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Balancing benefits and burdens: a systematic review on ethical and social dimensions of gene and cell therapies for hereditary blood diseases.权衡利弊:关于遗传性血液疾病基因和细胞疗法伦理与社会层面的系统评价
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Single-stranded DNA with internal base modifications mediates highly efficient knock-in in primary cells using CRISPR-Cas9.具有内部碱基修饰的单链DNA利用CRISPR-Cas9在原代细胞中介导高效的基因敲入。
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Progress Note 2024: Curing HIV; Not in My Lifetime or Just Around the Corner?2024年病程记录:治愈艾滋病病毒;是我有生之年无法实现还是指日可待?
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Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.制定改善镰状细胞病结局的全球战略:柳叶刀血液学委员会。
Lancet Haematol. 2023 Aug;10(8):e633-e686. doi: 10.1016/S2352-3026(23)00096-0. Epub 2023 Jul 11.
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Ethical and practical considerations for cell and gene therapy toward an HIV cure: findings from a qualitative in-depth interview study in the United States.细胞和基因治疗实现 HIV 治愈的伦理与实践考量:来自美国深入定性访谈研究的发现。
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The genome-editing decade.基因组编辑的十年。
Mol Ther. 2021 Nov 3;29(11):3093-3094. doi: 10.1016/j.ymthe.2021.10.016. Epub 2021 Oct 26.

本文引用的文献

1
Multi-stakeholder consensus on a target product profile for an HIV cure.多方利益相关者对 HIV 治愈的目标产品特征达成共识。
Lancet HIV. 2021 Jan;8(1):e42-e50. doi: 10.1016/S2352-3018(20)30234-4. Epub 2020 Nov 30.
2
The case for an HIV cure and how to get there.实现 HIV 治愈的理由与路径
Lancet HIV. 2021 Jan;8(1):e51-e58. doi: 10.1016/S2352-3018(20)30232-0. Epub 2020 Nov 30.
3
Bringing Gene Therapies for HIV Disease to Resource-Limited Parts of the World.将针对艾滋病的基因疗法推广至世界资源匮乏地区。
Hum Gene Ther. 2021 Jan;32(1-2):21-30. doi: 10.1089/hum.2020.252. Epub 2020 Oct 30.
4
Treating sickle cell anemia.治疗镰状细胞贫血。
Science. 2020 Mar 13;367(6483):1198-1199. doi: 10.1126/science.aba3827.
5
Why and where an HIV cure is needed and how it might be achieved.为什么需要艾滋病治愈以及在何处需要艾滋病治愈,以及如何实现艾滋病治愈。
Nature. 2019 Dec;576(7787):397-405. doi: 10.1038/s41586-019-1841-8. Epub 2019 Dec 18.
6
Finding a cure for sickle cell disease.寻找镰状细胞病的治愈方法。
Nat Med. 2019 Dec;25(12):1811. doi: 10.1038/s41591-019-0691-0.
7
Gates and NIH join forces on HIV and sickle cell diseases.盖茨基金会与美国国立卫生研究院携手应对艾滋病和镰状细胞病。
Science. 2019 Nov 1;366(6465):558-559. doi: 10.1126/science.366.6465.558-b.
8
HIV "cure": A shot in the arm?HIV“治愈”:一针见效?
EBioMedicine. 2019 Apr;42:3-5. doi: 10.1016/j.ebiom.2019.04.011. Epub 2019 Apr 11.
9
Gene therapy for blood diseases.血液疾病的基因治疗。
Curr Opin Biotechnol. 2019 Dec;60:39-45. doi: 10.1016/j.copbio.2018.11.016. Epub 2018 Dec 29.
10
Gene therapy comes of age.基因治疗走向成熟。
Science. 2018 Jan 12;359(6372). doi: 10.1126/science.aan4672.

Collaborative science to advance gene therapies in resource-limited parts of the world.

作者信息

McCune Joseph M, Stevenson Susan C, Doehle Brian P, Trenor Cameron C, Turner Emily H, Spector Jonathan M

机构信息

HIV Frontiers, Global Health Innovative Technology Solutions Bill & Melinda Gates Foundation, Seattle, WA, USA.

Novartis Institutes for BioMedical Research, Cambridge, MA, USA.

出版信息

Mol Ther. 2021 Nov 3;29(11):3101-3102. doi: 10.1016/j.ymthe.2021.05.024. Epub 2021 Aug 30.

DOI:10.1016/j.ymthe.2021.05.024
PMID:34464598
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8571166/
Abstract
摘要