Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice (Q24654670)

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English	Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice	scientific article

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11 January 2020

Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice (English)

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11 January 2020

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coagulation factor IX

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Leszek Lisowski

3

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11 January 2020

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Alex H Chang

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11 January 2020

Matthias T Stephan

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11 January 2020

Michel Sadelain

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5 August 2008

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11 January 2020

Molecular Therapy

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11 January 2020

16

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11 January 2020

10

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11 January 2020

1745-1752

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11 January 2020

Isolation and characterization of a cDNA coding for human factor IX

1 reference

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7 April 2017

Vitamin K-dependent carboxylase: mRNA distribution and effects of vitamin K-deficiency and warfarin treatment

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7 April 2017

Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity

1 reference

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28 September 2017

Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors

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28 September 2017

Gene therapy for hemophilia "A" and "B": efficacy, safety and immune consequences

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28 September 2017

Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens

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28 September 2017

The genetic engineering of hematopoietic stem cells: the rise of lentiviral vectors, the conundrum of the ltr, and the promise of lineage-restricted vectors

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

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28 September 2017

Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model

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28 September 2017

Correction of murine hemophilia A by hematopoietic stem cell gene therapy

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28 September 2017

Gene therapy of storage disorders by retroviral and lentiviral vectors

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28 September 2017

Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients?

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28 September 2017

Lentivirus-mediated expression of mutant MGMTP140K protects human CD34+ cells against the combined toxicity of O6-benzylguanine and 1,3-bis(2-chloroethyl)-nitrosourea or temozolomide

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28 September 2017

Two novel mutations in EGF-like domains of human factor IX dramatically impair intracellular processing and secretion

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28 September 2017

In vivo selection of MGMT(P140K) lentivirus–transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning

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28 September 2017

Gene therapy for inborn and acquired immune deficiency disorders

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28 September 2017

Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction.

1 reference

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28 September 2017

Side effects of retroviral gene transfer into hematopoietic stem cells

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28 September 2017

Adult bone marrow stem cells for cell and gene therapies: implications for greater use.

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28 September 2017

Gene transfer as an approach to treating hemophilia

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https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2658893

28 September 2017

Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells

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28 September 2017

Lentiviral vectors for enhanced gene expression in human hematopoietic cells

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28 September 2017

Direct reversal of DNA damage by mutant methyltransferase protein protects mice against dose-intensified chemotherapy and leads to in vivo selection of hematopoietic stem cells

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28 September 2017

Therapeutic haemoglobin synthesis in beta-thalassaemic mice expressing lentivirus-encoded human beta-globin

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Isolation of human O6-alkylguanine-DNA alkyltransferase mutants highly resistant to inactivation by O6-benzylguanine

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28 September 2017

Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A

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28 September 2017

A factor IX-deficient mouse model for hemophilia B gene therapy

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28 September 2017

Gene transfer to hematopoietic stem cells: implications for gene therapy of human disease.

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28 September 2017

Molecular cloning of the gene for human anti-haemophilic factor IX.

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28 September 2017

Myeloablation is not required to select and maintain expression of the drug-resistance gene, mutant MGMT, in primary and secondary recipients

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30 May 2018

Enhanced gene expression conferred by stepwise modification of a nonprimate lentiviral vector

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28 November 2018

Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity

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28 November 2018

Stem cell-derived erythroid cells mediate long-term systemic protein delivery

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28 November 2018

Angiogenesis enhances factor IX delivery and persistence from retrievable human bioengineered muscle implants

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28 November 2018

Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX.

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28 November 2018

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

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28 November 2018

Insertional oncogenesis in gene therapy: how much of a risk?

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28 November 2018

Efficient production of human FVIII in hemophilic mice using lentiviral vectors

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28 November 2018

Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells

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28 November 2018

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

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28 November 2018

Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors

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28 November 2018

Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells

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12 December 2020

based on heuristic

inferred from PubMed ID database lookup

High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors

1 reference

https://pubmed.ncbi.nlm.nih.gov/18682698

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Recipient preparation for bone marrow transplantation. I. Efficacy of total-body irradiation and busulfan

1 reference

https://pubmed.ncbi.nlm.nih.gov/18682698

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1038/MT.2008.161

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Europe PubMed Central

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11 January 2020

1 reference

Europe PubMed Central

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11 January 2020

1 reference

Europe PubMed Central

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11 January 2020

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