Katherine A. High

Spark Therapeutics (United States) — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

91h-index39.0kcitations378works13.02yr avg
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Research Topics

Virus-based gene therapy research(242), CAR-T cell therapy research(118), Hemophilia Treatment and Research(106), CRISPR and Genetic Engineering(86), Viral Infectious Diseases and Gene Expression in Insects(71)

Publications378 total

Roadmap for direct and indirect translation of optogenetics into discoveries and therapies for humans
Nature Neuroscience·2025· 1 citedOpen Access
Gene therapy for hemophilia – From basic science to first approvals of “one-and-done” therapies
Molecular Therapy·2025· 16 cited
Validation of a quantitative cell-based relative potency assay for LUXTURNA
Molecular Therapy — Methods & Clinical Development·2025Open Access
Hemophilia B and gene therapy: a new chapter with etranacogene dezaparvovec
Blood Advances·2024· 35 citedOpen Access
Gene and cell therapy in 2023: Rich pipeline, slimming resources?
Molecular Therapy·2023Open Access
THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.
PubMed·2023Open Access
Trial by “Firsts”: Clinical Trial Design and Regulatory Considerations in the Development and Approval of the First AAV Gene Therapy Product in the United States
Cold Spring Harbor Perspectives in Medicine·2022· 11 citedOpen Access
Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A
Molecular Therapy — Methods & Clinical Development·2021· 15 citedOpen Access
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
New England Journal of Medicine·2021· 235 citedOpen Access
89 Five-year update for the Phase III voretigene neparvovec study in biallelic RPE65 mutation–associated inherited retinal disease
Abstracts·2021· 3 citedOpen Access
Structural Integrity of the -Carboxyglutamic Acid Domain of Human Blood Coagulation Factor IXa Is Required for Its Binding to Cofactor VIIIa
UNC Libraries·2021· 1 citedOpen Access
Molecular defect (Gla+ 14----Lys) and its functional consequences in a hereditary factor X deficiency (factor X" Vorarlberg").
UNC Libraries·2021Open Access
Five-Year Post-Injection Results of the Phase 3 Trial of Voretigene Neparvovec-rzyl in Biallelic RPE65 Mutation-Associated Inherited Retinal Disease
Investigative Ophthalmology & Visual Science·2021· 3 citedOpen Access
Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease
Ophthalmology·2021· 176 cited
Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua
Journal of Thrombosis and Haemostasis·2021· 31 citedOpen Access
Turning genes into medicines—what have we learned from gene therapy drug development in the past decade?
Nature Communications·2020· 29 citedOpen Access
ARFI Ultrasound Monitoring of Hemorrhage and Hemostasis In Vivo in Canine Von Willebrand Disease and Hemophilia
UNC Libraries·2020Open Access
A NlalV polymorphism within the human Factor X gene
UNC Libraries·2020Open Access
Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs
UNC Libraries·2020Open Access
Protein Replacement Therapy and Gene Transfer in Canine Models of Hemophilia A, Hemophilia B, von Willebrand Disease, and Factor VII Deficiency
UNC Libraries·2020· 6 citedOpen Access
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Frequent Co-authors

Federico Mingozzi(25), J. Fraser Wright(14), Valder R. Arruda(12), Albert M. Maguire(9), Roland W. Herzog(9), Francesca Simonelli(8), Kathleen Marshall(8), Jean Bennett(8), Xavier M. Anguela(8), Margaret V. Ragni(7), Eric A. Pierce(6), Jeannette Bennicelli(6), Junwei Sun(6), Alberto Auricchio(6), Jennifer Wellman(6), Daniel C. Chung(6), Daniel J. Hui(6), Shangzhen Zhou(6), Xiaosong Zhu(5), Catherine S. Manno(5)