Suppr
超能文献

Gene Therapy.

作者信息

High Katherine A, Roncarolo Maria G

机构信息

From Spark Therapeutics, Philadelphia (K.A.H.); and Stanford University, Stanford, CA (M.G.R.).

出版信息

N Engl J Med. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910.

DOI:10.1056/NEJMra1706910

PMID:31365802

Abstract

摘要

相似文献

Gene Therapy.

N Engl J Med. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910.

Regulating the gene-therapy revolution.

Nature. 2018 Dec;564(7735):S20-S22. doi: 10.1038/d41586-018-07641-1.

Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.

Adv Exp Med Biol. 2017;1013:155-176. doi: 10.1007/978-1-4939-7299-9_6.

Gene Therapy Approaches to Hemoglobinopathies.

Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010.

Major challenges for gene therapy of thalassemia and sickle cell disease.

Curr Gene Ther. 2010 Oct;10(5):404-12. doi: 10.2174/156652310793180724.

Gene Therapy Briefs.

Hum Gene Ther Clin Dev. 2019 Dec;30(4):149-151. doi: 10.1089/humc.2019.29050.bfs.

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.

Hum Gene Ther. 2016 Feb;27(2):148-65. doi: 10.1089/hum.2016.007.

Stem cell therapy for sickle cell disease: transplantation and gene therapy.

Hematology Am Soc Hematol Educ Program. 2005:66-73. doi: 10.1182/asheducation-2005.1.66.

Development of gene therapies-lessons from nusinersen.

Gene Ther. 2017 Sep;24(9):527-528. doi: 10.1038/gt.2017.64. Epub 2017 Jul 24.

Gene Therapy for Sickle Cell Disease, β-Thalassemia Enters Regulatory Reviews.

JAMA. 2022 Nov 8;328(18):1798. doi: 10.1001/jama.2022.18448.

引用本文的文献

CAR Cell-Derived Exosomes in Cancer Therapy: Biogenesis, Engineering Strategies and Antitumor Mechanisms.

Int J Mol Sci. 2025 Aug 15;26(16):7890. doi: 10.3390/ijms26167890.

VEGFR1 as a Target for Cardiovascular Gene Therapy.

J Cardiovasc Transl Res. 2025 Aug 21. doi: 10.1007/s12265-025-10672-5.

Enhanced B cell electroporation efficiency via inhibition of DNA-induced apoptosis and pyroptosis with pan-caspase inhibitor.

Mol Ther Methods Clin Dev. 2025 Jul 28;33(3):101542. doi: 10.1016/j.omtm.2025.101542. eCollection 2025 Sep 11.

Pharmacovigilance in Cell and Gene Therapy: Evolving Challenges in Risk Management and Long-Term Follow-Up.

Drug Saf. 2025 Aug 9. doi: 10.1007/s40264-025-01596-9.

Application of Gene Therapy to Oral Diseases.

Pharmaceutics. 2025 Jun 30;17(7):859. doi: 10.3390/pharmaceutics17070859.

Retinitis Pigmentosa: From Genetic Insights to Innovative Therapeutic Approaches-A Literature Review.

Medicina (Kaunas). 2025 Jun 29;61(7):1179. doi: 10.3390/medicina61071179.

Natural products proposed for the management of Huntington's disease (HD): a comprehensive review.

Naunyn Schmiedebergs Arch Pharmacol. 2025 Jul 17. doi: 10.1007/s00210-025-04444-w.

Unlocking the Puzzle of Mammalian Transfection: The Role of the RNA-sensing-Mediated Interferon Response in the Cellular Defense Against Foreign DNA Intrusion.

Int J Biol Sci. 2025 Jun 9;21(9):3886-3900. doi: 10.7150/ijbs.107510. eCollection 2025.

Hematopoietic stem cell gene therapy of neurometabolic lysosomal storage diseases.

Brain Dev. 2025 Aug;47(4):104384. doi: 10.1016/j.braindev.2025.104384. Epub 2025 Jun 25.

An optimizing lentiviral titer determination assay based on Raji cells.

Am J Transl Res. 2025 May 15;17(5):3939-3950. doi: 10.62347/ZTKM3637. eCollection 2025.

文献AI研究员

20分钟写一篇综述，助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型，支持多种主流文档格式。

立即体验

相似文献

Gene Therapy.

N Engl J Med. 2019 Aug 1;381(5):455-464. doi: 10.1056/NEJMra1706910.

Regulating the gene-therapy revolution.

Nature. 2018 Dec;564(7735):S20-S22. doi: 10.1038/d41586-018-07641-1.

Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.

Adv Exp Med Biol. 2017;1013:155-176. doi: 10.1007/978-1-4939-7299-9_6.

Gene Therapy Approaches to Hemoglobinopathies.

Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010.

Major challenges for gene therapy of thalassemia and sickle cell disease.

Curr Gene Ther. 2010 Oct;10(5):404-12. doi: 10.2174/156652310793180724.

Gene Therapy Briefs.

Hum Gene Ther Clin Dev. 2019 Dec;30(4):149-151. doi: 10.1089/humc.2019.29050.bfs.

Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.

Hum Gene Ther. 2016 Feb;27(2):148-65. doi: 10.1089/hum.2016.007.

Stem cell therapy for sickle cell disease: transplantation and gene therapy.

Hematology Am Soc Hematol Educ Program. 2005:66-73. doi: 10.1182/asheducation-2005.1.66.

Development of gene therapies-lessons from nusinersen.

Gene Ther. 2017 Sep;24(9):527-528. doi: 10.1038/gt.2017.64. Epub 2017 Jul 24.

Gene Therapy for Sickle Cell Disease, β-Thalassemia Enters Regulatory Reviews.

JAMA. 2022 Nov 8;328(18):1798. doi: 10.1001/jama.2022.18448.

引用本文的文献

CAR Cell-Derived Exosomes in Cancer Therapy: Biogenesis, Engineering Strategies and Antitumor Mechanisms.

Int J Mol Sci. 2025 Aug 15;26(16):7890. doi: 10.3390/ijms26167890.

VEGFR1 as a Target for Cardiovascular Gene Therapy.

J Cardiovasc Transl Res. 2025 Aug 21. doi: 10.1007/s12265-025-10672-5.

Enhanced B cell electroporation efficiency via inhibition of DNA-induced apoptosis and pyroptosis with pan-caspase inhibitor.

Mol Ther Methods Clin Dev. 2025 Jul 28;33(3):101542. doi: 10.1016/j.omtm.2025.101542. eCollection 2025 Sep 11.

Pharmacovigilance in Cell and Gene Therapy: Evolving Challenges in Risk Management and Long-Term Follow-Up.

Drug Saf. 2025 Aug 9. doi: 10.1007/s40264-025-01596-9.

Application of Gene Therapy to Oral Diseases.

Pharmaceutics. 2025 Jun 30;17(7):859. doi: 10.3390/pharmaceutics17070859.

Retinitis Pigmentosa: From Genetic Insights to Innovative Therapeutic Approaches-A Literature Review.

Medicina (Kaunas). 2025 Jun 29;61(7):1179. doi: 10.3390/medicina61071179.

Natural products proposed for the management of Huntington's disease (HD): a comprehensive review.

Naunyn Schmiedebergs Arch Pharmacol. 2025 Jul 17. doi: 10.1007/s00210-025-04444-w.

Unlocking the Puzzle of Mammalian Transfection: The Role of the RNA-sensing-Mediated Interferon Response in the Cellular Defense Against Foreign DNA Intrusion.

Int J Biol Sci. 2025 Jun 9;21(9):3886-3900. doi: 10.7150/ijbs.107510. eCollection 2025.

Hematopoietic stem cell gene therapy of neurometabolic lysosomal storage diseases.

Brain Dev. 2025 Aug;47(4):104384. doi: 10.1016/j.braindev.2025.104384. Epub 2025 Jun 25.

An optimizing lentiviral titer determination assay based on Raji cells.

Am J Transl Res. 2025 May 15;17(5):3939-3950. doi: 10.62347/ZTKM3637. eCollection 2025.

Suppr超能文献

Gene Therapy.

作者信息

机构信息

出版信息

相似文献

引用本文的文献

文献AI研究员

用中文搜PubMed

文档翻译