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Items: 1 to 50 of 218

1.

Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers?

Anguela XM, High KA.

Res Pract Thromb Haemost. 2019 Feb 28;3(2):149-151. doi: 10.1002/rth2.12190. eCollection 2019 Apr. No abstract available.

2.

Entering the Modern Era of Gene Therapy.

Anguela XM, High KA.

Annu Rev Med. 2019 Jan 27;70:273-288. doi: 10.1146/annurev-med-012017-043332. Epub 2018 Nov 26.

PMID:
30477394
3.

The Natural History of Inherited Retinal Dystrophy Due to Biallelic Mutations in the RPE65 Gene.

Chung DC, Bertelsen M, Lorenz B, Pennesi ME, Leroy BP, Hamel CP, Pierce E, Sallum J, Larsen M, Stieger K, Preising M, Weleber R, Yang P, Place E, Liu E, Schaefer G, DiStefano-Pappas J, Elci OU, McCague S, Wellman JA, High KA, Reape KZ.

Am J Ophthalmol. 2019 Mar;199:58-70. doi: 10.1016/j.ajo.2018.09.024. Epub 2018 Sep 28.

PMID:
30268864
4.

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.

5.

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
6.

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.

7.

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
8.

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. Erratum in: Lancet. 2017 Aug 26;390(10097):848.

9.

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.

10.
11.

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
12.

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.

13.

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.

14.

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.

15.

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.

16.

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.

17.

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.

18.

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.

19.

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.

20.

Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):5-14. doi: 10.1089/humc.2014.153. Epub 2015 Feb 12. Review.

21.

Perspectives on best practices for gene therapy programs.

Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, McDonald CL, McLaughlin J, Weiss McLeod B, Mendell JR, Nuckolls G, Stedman HH, Tagle DA, Vandenberghe LH, Wang H, Wernett PJ, Wilson JM, Porter JD, Gubitz AK.

Hum Gene Ther. 2015 Mar;26(3):127-33. doi: 10.1089/hum.2014.147. Epub 2015 Mar 3.

22.

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

Nathwani AC, Reiss UM, Tuddenham EG, Rosales C, Chowdary P, McIntosh J, Della Peruta M, Lheriteau E, Patel N, Raj D, Riddell A, Pie J, Rangarajan S, Bevan D, Recht M, Shen YM, Halka KG, Basner-Tschakarjan E, Mingozzi F, High KA, Allay J, Kay MA, Ng CY, Zhou J, Cancio M, Morton CL, Gray JT, Srivastava D, Nienhuis AW, Davidoff AM.

N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.

23.

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.

24.

Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies.

Markusic DM, Hoffman BE, Perrin GQ, Nayak S, Wang X, LoDuca PA, High KA, Herzog RW.

EMBO Mol Med. 2013 Nov;5(11):1698-709. doi: 10.1002/emmm.201302859. Epub 2013 Sep 16.

25.

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.

26.

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.

27.

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.

28.

Overcoming preexisting humoral immunity to AAV using capsid decoys.

Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA.

Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795.

29.

IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression.

Gil-Fariña I, Di Scala M, Vanrell L, Olagüe C, Vales A, High KA, Prieto J, Mingozzi F, Gonzalez-Aseguinolaza G.

PLoS One. 2013 Jul 2;8(7):e67748. doi: 10.1371/journal.pone.0067748. Print 2013.

30.

Enhanced T cell function in a mouse model of human glycosylation.

Buchlis G, Odorizzi P, Soto PC, Pearce OM, Hui DJ, Jordan MS, Varki A, Wherry EJ, High KA.

J Immunol. 2013 Jul 1;191(1):228-37. doi: 10.4049/jimmunol.1202905. Epub 2013 May 24.

31.

Immune responses to AAV vectors: overcoming barriers to successful gene therapy.

Mingozzi F, High KA.

Blood. 2013 Jul 4;122(1):23-36. doi: 10.1182/blood-2013-01-306647. Epub 2013 Apr 17. Review.

32.

Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012.

O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, Montgomery M, Rosenthal E, Samulski RJ, Skarlatos SI, Sorrentino B, Wilson JM, Xie Y, Corrigan-Curay J.

Hum Gene Ther. 2013 Apr;24(4):355-62. doi: 10.1089/hum.2013.064.

33.

Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.

Testa F, Maguire AM, Rossi S, Pierce EA, Melillo P, Marshall K, Banfi S, Surace EM, Sun J, Acerra C, Wright JF, Wellman J, High KA, Auricchio A, Bennett J, Simonelli F.

Ophthalmology. 2013 Jun;120(6):1283-91. doi: 10.1016/j.ophtha.2012.11.048. Epub 2013 Mar 6.

34.

Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans.

Parzych EM, Li H, Yin X, Liu Q, Wu TL, Podsakoff GM, High KA, Levine MH, Ertl HC.

Hum Gene Ther. 2013 Apr;24(4):431-42. doi: 10.1089/hum.2012.246.

35.

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant.

McIntosh J, Lenting PJ, Rosales C, Lee D, Rabbanian S, Raj D, Patel N, Tuddenham EG, Christophe OD, McVey JH, Waddington S, Nienhuis AW, Gray JT, Fagone P, Mingozzi F, Zhou SZ, High KA, Cancio M, Ng CY, Zhou J, Morton CL, Davidoff AM, Nathwani AC.

Blood. 2013 Apr 25;121(17):3335-44. doi: 10.1182/blood-2012-10-462200. Epub 2013 Feb 20.

36.

Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

Callejas D, Mann CJ, Ayuso E, Lage R, Grifoll I, Roca C, Andaluz A, Ruiz-de Gopegui R, Montané J, Muñoz S, Ferre T, Haurigot V, Zhou S, Ruberte J, Mingozzi F, High KA, Garcia F, Bosch F.

Diabetes. 2013 May;62(5):1718-29. doi: 10.2337/db12-1113. Epub 2013 Feb 1.

37.

Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases.

Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G.

Mol Ther. 2013 Feb;21(2):266-8. doi: 10.1038/mt.2013.4. No abstract available.

38.

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells.

Martino AT, Basner-Tschakarjan E, Markusic DM, Finn JD, Hinderer C, Zhou S, Ostrov DA, Srivastava A, Ertl HC, Terhorst C, High KA, Mingozzi F, Herzog RW.

Blood. 2013 Mar 21;121(12):2224-33. doi: 10.1182/blood-2012-10-460733. Epub 2013 Jan 16.

39.

The gene therapy journey for hemophilia: are we there yet?

High KA.

Hematology Am Soc Hematol Educ Program. 2012;2012:375-81. doi: 10.1182/asheducation-2012.1.375. Review.

PMID:
23233607
40.

The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

Finn JD, Nichols TC, Svoronos N, Merricks EP, Bellenger DA, Zhou S, Simioni P, High KA, Arruda VR.

Blood. 2012 Nov 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123. Epub 2012 Aug 23.

41.

The gene therapy journey for hemophilia: are we there yet?

High KA.

Blood. 2012 Nov 29;120(23):4482-7. doi: 10.1182/blood-2012-05-423210. Epub 2012 Jul 24. Review.

42.

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue.

Mingozzi F, Chen Y, Edmonson SC, Zhou S, Thurlings RM, Tak PP, High KA, Vervoordeldonk MJ.

Gene Ther. 2013 Apr;20(4):417-24. doi: 10.1038/gt.2012.55. Epub 2012 Jul 12.

43.

Gene therapy research at the frontiers of viral immunology.

Hoffman BE, Ertl HC, Terhorst C, High KA, Herzog RW.

Front Microbiol. 2012 May 24;3:182. doi: 10.3389/fmicb.2012.00182. eCollection 2012. No abstract available.

44.

Safe, long-term hepatic expression of anti-HCV shRNA in a nonhuman primate model.

Suhy DA, Kao SC, Mao T, Whiteley L, Denise H, Souberbielle B, Burdick AD, Hayes K, Wright JF, Lavender H, Roelvink P, Kolykhalov A, Brady K, Moschos SA, Hauck B, Zelenaia O, Zhou S, Scribner C, High KA, Renison SH, Corbau R.

Mol Ther. 2012 Sep;20(9):1737-49. doi: 10.1038/mt.2012.119. Epub 2012 Jun 26.

45.

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

Mingozzi F, Chen Y, Murphy SL, Edmonson SC, Tai A, Price SD, Metzger ME, Zhou S, Wright JF, Donahue RE, Dunbar CE, High KA.

Mol Ther. 2012 Jul;20(7):1410-6. doi: 10.1038/mt.2012.84. Epub 2012 May 8.

46.

AAV2 gene therapy readministration in three adults with congenital blindness.

Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM.

Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.

47.

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

Buchlis G, Podsakoff GM, Radu A, Hawk SM, Flake AW, Mingozzi F, High KA.

Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23. Erratum in: Blood. 2014 Mar 13;123(11):1768.

48.

Self-complementary AAVs induce more potent transgene product-specific immune responses compared to a single-stranded genome.

Wu T, Töpfer K, Lin SW, Li H, Bian A, Zhou XY, High KA, Ertl HC.

Mol Ther. 2012 Mar;20(3):572-9. doi: 10.1038/mt.2011.280. Epub 2011 Dec 20.

49.

Adenovirus-associated virus vector-mediated gene transfer in hemophilia B.

Nathwani AC, Tuddenham EG, Rangarajan S, Rosales C, McIntosh J, Linch DC, Chowdary P, Riddell A, Pie AJ, Harrington C, O'Beirne J, Smith K, Pasi J, Glader B, Rustagi P, Ng CY, Kay MA, Zhou J, Spence Y, Morton CL, Allay J, Coleman J, Sleep S, Cunningham JM, Srivastava D, Basner-Tschakarjan E, Mingozzi F, High KA, Gray JT, Reiss UM, Nienhuis AW, Davidoff AM.

N Engl J Med. 2011 Dec 22;365(25):2357-65. doi: 10.1056/NEJMoa1108046. Epub 2011 Dec 10.

50.

Clinical trial opportunities in hemostasis and thrombosis: NHBLI State-of-the-Science symposium.

Cines DB, Blajchman MA, High KA, Bussel JB; State-of-the Science Symposium Hemostasis-Thrombosis Committee.

Am J Hematol. 2012 Feb;87(2):235-8. doi: 10.1002/ajh.22225. Epub 2011 Nov 25.

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