This site needs JavaScript to work properly. Please enable it to take advantage of the complete set of features!

Skip to main page content

Add to Collections

Your saved search

Name of saved search:

Search terms:

Test search terms

Would you like email updates of new search results?

Yes
No

Email: (change)

Frequency:

Which day?

Which day?

Report format:

Send at most:

Send even when there aren't any new results

Optional text in email:

Your RSS Feed

Review

. 2019 Aug 1;381(5):455-464.

doi: 10.1056/NEJMra1706910.

Gene Therapy

Katherine A High¹, Maria G Roncarolo¹

Affiliations

PMID: 31365802
DOI: 10.1056/NEJMra1706910

Review

Gene Therapy

Katherine A High et al. N Engl J Med. 2019.

. 2019 Aug 1;381(5):455-464.

doi: 10.1056/NEJMra1706910.

Authors

Katherine A High¹, Maria G Roncarolo¹

Affiliation

¹ From Spark Therapeutics, Philadelphia (K.A.H.); and Stanford University, Stanford, CA (M.G.R.).

PMID: 31365802
DOI: 10.1056/NEJMra1706910

No abstract available

PubMed Disclaimer

Similar articles

Regulating the gene-therapy revolution.
Bender E. Bender E. Nature. 2018 Dec;564(7735):S20-S22. doi: 10.1038/d41586-018-07641-1. Nature. 2018. PMID: 30542185 No abstract available.
Gene Addition Strategies for β-Thalassemia and Sickle Cell Anemia.
Dong AC, Rivella S. Dong AC, et al. Adv Exp Med Biol. 2017;1013:155-176. doi: 10.1007/978-1-4939-7299-9_6. Adv Exp Med Biol. 2017. PMID: 29127680 Free PMC article. Review.
Gene Therapy Approaches to Hemoglobinopathies.
Ferrari G, Cavazzana M, Mavilio F. Ferrari G, et al. Hematol Oncol Clin North Am. 2017 Oct;31(5):835-852. doi: 10.1016/j.hoc.2017.06.010. Hematol Oncol Clin North Am. 2017. PMID: 28895851 Review.
Major challenges for gene therapy of thalassemia and sickle cell disease.
Papanikolaou E, Anagnou NP. Papanikolaou E, et al. Curr Gene Ther. 2010 Oct;10(5):404-12. doi: 10.2174/156652310793180724. Curr Gene Ther. 2010. PMID: 20712578 Review.
Gene Therapy Briefs.
Philippidis A. Philippidis A. Hum Gene Ther Clin Dev. 2019 Dec;30(4):149-151. doi: 10.1089/humc.2019.29050.bfs. Hum Gene Ther Clin Dev. 2019. PMID: 31855473 No abstract available.

See all similar articles

Cited by

Peptide-guided JC polyomavirus-like particles specifically target bladder cancer cells for gene therapy.
Lai WH, Fang CY, Chou MC, Lin MC, Shen CH, Chao CN, Jou YC, Chang D, Wang M. Lai WH, et al. Sci Rep. 2021 Jun 4;11(1):11889. doi: 10.1038/s41598-021-91328-7. Sci Rep. 2021. PMID: 34088940 Free PMC article.
Engineering lentivirus envelope VSV-G for liver targeted delivery of IDOL-shRNA to ameliorate hypercholesterolemia and atherosclerosis.
Wang W, Chen X, Chen J, Xu M, Liu Y, Yang S, Zhao W, Tan S. Wang W, et al. Mol Ther Nucleic Acids. 2024 Jan 11;35(1):102115. doi: 10.1016/j.omtn.2024.102115. eCollection 2024 Mar 12. Mol Ther Nucleic Acids. 2024. PMID: 38314097 Free PMC article.
Adenoviral Gene Therapy Vectors in Clinical Use-Basic Aspects with a Special Reference to Replication-Competent Adenovirus Formation and Its Impact on Clinical Safety.
Leikas AJ, Ylä-Herttuala S, Hartikainen JEK. Leikas AJ, et al. Int J Mol Sci. 2023 Nov 20;24(22):16519. doi: 10.3390/ijms242216519. Int J Mol Sci. 2023. PMID: 38003709 Free PMC article. Review.
Peptide Amphiphiles as Biodegradable Adjuvants for Efficient Retroviral Gene Delivery.
Kaygisiz K, Rauch-Wirth L, Iscen A, Hartenfels J, Kremer K, Münch J, Synatschke CV, Weil T. Kaygisiz K, et al. Adv Healthc Mater. 2024 Feb;13(4):e2301364. doi: 10.1002/adhm.202301364. Epub 2023 Nov 23. Adv Healthc Mater. 2024. PMID: 37947246 Free PMC article.
The Arrival of Gene Therapy for Patients with Hemophilia A.
Castaman G, Di Minno G, De Cristofaro R, Peyvandi F. Castaman G, et al. Int J Mol Sci. 2022 Sep 6;23(18):10228. doi: 10.3390/ijms231810228. Int J Mol Sci. 2022. PMID: 36142153 Free PMC article. Review.

See all "Cited by" articles

Publication types

Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

LinkOut - more resources

Full Text Sources
- Atypon
- Ovid Technologies, Inc.
Other Literature Sources
- H1 Connect - Access expert opinions and insights on biomedical research.
- The Lens - Patent Citations Database
Medical
- MedlinePlus Health Information