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Items: 1 to 20 of 121

1.

Hepato-entrained B220+CD11c+NK1.1+ cells regulate pre-metastatic niche formation in the lung.

Hiratsuka S, Tomita T, Mishima T, Matsunaga Y, Omori T, Ishibashi S, Yamaguchi S, Hosogane T, Watarai H, Omori-Miyake M, Yamamoto T, Shibata N, Watanabe A, Aburatani H, Tomura M, High KA, Maru Y.

EMBO Mol Med. 2018 Jul;10(7). pii: e8643. doi: 10.15252/emmm.201708643.

2.

Gene therapy comes of age.

Dunbar CE, High KA, Joung JK, Kohn DB, Ozawa K, Sadelain M.

Science. 2018 Jan 12;359(6372). pii: eaan4672. doi: 10.1126/science.aan4672. Review.

PMID:
29326244
3.

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA.

N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.

4.

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape.

Mingozzi F, High KA.

Annu Rev Virol. 2017 Sep 29;4(1):511-534. doi: 10.1146/annurev-virology-101416-041936. Review.

PMID:
28961410
5.

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM.

Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14.

PMID:
28712537
6.

Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.

Chung DC, McCague S, Yu ZF, Thill S, DiStefano-Pappas J, Bennett J, Cross D, Marshall K, Wellman J, High KA.

Clin Exp Ophthalmol. 2018 Apr;46(3):247-259. doi: 10.1111/ceo.13022. Epub 2017 Aug 31.

PMID:
28697537
7.
8.

An edible switch for gene therapy.

Anguela XM, High KA.

Nat Biotechnol. 2016 Aug 9;34(8):824-5. doi: 10.1038/nbt.3645. No abstract available.

PMID:
27504775
9.

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM.

Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.

10.

Clinical development of gene therapy: results and lessons from recent successes.

Kumar SR, Markusic DM, Biswas M, High KA, Herzog RW.

Mol Ther Methods Clin Dev. 2016 May 25;3:16034. doi: 10.1038/mtm.2016.34. eCollection 2016. Review.

11.

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid.

Vercauteren K, Hoffman BE, Zolotukhin I, Keeler GD, Xiao JW, Basner-Tschakarjan E, High KA, Ertl HC, Rice CM, Srivastava A, de Jong YP, Herzog RW.

Mol Ther. 2016 Jun;24(6):1042-1049. doi: 10.1038/mt.2016.61. Epub 2016 Mar 29.

12.

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

Callan MB, Haskins ME, Wang P, Zhou S, High KA, Arruda VR.

PLoS One. 2016 Mar 24;11(3):e0151800. doi: 10.1371/journal.pone.0151800. eCollection 2016.

13.

Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII.

Marcos-Contreras OA, Smith SM, Bellinger DA, Raymer RA, Merricks E, Faella A, Pavani G, Zhou S, Nichols TC, High KA, Margaritis P.

Blood. 2016 Feb 4;127(5):565-71. doi: 10.1182/blood-2015-09-671420. Epub 2015 Dec 23.

14.

Adeno-associated viral vectors for the treatment of hemophilia.

High KA, Anguela XM.

Hum Mol Genet. 2016 Apr 15;25(R1):R36-41. doi: 10.1093/hmg/ddv475. Epub 2015 Nov 27. Review.

15.

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes.

Hui DJ, Edmonson SC, Podsakoff GM, Pien GC, Ivanciu L, Camire RM, Ertl H, Mingozzi F, High KA, Basner-Tschakarjan E.

Mol Ther Methods Clin Dev. 2015 Sep 30;2:15029. doi: 10.1038/mtm.2015.29. eCollection 2015.

16.

In vivo genome editing of the albumin locus as a platform for protein replacement therapy.

Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ, High KA.

Blood. 2015 Oct 8;126(15):1777-84. doi: 10.1182/blood-2014-12-615492. Epub 2015 Aug 21.

17.

Gene therapy for hemophilia: the clot thickens.

High KA.

Hum Gene Ther. 2014 Nov;25(11):915-22. doi: 10.1089/hum.2014.2541. No abstract available.

18.

Robust ZFN-mediated genome editing in adult hemophilic mice.

Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA.

Blood. 2013 Nov 7;122(19):3283-7. doi: 10.1182/blood-2013-04-497354. Epub 2013 Oct 1.

19.

CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome.

Wu TL, Li H, Faust SM, Chi E, Zhou S, Wright F, High KA, Ertl HC.

Mol Ther. 2014 Jan;22(1):42-51. doi: 10.1038/mt.2013.218. Epub 2013 Sep 30.

20.

Cellular localization and characterization of cytosolic binding partners for Gla domain-containing proteins PRRG4 and PRRG2.

Yazicioglu MN, Monaldini L, Chu K, Khazi FR, Murphy SL, Huang H, Margaritis P, High KA.

J Biol Chem. 2013 Sep 6;288(36):25908-14. doi: 10.1074/jbc.M113.484683. Epub 2013 Jul 19.

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