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Blood Advances
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February 24, 2026
Safety, efficacy and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B
Benjamin J Samelson-Jones, John Ej Rasko, Jonathan Ducore, et al.
JCI Insight
|
October 14, 2016
Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models
Joshua I Siner, Benjamin J Samelson-Jones, Julie M Crudele, et al.
Molecular Therapy. Methods & Clinical Development
|
December 14, 2023
Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog
Lucas Van Gorder, Bhavya S Doshi, Elinor Willis, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 9, 2025
Use of CD19-targeted immune modulation to eradicate AAV-neutralizing antibodies
Bhavya S Doshi, Caroline A Markmann, Noelle Novak, et al.
The New England Journal of Medicine
|
April 16, 2025
Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up Study
John E J Rasko, Benjamin J Samelson-Jones, Lindsey A George, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 20, 2020
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Lindsey A George, Margaret V Ragni, John E J Rasko, et al.
The New England Journal of Medicine
|
November 17, 2021
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
Lindsey A George, Paul E Monahan, M Elaine Eyster, et al.
Human Gene Therapy
|
February 13, 2026
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management
Perry B Shieh, Crystal Proud, Tamir Diamond, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 6, 2025
Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns
Dennis J Keselman, Juliana C Small, Tiffany Seneviratne, et al.
The New England Journal of Medicine
|
May 13, 2026
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
Rebecca C Ahrens-Nicklas, Chelsea Kotch, Aoife M Roche, et al.
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of 6
Search research articles
Search
Showing results (41-50 of 51) with videos related to
Sort By:
Page
of 6
Blood Advances
|
February 24, 2026
Safety, efficacy and patient-reported outcomes 6 years after fidanacogene elaparvovec in adults with hemophilia B
Benjamin J Samelson-Jones, John Ej Rasko, Jonathan Ducore, et al.
JCI Insight
|
October 14, 2016
Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models
Joshua I Siner, Benjamin J Samelson-Jones, Julie M Crudele, et al.
Molecular Therapy. Methods & Clinical Development
|
December 14, 2023
Analysis of vector genome integrations in multicentric lymphoma after AAV gene therapy in a severe hemophilia A dog
Lucas Van Gorder, Bhavya S Doshi, Elinor Willis, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
March 9, 2025
Use of CD19-targeted immune modulation to eradicate AAV-neutralizing antibodies
Bhavya S Doshi, Caroline A Markmann, Noelle Novak, et al.
The New England Journal of Medicine
|
April 16, 2025
Fidanacogene Elaparvovec for Hemophilia B - A Multiyear Follow-up Study
John E J Rasko, Benjamin J Samelson-Jones, Lindsey A George, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
June 20, 2020
Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B
Lindsey A George, Margaret V Ragni, John E J Rasko, et al.
The New England Journal of Medicine
|
November 17, 2021
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
Lindsey A George, Paul E Monahan, M Elaine Eyster, et al.
Human Gene Therapy
|
February 13, 2026
Learnings from Patient Mortality after Delandistrogene Moxeparvovec Administration: A Report of Two Cases and Expert Committee Considerations for Future Mitigation and Management
Perry B Shieh, Crystal Proud, Tamir Diamond, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy
|
October 6, 2025
Real-world outcomes of delandistrogene moxeparvovec gene therapy: Motor outcomes and emerging safety concerns
Dennis J Keselman, Juliana C Small, Tiffany Seneviratne, et al.
The New England Journal of Medicine
|
May 13, 2026
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery
Rebecca C Ahrens-Nicklas, Chelsea Kotch, Aoife M Roche, et al.
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of 6