-particles containing functional E1-E2 envelope protein complexes

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

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

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Initial sequencing and analysis of the human genome

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

17 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

The interleukin-2 receptor gamma chain: its role in the multiple cytokine receptor complexes and T cell development in XSCID

17 March 2017

2 references

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

17 March 2017

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

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

23 March 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas

7 April 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

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

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Helper-dependent adenoviral vectors for liver-directed gene therapy.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Gene therapy for leukodystrophies

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Update on gene therapy for adenosine deaminase-deficient severe combined immunodeficiency.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Transient pretreatment with glucocorticoid ablates innate toxicity of systemically delivered adenoviral vectors without reducing efficacy.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Potential of AAV vectors in the treatment of metabolic disease

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

X-linked adrenoleukodystrophy.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Current status of gendicine in China: recombinant human Ad-p53 agent for treatment of cancers

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Helper-dependent adenoviral vectors for gene therapy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Biology of adenovirus and its use as a vector for gene therapy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Helper-dependent adenovirus vectors elicit intact innate but attenuated adaptive host immune responses in vivo

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Biology of adeno-associated virus.

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

The molecular basis of X-linked severe combined immunodeficiency: defective cytokine receptor signaling

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Prospects for the use of adeno-associated virus as a vector for human gene therapy

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Structural Proteins of Adenovirus-Associated Viruses

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Bacterial virus gene expression in human cells

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Development of adenovirus vectors for the expression of heterologous genes

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Characterization of a preferred site on human chromosome 19q for integration of adeno-associated virus DNA by non-homologous recombination

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Interleukin-2 receptor gamma chain mutation results in X-linked severe combined immunodeficiency in humans

29 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

A cascade of adenovirus early functions is required for expression of adeno-associated virus

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Adeno-associated virus DNA replication is induced by genes that are essential for HSV-1 DNA synthesis

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

Isolation of adenovirus type 5 host range deletion mutants defective for transformation of rat embryo cells

3 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5108627

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

3 June 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

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

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Gene therapy for aromatic L-amino acid decarboxylase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Strategies for retrovirus-based correction of severe, combined immunodeficiency (SCID).

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Long-term outcome after hematopoietic stem cell transplantation of a single-center cohort of 90 patients with severe combined immunodeficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Hematopoietic stem cell gene therapy for adenosine deaminase-deficient severe combined immunodeficiency leads to long-term immunological recovery and metabolic correction

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Next-generation adenoviral vectors: new and improved

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates.

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

AAV vector integration sites in mouse hepatocellular carcinoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

CD8(+) T-cell responses to adeno-associated virus capsid in humans

21 January 2018

1 reference

https://api.crossref.org/works/10.3233%2FTRD-160007

Reversal of early neurologic and neuroradiologic manifestations of X-linked adrenoleukodystrophy by bone marrow transplantation

21 January 2018

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27853673

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy

1 reference

PubMed

https://pubmed.ncbi.nlm.nih.gov/27853673

inferred from PubMed ID database lookup

12 December 2020

based on heuristic

Identifiers

DOI

10.3233/TRD-160007

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:27853673%20AND%20SRC:MED&resulttype=core&format=json

5 June 2020

PubMed publication ID

27853673

1 reference