Search Results - "Shields, Jordan E"
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Considerations for the use of Cre recombinase for conditional gene deletion in the mouse lens
Published in Human genomics (15-02-2019)“…Despite a number of different transgenes that can mediate DNA deletion in the developing lens, each has unique features that can make a given transgenic line…”
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2
Bioengineered coagulation factor VIII enables long-term correction of murine hemophilia A following liver-directed adeno-associated viral vector delivery
Published in Molecular therapy. Methods & clinical development (01-01-2014)“…Clinical data support the feasibility and safety of adeno-associated viral (AAV) vectors in gene therapy applications. Despite several clinical trials of…”
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3
F5‐Atlanta: A novel mutation in F5 associated with enhanced East Texas splicing and FV‐short production
Published in Journal of thrombosis and haemostasis (01-07-2021)“…Background Elucidating the molecular pathogenesis underlying East Texas bleeding disorder (ET) led to the discovery of alternatively spliced F5 transcripts…”
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4
Microfluidic Transduction Harnesses Mass Transport Principles to Enhance Gene Transfer Efficiency
Published in Molecular therapy (04-10-2017)“…Ex vivo gene therapy using lentiviral vectors (LVs) is a proven approach to treat and potentially cure many hematologic disorders and malignancies but remains…”
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5
Treatment of a solid tumor using engineered drug-resistant immunocompetent cells and cytotoxic chemotherapy
Published in Human gene therapy (01-07-2012)“…Multimodal therapy approaches, such as combining chemotherapy agents with cellular immunotherapy, suffers from potential drug-mediated toxicity to immune…”
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6
Administration of FVIII-Expressing Human Placental Cells to Juvenile Sheep Yields Multi-Organ Engraftment, Therapeutic Plasma FVIII Levels and Alter Immune Signaling Pathways to Evade FVIII Inhibitor Induction
Published in Blood (23-11-2021)“…We have previously reported that normal juvenile sheep that received weekly intravenous (IV) infusions of human (n=3) or an expression/secretion-optimized,…”
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In Utero Transplantation of FVIII-Expressing Human Placental Cells Upregulates Gene Pathways Associated with Immune Tolerance, Altering the Response to Postnatal FVIII Infusion
Published in Blood (23-11-2021)“…We have previously reported that in utero transplantation (IUTx) of sheep fetuses (n=14) with human placental cells (PLC) transduced with a lentiviral vector…”
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Delivery of Fviii-mcoET3 to Previously Untreated Sheep Using Human Placental Cells Enables Durable Elevation of Plasma FVIII Levels and Avoids Inhibitor Formation
Published in Blood (05-11-2020)“…Hemophilia A (HA) is characterized by a decrease in the functional clotting protein factor VIII (FVIII). Current hemophilia treatments consist of either…”
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Administration of Cells Engineered to Secrete Fviii-mcoET3 in Prenatal Sheep Recipients Results in Sustained Curative Fviii Plasma Levels for 3 Years after Birth, without Immune or Toxicity-Related Adverse Events
Published in Blood (05-11-2020)“…The treatment of hemophilia A (HA) patients by prenatal transplantation (PNT) is a feasible, yet underestimated and unexplored clinical approach. The…”
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Preclinical Development and Validation of a Hematopoietic Stem Cell-Based Lentivector Treatment for Hemophilia a
Published in Blood (03-12-2015)“…Hemophilia A is a monogenic bleeding disorder currently treated by lifelong infusions of factor VIII (fVIII). The development of gene-based treatments for…”
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Prenatal Transplantation of Placental Cells Engineered to Express FVIII Leads to Corrective Plasma Levels of FVIII after Birth
Published in Blood (08-12-2017)“…Prenatal transplantation (PNTx) is a clinically viable procedure that has been performed in ~50 human patients for 14 different genetic disorders. When…”
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Factor V Atlanta: A Novel Mutation in the F5 Gene Reveals Potential New Cis-Acting Elements Involved in Regulating FV-Short and TFPI Levels
Published in Blood (08-12-2017)“…Coagulation factor V (FV) is procofactor essential to normal hemostasis. Recently, a point mutation in exon 13 of the F5 gene (FV-East Texas; FV-ET), which…”
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Improving Lentiviral Transduction Efficiency with Microfluidic Systems
Published in Blood (03-12-2015)“…Background: Recent clinical trials have demonstrated the efficacy and safety of gene therapy utilizing HIV-derived lentiviral vectors (LVs) for blood…”
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14
Bioengineered Coagulation Factor VIII Enables Long-Term Correction Of Murine Hemophilia A Following Liver-Directed Adeno-Associated Viral Vector Delivery
Published in Blood (15-11-2013)“…Preclinical and, more recently, clinical data support the feasibility and safety of recombinant adeno-associated viral vectors (rAAV) in gene therapy…”
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