(Q45883238)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

title

Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

0 references

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

Immacolata Brigida

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

Francesca Ferrua

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

Maria Grazia Roncarolo

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

author name string

Barbara Cappelli

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

Robert Chiesa

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

Sarah Marktel

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

publication date

1 January 2009

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

volume

44

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

issue

1-3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

21 January 2020

page(s)

150-159

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19224139%20AND%20SRC:MED&resulttype=core&format=json

Prolonged pancytopenia in a gene therapy patient with ADA-deficient SCID and trisomy 8 mosaicism: a case report

21 January 2020

cites work

1 reference

12 December 2020

Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration

1 reference

12 December 2020

Adenosine deaminase deficiency: clinical expression, molecular basis, and therapy

1 reference

12 December 2020

Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1.

1 reference

12 December 2020

Severe combined immunodeficiency. A model disease for molecular immunology and therapy

1 reference

12 December 2020

Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement

1 reference

12 December 2020

Non-myeloablative stem cell transplantation for the treatment of cancer and life-threatening non-malignant disorders; past accomplishments and future goals

1 reference

12 December 2020

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

1 reference

12 December 2020

Successful peripheral T-lymphocyte-directed gene transfer for a patient with severe combined immune deficiency caused by adenosine deaminase deficiency

1 reference

12 December 2020

Retroviral marking and transplantation of rhesus hematopoietic cells by nonmyeloablative conditioning.

1 reference

12 December 2020

Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: long-term results of the first clinical gene therapy trial.

1 reference

12 December 2020

Retroviral-fibronectin interactions in transduction of mammalian cells

1 reference

12 December 2020

An in vivo model of somatic cell gene therapy for human severe combined immunodeficiency

1 reference

12 December 2020

Gene therapy for immunodeficiency due to adenosine deaminase deficiency.

1 reference

12 December 2020

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

1 reference

12 December 2020

Therapeutic applications for hematopoietic stem cell gene transfer

1 reference

12 December 2020

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

1 reference

12 December 2020

Gene therapy for adenosine deaminase deficiency

1 reference

12 December 2020

Management options for adenosine deaminase deficiency; proceedings of the EBMT satellite workshop (Hamburg, March 2006).

1 reference

12 December 2020

Patients with adenosine deaminase deficiency surviving after hematopoietic stem cell transplantation are at high risk of CNS complications

1 reference

12 December 2020

The impact of low-dose busulfan on clonal dynamics in nonhuman primates

1 reference

12 December 2020

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

1 reference

12 December 2020

Efficient and durable gene marking of hematopoietic progenitor cells in nonhuman primates after nonablative conditioning

1 reference

12 December 2020

Optimisation of retroviral supernatant production conditions for the genetic modification of human CD34+ cells

1 reference

12 December 2020

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects

1 reference

12 December 2020

IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34+ cells while maintaining in vivo lymphoid potential.

1 reference

12 December 2020

Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells

1 reference

12 December 2020

Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates

1 reference

12 December 2020

T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

1 reference

12 December 2020

Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency

1 reference

12 December 2020

Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning

1 reference

12 December 2020

Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

1 reference

12 December 2020

An evaluation of engraftment, toxicity and busulfan concentration in children receiving bone marrow transplantation for leukemia or genetic disease

1 reference

12 December 2020

Progress and prospects: gene therapy clinical trials (part 2).

1 reference

12 December 2020

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

1 reference

12 December 2020

Altered intracellular and extracellular signaling leads to impaired T-cell functions in ADA-SCID patients

1 reference

12 December 2020

Development of lentiviral gene therapy for Wiskott Aldrich syndrome

1 reference

12 December 2020

Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy

1 reference

12 December 2020

Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences

1 reference

12 December 2020

Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation.

1 reference

12 December 2020

Gene therapy for childhood immunological diseases

1 reference

12 December 2020

Gene therapy insertional mutagenesis insights

1 reference

12 December 2020

Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients.

1 reference

12 December 2020

Mutagenesis and oncogenesis by chromosomal insertion of gene transfer vectors.

1 reference

12 December 2020