التفاصيل البيبلوغرافية
| العنوان: |
Dismantling cost and infrastructure barriers to equitable access to gene therapies for sickle cell disease. |
| المؤلفون: |
Sharma A; Department of Bone Marrow Transplantation and Cellular Therapy, St Jude Children's Research Hospital, Memphis, TN 38105, USA. Electronic address: akshay.sharma@stjude.org., John TD; Department of Pediatrics, Hematology, Oncology, Stem Cell, and Regenerative Medicine, Stanford University, Palo Alto, CA, USA. |
| المصدر: |
The Lancet. Haematology [Lancet Haematol] 2024 Jul 01. Date of Electronic Publication: 2024 Jul 01. |
| Publication Model: |
Ahead of Print |
| نوع المنشور: |
Journal Article |
| اللغة: |
English |
| بيانات الدورية: |
Publisher: Elsevier Ltd Country of Publication: England NLM ID: 101643584 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 2352-3026 (Electronic) Linking ISSN: 23523026 NLM ISO Abbreviation: Lancet Haematol Subsets: MEDLINE |
| أسماء مطبوعة: |
Original Publication: [Oxford] : Elsevier Ltd., [2014]- |
| مستخلص: |
Competing Interests: AS has received consultant fees from Spotlight Therapeutics, Medexus, Vertex Pharmaceuticals, Sangamo Therapeutics, and Editas Medicine. He is a medical monitor for a Resource for Clinical Investigations in Blood and Marrow Transplantation Conditioning SCID Infants Diagnosed Early clinical trial for which he receives financial compensation. He has also received research funding from CRISPR Therapeutics and honoraria from Vindico Medical Education. AS is the St Jude Children's Research Hospital site principal investigator of clinical trials for genome editing of sickle cell disease sponsored by Vertex Pharmaceuticals/CRISPR Therapeutics (NCT03745287), Novartis Pharmaceuticals (NCT04443907), and Beam Therapeutics (NCT05456880). The industry sponsors provide funding for the clinical trial, which includes salary support paid to AS's institution. AS has no direct financial interest in these therapies. AS is supported by a grant (1U01HL163983) from the National Institutes of Health/National Heart, Lung, and Blood Institute. The content of this Comment is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. TDJ has participated as an advisory board member and received consulting fees from Bluebird Bio and Vertex Pharmaceuticals. She is a medical monitor for BMT CTN 2001 GRASP study for which she receives compensation. TDJ is the Stanford site principal investigator of clinical trials for genome editing sponsored by Beam Therapeutics (NCT04443907). TDJ has no direct financial interest in this therapy. We thank Keith A Laycock for scientific editing of the Comment. We also thank Jennifer Adair, Fred Hutch Cancer Center, Jane Hankins, St Jude Children's Research Hospital, and Zainab Garba-Sani, Stanford University and NHS England, for offering critical insights on the topic and reviewing an early draft of the manuscript. |
| تواريخ الأحداث: |
Date Created: 20240704 Latest Revision: 20240704 |
| رمز التحديث: |
20240705 |
| DOI: |
10.1016/S2352-3026(24)00175-3 |
| PMID: |
38964356 |
| قاعدة البيانات: |
MEDLINE |
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