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Interactions between Nitric Oxide and Indoleamine 2,3-Dioxygenase

Biochemistry (Easton), 2006-07, Vol.45 (28), p.8527-8538

2006

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Obstacles and future of gene therapy for hemophilia

Expert opinion on orphan drugs, 2015-01, Vol.3 (9), p.997-1010

2015

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Transfusion-Associated Reversible Cerebral Vasoconstriction Syndrome in Pediatrics: A Case Report and Review of Literature

Blood, 2023-11, Vol.142 (Supplement 1), p.5562-5562

2023

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Gene therapy for immune tolerance induction in hemophilia with inhibitors

Journal of thrombosis and haemostasis, 2016-06, Vol.14 (6), p.1121-1134

2016

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Evolution Directed Gene Engineering (EDGE) Identifies Single Amino Acid Substitutions in FVIII with Six-Fold Increased in Vitro and In Vivo Activity

Blood, 2023-11, Vol.142 (Supplement 1), p.21-21

2023

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rAAV8-PC Gene Therapy for Severe Protein C Deficiency Using a Novel Murine Model

Blood, 2023-11, Vol.142 (Supplement 1), p.7119-7119

2023

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Factor IX Padua: From Biochemistry to Gene Therapy

Blood, 2016-12, Vol.128 (22), p.SCI-9-SCI-9

2016

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Factor VIII Mimetic Rescue of Hemophilia B Causing Factor IX Variants

Blood, 2022-11, Vol.140 (Supplement 1), p.5570-5571

2022

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Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia Α Dog after AAV Gene Therapy

Blood, 2022-11, Vol.140 (Supplement 1), p.4919-4920

2022

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Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inbibitors in Privately Owned Dogs

Blood, 2022-11, Vol.140 (Supplement 1), p.7780-7781

2022

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Protein Translocation by Bacterial Toxin Channels: A Comparison of Diphtheria Toxin and Colicin Ia

Biophysical journal, 2006-11, Vol.91 (9), p.3249-3256

2006

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Ontogeny of the Alloimmune Anti-Canine Factor VIII Inhibitor Response in Severe Hemophilia Α Dogs

Blood, 2021-11, Vol.138 (Supplement 1), p.3173-3173

2021

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Leveraging CAR T cells to Achieve Desensitization and Enable Transplantation

The Journal of immunology (1950), 2022-05, Vol.208 (1_Supplement), p.175-175.23

2022

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Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Blood, 2023-11, Vol.142 (Supplement 1), p.3628-3628

2023

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Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

Blood, 2021-11, Vol.138 (Supplement 1), p.3975-3975

2021

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The Role of Transgene Levels in Immune Tolerance Induction after AAV Gene Therapy in Hemophilia B Canine Models

Blood, 2017-12, Vol.130, p.2057-2057

2017

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New Hyperactive Factor IX Variants Bioengineered Based upon Biochemical Studies of Factor IX Padua

Blood, 2016-12, Vol.128 (22), p.1384-1384

2016

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Regulation of Factor VIII By Activated Protein C: Assessment In Vitro and In Vivo

Blood, 2017-12, Vol.130, p.3650-3650

2017

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Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

Blood, 2017-12, Vol.130, p.2056-2056

2017

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Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

Blood, 2017-12, Vol.130, p.604-604

2017

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AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life

Molecular therapy. Methods & clinical development, 2024-03, Vol.32 (1), p.101205-101205, Article 101205

2024

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Adeno-Associated Viral
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Biophysics
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Cardiovascular System & Cardiology
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Cd4-Positive T-Lymphocytes - Cytology
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Channels, Receptors, And Electrical Signaling
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Colicins - Chemistry
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Companion Animals
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Diphtheria Toxin - Chemistry
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Dogs
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Efficacy
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Molecular Therapy. Methods & Clinical Development
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Interactions between Nitric Oxide and Indoleamine 2,3-Dioxygenase

Obstacles and future of gene therapy for hemophilia

Transfusion-Associated Reversible Cerebral Vasoconstriction Syndrome in Pediatrics: A Case Report and Review of Literature

Gene therapy for immune tolerance induction in hemophilia with inhibitors

Evolution Directed Gene Engineering (EDGE) Identifies Single Amino Acid Substitutions in FVIII with Six-Fold Increased in Vitro and In Vivo Activity

rAAV8-PC Gene Therapy for Severe Protein C Deficiency Using a Novel Murine Model

Factor IX Padua: From Biochemistry to Gene Therapy

Factor VIII Mimetic Rescue of Hemophilia B Causing Factor IX Variants

Vector Analysis of Multicentric Lymphoma in a Severe Hemophilia Α Dog after AAV Gene Therapy

Long-Term Real-World Safety and Efficacy of Liver-Directed AAV Gene Therapy for Severe Hemophilia with and without Inbibitors in Privately Owned Dogs

Protein Translocation by Bacterial Toxin Channels: A Comparison of Diphtheria Toxin and Colicin Ia

Ontogeny of the Alloimmune Anti-Canine Factor VIII Inhibitor Response in Severe Hemophilia Α Dogs

Leveraging CAR T cells to Achieve Desensitization and Enable Transplantation

Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec

Follow-up of More Than 5 Years in a Cohort of Patients with Hemophilia B Treated with Fidanacogene Elaparvovec Adeno-Associated Virus Gene Therapy

The Role of Transgene Levels in Immune Tolerance Induction after AAV Gene Therapy in Hemophilia B Canine Models

New Hyperactive Factor IX Variants Bioengineered Based upon Biochemical Studies of Factor IX Padua

Regulation of Factor VIII By Activated Protein C: Assessment In Vitro and In Vivo

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Factor IX with Minimal Immune Response

A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence

Spk-8011: Preliminary Results from a Phase 1/2 Dose Escalation Trial of an Investigational AAV-Mediated Gene Therapy for Hemophilia a

AAV gene therapy in companion dogs with severe hemophilia: Real-world long-term data on immunogenicity, efficacy, and quality of life

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