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List of works by Katherine A. High

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

scientific article

AAV2 gene therapy readministration in three adults with congenital blindness.

scientific article

Adeno-associated viral vectors for the treatment of hemophilia.

scientific article published on 27 November 2015

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

scientific article

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

scientific article published on 10 December 2011

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

scientific article

An edible switch for gene therapy.

scientific article published in August 2016

Assessing the potential for AAV vector genotoxicity in a murine model

scientific article

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

scientific article published on 30 September 2013

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

scientific article published on 17 May 2011

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

scientific article published on 16 February 2011

Cell Phones and Landlines: The Impact of Gene Therapy on the Cost and Availability of Treatment for Hemophilia ⬇️

scientific article published on October 1, 2011

Cellular Localization and Characterization of Cytosolic Binding Partners for Gla Domain-containing Proteins PRRG4 and PRRG2 ⬇️

scientific article published on July 19, 2013

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

scientific article

Clinical trial opportunities in hemostasis and thrombosis: NHBLI State‐of‐the‐science symposium ⬇️

scientific article published on November 25, 2011

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies

scientific article published on 16 September 2013

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

scientific article published on April 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.

scientific article published on 13 July 2017

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

scientific article published on 16 January 2013

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

scientific article published on 24 May 2013

Entering the Modern Era of Gene Therapy

scientific article published on 26 November 2018

Gene Therapy

scientific article published on 01 August 2019

Gene therapy comes of age.

scientific article published in January 2018

Gene therapy for haemophilia: a long and winding road.

scientific article published on July 2011

Gene therapy for hemophilia: the clot thickens.

scientific article published on November 2014

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

scientific article (publication date: April 2013)

Gene therapy in haemophilia--going for cure?

scientific article published on May 2010

Gene therapy research at the frontiers of viral immunology.

scientific article published on 24 May 2012

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

scientific article published on 21 May 2010

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

scientific article published in December 2017

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

scientific article published in July 2018

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

scientific article published on 17 April 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.

scientific article

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

scientific article

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

scientific article published on 02 September 2012

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

scientific article

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

scientific article (publication date: 26 June 2011)

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

scientific article published on May 1, 2003

Lack of Germline Transmission of Vector Sequences Following Systemic Administration of Recombinant AAV-2 Vector in Males ⬇️

scientific article published on December 1, 2001

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.

scientific article

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

scientific article

Manufacturing and regulatory strategies for clinical AAV2-hRPE65.

scientific article published on October 2010

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

scientific article

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

scientific article published on 28 February 2019

Overcoming preexisting humoral immunity to AAV using capsid decoys

scientific article published on July 2013

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

scientific article published on September 2017

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

scientific article published on 3 February 2011

Patients Beware: Commercialized Stem Cell Treatments on the Web

scientific article published on 17 June 2010

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

scientific article

Perspectives on best practices for gene therapy programs

scientific article

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

scientific article

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue ⬇️

scientific article published on July 12, 2012

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

scientific article

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

scientific article published on 10 November 2009

Robust ZFN-mediated genome editing in adult hemophilic mice.

scientific article published on October 2013

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

scientific article

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.

scientific article published on 30 June 2016

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

scientific article

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

scientific article

Special lectures in haemophilia management.

scientific article published on July 2010

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

scientific article

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

scientific article published on 29 March 2016

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

scientific article published on 23 December 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.

scientific article

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

scientific article published on 01 January 2012

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges ⬇️

scientific article published on May 1, 2011

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

scientific article published on 20 February 2013

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

scientific article published on 6 March 2013

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

scientific article

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

scientific article

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

scientific article published on 16 November 2020

Zinc fingers hit off target ⬇️

scientific article published on October 11, 2011

rAAV Human Trial Experience ⬇️

scientific article published on January 1, 2011

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