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Gene therapy for hemophilia: the clot thickens.
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Status of gene transfer for hemophilia A and B.
Thromb Haemost. 1991 Jul 12;66(1):119-22.
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A golden age for Haemophilia treatment?
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Genie in a vector.
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Molecular basis of hemophilia.
Hematol Pathol. 1990;4(1):1-26.
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Looking to the future of gene therapy for hemophilia A and B.
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Gene therapy for haemophilia.
Baillieres Clin Haematol. 1996 Jun;9(2):305-17. doi: 10.1016/s0950-3536(96)80065-5.
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Deficiencies in factors IX and VIII: what is now known.
Hosp Pract (Off Ed). 1992 Feb 15;27(2):41-51. doi: 10.1080/21548331.1992.11705358.
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Molecular biology of the hemophilias.
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First hemophilia B gene therapy approved: More than two decades in the making.
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Congenital Bleeding Disorders: Managing Central Nervous System Bleeding in an Adult Hemophiliac.
Cureus. 2023 Mar 30;15(3):e36906. doi: 10.7759/cureus.36906. eCollection 2023 Mar.
2
Comparison of different gene addition strategies to modify placental derived-mesenchymal stromal cells to produce FVIII.
Front Immunol. 2022 Dec 15;13:954984. doi: 10.3389/fimmu.2022.954984. eCollection 2022.
3
Organoids and microphysiological systems: Promising models for accelerating AAV gene therapy studies.
Front Immunol. 2022 Sep 26;13:1011143. doi: 10.3389/fimmu.2022.1011143. eCollection 2022.
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Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A.
Mol Ther Methods Clin Dev. 2020 Mar 14;17:465-477. doi: 10.1016/j.omtm.2020.03.001. eCollection 2020 Jun 12.
7
Clinical Considerations for Capsid Choice in the Development of Liver-Targeted AAV-Based Gene Transfer.
Mol Ther Methods Clin Dev. 2019 Sep 10;15:170-178. doi: 10.1016/j.omtm.2019.08.015. eCollection 2019 Dec 13.
8
Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors.
Mol Ther Methods Clin Dev. 2016 Dec 24;4:149-158. doi: 10.1016/j.omtm.2016.12.004. eCollection 2017 Mar 17.
9
Clinical and imaging analysis of subclinical hemophilia combined with coxarthrosis: case report and literature review.
Springerplus. 2016 Dec 1;5(1):2058. doi: 10.1186/s40064-016-3727-7. eCollection 2016.

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