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Hemophilia A Gene Therapy — Some Answers, More Questions

The New England journal of medicine, 2023-02, Vol.388 (8), p.761-763

George, Lindsey A.

2023

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Adeno-Associated Virus Gene Therapy for Hemophilia

Annual review of medicine, 2023-01, Vol.74 (1), p.231-247

2023

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Hemophilia gene therapy: ushering in a new treatment paradigm?

Hematology, 2021-12, Vol.2021 (1), p.226-233

George, Lindsey A

2021

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No CpGs for AAVs?

Blood, 2021-02, Vol.137 (6), p.721-723

George, Lindsey A.

2021

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Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Molecular therapy, 2020-09, Vol.28 (9), p.2073-2082

2020

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Current Clinical Applications of In Vivo Gene Therapy with AAVs

Molecular therapy, 2021-02, Vol.29 (2), p.464-488

2021

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Hemophilia gene therapy comes of age

Blood advances, 2017-12, Vol.1 (26), p.2591-2599

George, Lindsey A.

2017

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Hemophilia gene therapy comes of age

Hematology, 2017-12, Vol.2017 (1), p.587-594

George, Lindsey A

2017

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Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice

Nature communications, 2024-08, Vol.15 (1), p.7193-14

2024

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Factor IX Padua for haemophilia B gene addition: universal adaptation and repeated success

The Lancet. Haematology, 2022-07, Vol.9 (7), p.e465-e466

George, Lindsey A

2022

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Coagulation Factor VIII: Biological Basis of Emerging Hemophilia A Therapies

Blood, 2024-08

2024

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Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

The New England journal of medicine, 2021-11, Vol.385 (21), p.1961-1973

2021

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

The New England journal of medicine, 2017-12, Vol.377 (23), p.2215-2227

2017

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Activated protein C has a regulatory role in factor VIII function

Blood, 2021-05, Vol.137 (18), p.2532-2543

2021

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Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH

Journal of thrombosis and haemostasis, 2020-11, Vol.18 (11), p.3074-3077

2020

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The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors

Haemophilia : the official journal of the World Federation of Hemophilia, 2019-07, Vol.25 (4), p.581-589

2019

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Combined anti‐CD20 and mTOR inhibition with factor VIII for immune tolerance induction in hemophilia A patients with refractory inhibitors

Journal of thrombosis and haemostasis, 2020-04, Vol.18 (4), p.848-852

2020

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World Federation of Hemophilia Gene Therapy Registry

Haemophilia : the official journal of the World Federation of Hemophilia, 2020-07, Vol.26 (4), p.563-564

2020

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Total Knee Arthroplasty after Gene Therapy for Hemophilia B

The New England journal of medicine, 2022-10, Vol.387 (17), p.1622-1624

2022

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Gene therapy for hemophilia: past, present and future

Seminars in hematology, 2016, Vol.53 (1), p.46-54

2016

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Haemophilia care: the only constant is change

British journal of haematology, 2021-09, Vol.194 (5), p.805-807

2021

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Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab

Blood, 2024-09, Vol.144 (11), p.1230-1235

2024

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Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua

Journal of thrombosis and haemostasis, 2021-05, Vol.19 (5), p.1212-1218

2021

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Retraction notice to "Maternal Nutrient Restriction Predisposes Ventricular Remodeling in Adult Sheep Offspring" [The Journal of Nutritional Biochemistry 24 (2013) 1258-1265]

The Journal of nutritional biochemistry, 2023-02, Vol.112, p.109243-109243, Article 109243

2023

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Hemophilia A Gene Therapy — Some Answers, More Questions

Adeno-Associated Virus Gene Therapy for Hemophilia

Hemophilia gene therapy: ushering in a new treatment paradigm?

No CpGs for AAVs?

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Current Clinical Applications of In Vivo Gene Therapy with AAVs

Hemophilia gene therapy comes of age

Hemophilia gene therapy comes of age

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

Pre-clinical evaluation of an enhanced-function factor VIII variant for durable hemophilia A gene therapy in male mice

Factor IX Padua for haemophilia B gene addition: universal adaptation and repeated success

Coagulation Factor VIII: Biological Basis of Emerging Hemophilia A Therapies

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A

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

Activated protein C has a regulatory role in factor VIII function

Core data set on safety, efficacy, and durability of hemophilia gene therapy for a global registry: Communication from the SSC of the ISTH

The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors

Combined anti‐CD20 and mTOR inhibition with factor VIII for immune tolerance induction in hemophilia A patients with refractory inhibitors

World Federation of Hemophilia Gene Therapy Registry

Total Knee Arthroplasty after Gene Therapy for Hemophilia B

Gene therapy for hemophilia: past, present and future

Haemophilia care: the only constant is change

Enhanced procoagulant activity of select hemophilia B causing factor IX variants with emicizumab

Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX‐Padua

Retraction notice to "Maternal Nutrient Restriction Predisposes Ventricular Remodeling in Adult Sheep Offspring" [The Journal of Nutritional Biochemistry 24 (2013) 1258-1265]

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