J. McIntosh

发表

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

Michael Recht, Pratima Chowdary, Deepak Raj, 2014, The New England journal of medicine.

Investigations of the thrombin generation test for the measurement of factor VIII.

J. McIntosh, 2005 .

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

Pratima Chowdary, Savita Rangarajan, Cecilia Rosales, 2011, The New England journal of medicine.

Codon optimization of human factor VIII cDNAs leads to high-level expression.

A. Thrasher, T. VandenDriessche, M. Chuah, 2011, Blood.

A modified thrombin generation test for the measurement of factor VIII concentrates

C. Lee, T. Barrowcliffe, J. Mcintosh, 2003, Journal of thrombosis and haemostasis : JTH.

Potential limits of AAV‐based gene therapy with the use of new transgenes expressing factor IX fusion proteins

C. Négrier, A. Dane, A. Nathwani, 2018, Haemophilia : the official journal of the World Federation of Hemophilia.

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.

A. Nienhuis, J. Gray, M. Valentine, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

variant administration of rAAV vector encoding a novel human factor VIII Therapeutic levels of FVIII following a single peripheral vein

P. Fagone, A. Nienhuis, O. Christophe, 2014 .

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

P. Fagone, A. Nienhuis, O. Christophe, 2013, Blood.

Early Clinical Trial Results Following Administration of a Low Dose of a Novel Self Complementary Adeno-Associated Viral Vector Encoding Human Factor IX In Two Subjects with Severe Hemophilia B

M. Kay, A. Nienhuis, J. Gray, 2010 .

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector–mediated intrauterine gene transfer in early-gestation fetal macaques

M. Choolani, J. Chan, Manfred Schmidt, 2018, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.

Genetic Vaccines and Therapy

L. Work, A. Nathwani, A. Baker, 2008 .

Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates.

J. Gray, A. Davidoff, E. Tuddenham, 2007, Blood.

Expression of human FIX at therapeutic levels in nonhuman primates following a single peripheral vein infusion of low doses of self complementary AAV vector renews hope for haemophilia B gene therapy

A. Davidoff, A. Nathwani, E. Tuddenham, 2006 .

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver.

Geoffrey Kemball-Cook, Jenny McIntosh, Yunyu Spence, 2006, Blood.

Liver Gene Therapy.

Rose M. Sheridan, A. Nathwani, J. Mcintosh, 2022, Human gene therapy.

Adeno-Associated Mediated Gene Transfer for Hemophilia B:8 Year Follow up and Impact of Removing "Empty Viral Particles" on Safety and Efficacy of Gene Transfer

A. Nienhuis, C. Morton, A. Davidoff, 2018, Blood.

GO-8: Preliminary Results of a Phase I/II Dose Escalation Trial of Gene Therapy for Haemophilia a Using a Novel Human Factor VIII Variant

C. Morton, A. Davidoff, E. Tuddenham, 2018, Blood.

A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B

A. Davidoff, E. Tuddenham, A. Dane, 2018, Blood.

Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration.

S. Wong, T. McKay, C. Coutelle, 2008, Human gene therapy.

Continuous delivery of human type I interferons (α/β) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model

M. Wadhwa, Nalini Singh, R. Benjamin, 2007 .

Recombinant adeno-associated virus-mediated in utero gene transfer gives therapeutic transgene expression in the sheep.

Anna L David, Michael Boyd, Simon Waddington, 2011, Human gene therapy.

Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.

P. Fagone, A. Nienhuis, J. Gray, 2011, Human gene therapy.

Correlation of Monocyte‐Monolayer Assay Results, Number of Erythrocyte‐Bound IgG Molecules, and IgG Subclass Composition in the Study of Red Cell Alloantibodies Other Than D

E. Brojer, B. Żupańska, H. Seyfried, 1990, Vox sanguinis.

evidence of a mutational hotspot nocturnal hemoglobinuria ( AA / PNH ) : a high incidence of multiple The spectrum of PIG-A gene mutations in aplastic anemia / paroxysmal

Rutherford, H. Schrezenmeier, J. Mcintosh, 2002 .

Enhancing transduction of the liver by adeno-associated viral vectors

J. Gray, A. Davidoff, A. Nathwani, 2009, Gene Therapy.

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

M. Choolani, J. Chan, Manfred Schmidt, 2017, Molecular therapy : the journal of the American Society of Gene Therapy.

of FVII at levels that protect against fatal spontaneous hemorrhage AAV-mediated gene transfer in the perinatal period results in expression

J. Gray, C. Morton, A. Davidoff, 2014 .

expression of human FIX in nonhuman primates of self-complementary AAV vector results in stable therapeutic Safe and efficient transduction of the liver after peripheral vein infusion

J. Gray, A. Davidoff, E. Tuddenham, 2013 .

AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

J. Gray, C. Morton, A. Davidoff, 2011, Blood.

Successful attenuation of humoral immunity to viral capsid and transgenic protein following AAV-mediated gene transfer with a non-depleting CD4 antibody and cyclosporine

A. Davidoff, H. Waldmann, S. Cobbold, 2011, Gene Therapy.

Coating of adeno‐associated virus with reactive polymers can ablate virus tropism, enable retargeting and provide resistance to neutralising antisera

K. Ulbrich, V. Šubr, R. Benjamin, 2008, The journal of gene medicine.

The spectrum of PIG-A gene mutations in aplastic anemia/paroxysmal nocturnal hemoglobinuria (AA/PNH): a high incidence of multiple mutations and evidence of a mutational hot spot.

H. Schrezenmeier, Y. Mortazavi, J. Marsh, 2003, Blood.

Purification of recombinant adeno-associated virus type 8 vectors by ion exchange chromatography generates clinical grade vector stock.

J. Gray, A. Davidoff, A. Nathwani, 2004, Journal of virological methods.

Genetic Architecture of Tameness in a Rat Model of Animal Domestication

S. Pääbo, T. Schöneberg, L. Trut, 2009, Genetics.

Continuous delivery of human type I interferons (alpha/beta) has significant activity against acute myeloid leukemia cells in vitro and in a xenograft model.

M. Wadhwa, Nalini Singh, R. Benjamin, 2007, Blood.

Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice.

J. S. Owen, I. Graham, G. Dickson, 2009, Atherosclerosis.

Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8

S. E. Barker, R. Ali, Y. Durán, 2008, Gene Therapy.

The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins

L. Davies, S. Hyde, I. Pringle, 2018, Gene Therapy.

Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease

Rose M. Sheridan, W. Heywood, K. Mills, 2023, Gene Therapy.

Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors.

Mark Basche, Jenny McIntosh, Tiansen Li, 2009, Human molecular genetics.

transduction of murine and nonhuman primate liver efficient liver-specific human factor IX expression cassette enable highly Self-complementary adeno-associated virus vectors containing a novel

J. Gray, E. Tuddenham, K. Mertens, 2013 .

AAV-mediated knockdown of Peripherin-2 in vivo using miRNA-based hairpins

R R Ali, A. Georgiadis, R. Ali, 2010, Gene Therapy.

Liver-directed gene therapy corrects Fabry disease in mice

Rose M. Sheridan, R. Corbau, A. Nathwani, 2019, Molecular Genetics and Metabolism.

The murine lung as a factory to produce secreted intrapulmonary and circulatory proteins

L. Davies, S. Hyde, I. Pringle, 2018, Gene Therapy.

Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques.

C. von Kalle, D. Peebles, A. David, 2011, Molecular therapy : the journal of the American Society of Gene Therapy.

Genetic Vaccines and Therapy

L. Work, A. Nathwani, A. Baker, 2008 .

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

M. Kay, A. Nienhuis, J. Gray, 2014, The New England journal of medicine.

Adeno-associated virus gene therapy prevents progression of kidney disease in genetic models of nephrotic syndrome

Colin J. Chu, G. Mollet, C. Antignac, 2023, Science Translational Medicine.

Luciferin detection after intranasal vector delivery is improved by intranasal rather than intraperitoneal luciferin administration.

S. Wong, T. McKay, C. Coutelle, 2008, Human gene therapy.