Lista de obras - Katherine A. High - Dominio Público Uruguay

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

artículo científico publicado en 2015

AAV2 gene therapy readministration in three adults with congenital blindness.

artículo científico publicado en 2012

Adeno-associated viral vectors for the treatment of hemophilia.

artículo científico publicado en 2015

Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice.

artículo científico publicado en 2010

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

artículo científico publicado en 2011

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

artículo científico publicado en 2009

An edible switch for gene therapy.

artículo científico publicado en 2016

Assessing the potential for AAV vector genotoxicity in a murine model

artículo científico publicado en 2010

CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome.

artículo científico publicado en 2013

Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates

artículo científico publicado en 2011

Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo.

artículo científico publicado en 2011

Clinical development of gene therapy: results and lessons from recent successes

artículo científico publicado en 2016

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

artículo científico publicado en 2013

Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans

artículo científico publicado en 2013

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

artículo científico

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

artículo científico publicado en 2013

Enhanced T cell function in a mouse model of human glycosylation.

artículo científico publicado en 2013

Entering the Modern Era of Gene Therapy

artículo científico publicado en 2018

Gene Therapy

artículo científico publicado en 2019

Gene therapy comes of age.

artículo científico publicado en 2018

Gene therapy for haemophilia: a long and winding road.

artículo científico publicado en 2011

Gene therapy for hemophilia: the clot thickens.

artículo científico publicado en 2014

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012

artículo científico publicado en 2013

Gene therapy in haemophilia--going for cure?

scientific article published on May 2010

Gene therapy research at the frontiers of viral immunology.

artículo científico publicado en 2012

Generation of a novel factor IX with augmented clotting activities in vitro and in vivo.

artículo científico publicado en 2010

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

artículo científico publicado en 2017

Hepato-entrained B220+CD11c+NK1.1+ cells regulate pre-metastatic niche formation in the lung.

artículo científico publicado en 2018

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

artículo científico publicado en 2013

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy.

artículo científico publicado en 2017

Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.

artículo científico publicado en 2009

In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity

artículo científico

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

artículo científico publicado en 2015

In vivo genome editing restores haemostasis in a mouse model of haemophilia

artículo científico publicado en 2011

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer ⬇️

artículo científico publicado el 1 de mayo de 2003

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.

artículo científico publicado en 2011

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

artículo científico publicado en 2014

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

artículo científico publicado en 2010

Novel mobility test to assess functional vision in patients with inherited retinal dystrophies

artículo científico publicado en 2017

Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers?

artículo científico publicado en 2019

Overcoming preexisting humoral immunity to AAV using capsid decoys

artículo científico publicado en 2013

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape.

artículo científico publicado en 2017

PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9.

artículo científico publicado en 2011

Patients Beware: Commercialized Stem Cell Treatments on the Web

artículo científico publicado en 2010

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

artículo científico publicado en 2010

Perspectives on best practices for gene therapy programs

artículo científico publicado en 2015

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

artículo científico publicado en 2012

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.

artículo científico publicado en 2010

Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction.

artículo científico publicado en 2009

Robust ZFN-mediated genome editing in adult hemophilic mice.

artículo científico publicado en 2013

Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model

artículo científico publicado en 2012

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

artículo científico publicado en 2016

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

artículo científico publicado en 2010

Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome

artículo científico publicado en 2012

Special lectures in haemophilia management.

artículo científico publicado en 2010

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

artículo científico publicado en 2016

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

artículo científico publicado en 2016

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

artículo científico publicado en 2015

The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene

article

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

artículo científico publicado en 2012

The gene therapy journey for hemophilia: are we there yet?

artículo científico publicado en 2012

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

artículo científico publicado en 2013

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

artículo científico publicado en 2013

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs

artículo científico publicado en 2015

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

artículo científico publicado en 2013

Turning genes into medicines-what have we learned from gene therapy drug development in the past decade?

artículo científico publicado en 2020

Lista de obras de Katherine A. High

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

AAV2 gene therapy readministration in three adults with congenital blindness.

Adeno-associated viral vectors for the treatment of hemophilia.

Adeno-associated virus vectors serotype 2 induce prolonged proliferation of capsid-specific CD8+ T cells in mice.

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

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

An edible switch for gene therapy.

Assessing the potential for AAV vector genotoxicity in a murine model

CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome.

Capsid-specific T-cell responses to natural infections with adeno-associated viruses in humans differ from those of nonhuman primates

Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo.

Clinical development of gene therapy: results and lessons from recent successes

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

Enhanced T cell function in a mouse model of human glycosylation.

Entering the Modern Era of Gene Therapy

Gene Therapy

Gene therapy comes of age.

Gene therapy for haemophilia: a long and winding road.

Gene therapy for hemophilia: the clot thickens.

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012

Gene therapy in haemophilia--going for cure?

Gene therapy research at the frontiers of viral immunology.

Generation of a novel factor IX with augmented clotting activities in vitro and in vivo.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

Hepato-entrained B220+CD11c+NK1.1+ cells regulate pre-metastatic niche formation in the lung.

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Impact of AAV Capsid-Specific T-Cell Responses on Design and Outcome of Clinical Gene Transfer Trials with Recombinant Adeno-Associated Viral Vectors: An Evolving Controversy.

Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B.

In vitro and in vivo studies of IgG-derived Treg epitopes (Tregitopes): a promising new tool for tolerance induction and treatment of autoimmunity

In vivo genome editing of the albumin locus as a platform for protein replacement therapy

In vivo genome editing restores haemostasis in a mouse model of haemophilia

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer ⬇️

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.

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

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

Novel mobility test to assess functional vision in patients with inherited retinal dystrophies

Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers?

Overcoming preexisting humoral immunity to AAV using capsid decoys

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape.

PEG-modulated column chromatography for purification of recombinant adeno-associated virus serotype 9.

Patients Beware: Commercialized Stem Cell Treatments on the Web

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Perspectives on best practices for gene therapy programs

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

Prevention of spontaneous bleeding in dogs with haemophilia A and haemophilia B.

Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction.

Robust ZFN-mediated genome editing in adult hemophilic mice.

Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome

Special lectures in haemophilia management.

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII

The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

The gene therapy journey for hemophilia: are we there yet?

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

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

Turning genes into medicines-what have we learned from gene therapy drug development in the past decade?