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1.

Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A.

George, Lindsey A; Monahan, Paul E; Eyster, M Elaine; Sullivan, Spencer K; Ragni, Margaret V; Croteau, Stacy E; Rasko, John E J; Recht, Michael; Samelson-Jones, Benjamin J; MacDougall, Amy; Jaworski, Kristen; Noble, Robert; Curran, Marla; Kuranda, Klaudia; Mingozzi, Federico; Chang, Tiffany; Reape, Kathleen Z; Anguela, Xavier M; High, Katherine A.

N Engl J Med ; 385(21): 1961-1973, 2021 11 18.

Artículo en Inglés | MEDLINE | ID: mdl-34788507

2.

THE JEREMIAH METZGER LECTURE: TURNING GENES INTO MEDICINES: HIGHLIGHTS AND HURDLES IN THE DEVELOPMENT OF GENE THERAPY FOR GENETIC DISEASE.

High, Katherine A.

Trans Am Clin Climatol Assoc ; 133: 204-233, 2023.

Artículo en Inglés | MEDLINE | ID: mdl-37701622

3.

Entering the Modern Era of Gene Therapy.

Anguela, Xavier M; High, Katherine A.

Annu Rev Med ; 70: 273-288, 2019 01 27.

Artículo en Inglés | MEDLINE | ID: mdl-30477394

4.

Durability of Voretigene Neparvovec for Biallelic RPE65-Mediated Inherited Retinal Disease: Phase 3 Results at 3 and 4 Years.

Maguire, Albert M; Russell, Stephen; Chung, Daniel C; Yu, Zi-Fan; Tillman, Amy; Drack, Arlene V; Simonelli, Francesca; Leroy, Bart P; Reape, Kathleen Z; High, Katherine A; Bennett, Jean.

Ophthalmology ; 128(10): 1460-1468, 2021 10.

Artículo en Inglés | MEDLINE | ID: mdl-33798654

5.

The Effect of CpG Sequences on Capsid-Specific CD8⁺ T Cell Responses to AAV Vector Gene Transfer.

Xiang, ZhiQuan; Kurupati, Raj K; Li, Yan; Kuranda, Klaudia; Zhou, Xiangyang; Mingozzi, Federico; High, Katherine A; Ertl, Hildegund C J.

Mol Ther ; 28(3): 771-783, 2020 03 04.

Artículo en Inglés | MEDLINE | ID: mdl-31839483

6.

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B.

George, Lindsey A; Ragni, Margaret V; Rasko, John E J; Raffini, Leslie J; Samelson-Jones, Benjamin J; Ozelo, Margareth; Hazbon, Maria; Runowski, Alexa R; Wellman, Jennifer A; Wachtel, Katie; Chen, Yifeng; Anguela, Xavier M; Kuranda, Klaudia; Mingozzi, Federico; High, Katherine A.

Mol Ther ; 28(9): 2073-2082, 2020 09 02.

Artículo en Inglés | MEDLINE | ID: mdl-32559433

7.

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

George, Lindsey A; Sullivan, Spencer K; Giermasz, Adam; Rasko, John E J; Samelson-Jones, Benjamin J; Ducore, Jonathan; Cuker, Adam; Sullivan, Lisa M; Majumdar, Suvankar; Teitel, Jerome; McGuinn, Catherine E; Ragni, Margaret V; Luk, Alvin Y; Hui, Daniel; Wright, J Fraser; Chen, Yifeng; Liu, Yun; Wachtel, Katie; Winters, Angela; Tiefenbacher, Stefan; Arruda, Valder R; van der Loo, Johannes C M; Zelenaia, Olga; Takefman, Daniel; Carr, Marcus E; Couto, Linda B; Anguela, Xavier M; High, Katherine A.

N Engl J Med ; 377(23): 2215-2227, 2017 12 07.

Artículo en Inglés | MEDLINE | ID: mdl-29211678

8.

Gene and cell therapy in 2023: Rich pipeline, slimming resources?

High, Katherine A.

Mol Ther ; 32(1): 3-4, 2024 Jan 03.

Artículo en Inglés | MEDLINE | ID: mdl-38118443

9.

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.

Maguire, Albert M; Russell, Stephen; Wellman, Jennifer A; Chung, Daniel C; Yu, Zi-Fan; Tillman, Amy; Wittes, Janet; Pappas, Julie; Elci, Okan; Marshall, Kathleen A; McCague, Sarah; Reichert, Hannah; Davis, Maria; Simonelli, Francesca; Leroy, Bart P; Wright, J Fraser; High, Katherine A; Bennett, Jean.

Ophthalmology ; 126(9): 1273-1285, 2019 09.

Artículo en Inglés | MEDLINE | ID: mdl-31443789

10.

Adeno-associated viral vectors for the treatment of hemophilia.

High, Katherine A; Anguela, Xavier M.

Hum Mol Genet ; 25(R1): R36-41, 2016 Apr 15.

Artículo en Inglés | MEDLINE | ID: mdl-26614390

11.

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, Stephen; Bennett, Jean; Wellman, Jennifer A; Chung, Daniel C; Yu, Zi-Fan; Tillman, Amy; Wittes, Janet; Pappas, Julie; Elci, Okan; McCague, Sarah; Cross, Dominique; Marshall, Kathleen A; Walshire, Jean; Kehoe, Taylor L; Reichert, Hannah; Davis, Maria; Raffini, Leslie; George, Lindsey A; Hudson, F Parker; Dingfield, Laura; Zhu, Xiaosong; Haller, Julia A; Sohn, Elliott H; Mahajan, Vinit B; Pfeifer, Wanda; Weckmann, Michelle; Johnson, Chris; Gewaily, Dina; Drack, Arlene; Stone, Edwin; Wachtel, Katie; Simonelli, Francesca; Leroy, Bart P; Wright, J Fraser; High, Katherine A; Maguire, Albert M.

Lancet ; 390(10097): 849-860, 2017 Aug 26.

Artículo en Inglés | MEDLINE | ID: mdl-28712537

12.

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

Marcos-Contreras, Oscar A; Smith, Shannon M; Bellinger, Dwight A; Raymer, Robin A; Merricks, Elizabeth; Faella, Armida; Pavani, Giulia; Zhou, Shangzhen; Nichols, Timothy C; High, Katherine A; Margaritis, Paris.

Blood ; 127(5): 565-71, 2016 Feb 04.

Artículo en Inglés | MEDLINE | ID: mdl-26702064

13.

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

Chung, Daniel C; McCague, Sarah; Yu, Zi-Fan; Thill, Satha; DiStefano-Pappas, Julie; Bennett, Jean; Cross, Dominique; Marshall, Kathleen; Wellman, Jennifer; High, Katherine A.

Clin Exp Ophthalmol ; 46(3): 247-259, 2018 04.

Artículo en Inglés | MEDLINE | ID: mdl-28697537

14.

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, Jean; Wellman, Jennifer; Marshall, Kathleen A; McCague, Sarah; Ashtari, Manzar; DiStefano-Pappas, Julie; Elci, Okan U; Chung, Daniel C; Sun, Junwei; Wright, J Fraser; Cross, Dominique R; Aravand, Puya; Cyckowski, Laura L; Bennicelli, Jeannette L; Mingozzi, Federico; Auricchio, Alberto; Pierce, Eric A; Ruggiero, Jason; Leroy, Bart P; Simonelli, Francesca; High, Katherine A; Maguire, Albert M.

Lancet ; 388(10045): 661-72, 2016 Aug 13.

Artículo en Inglés | MEDLINE | ID: mdl-27375040

15.

Gene Therapy.

High, Katherine A; Roncarolo, Maria G.

N Engl J Med ; 381(5): 455-464, 2019 08 01.

Artículo en Inglés | MEDLINE | ID: mdl-31365802

16.

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

Nathwani, Amit C; Reiss, Ulreke M; Tuddenham, Edward G D; Rosales, Cecilia; Chowdary, Pratima; McIntosh, Jenny; Della Peruta, Marco; Lheriteau, Elsa; Patel, Nishal; Raj, Deepak; Riddell, Anne; Pie, Jun; Rangarajan, Savita; Bevan, David; Recht, Michael; Shen, Yu-Min; Halka, Kathleen G; Basner-Tschakarjan, Etiena; Mingozzi, Federico; High, Katherine A; Allay, James; Kay, Mark A; Ng, Catherine Y C; Zhou, Junfang; Cancio, Maria; Morton, Christopher L; Gray, John T; Srivastava, Deokumar; Nienhuis, Arthur W; Davidoff, Andrew M.

N Engl J Med ; 371(21): 1994-2004, 2014 Nov 20.

Artículo en Inglés | MEDLINE | ID: mdl-25409372

17.

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

Sharma, Rajiv; Anguela, Xavier M; Doyon, Yannick; Wechsler, Thomas; DeKelver, Russell C; Sproul, Scott; Paschon, David E; Miller, Jeffrey C; Davidson, Robert J; Shivak, David; Zhou, Shangzhen; Rieders, Julianne; Gregory, Philip D; Holmes, Michael C; Rebar, Edward J; High, Katherine A.

Blood ; 126(15): 1777-84, 2015 Oct 08.

Artículo en Inglés | MEDLINE | ID: mdl-26297739

18.

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.

Mingozzi, Federico; High, Katherine A.

Nat Rev Genet ; 12(5): 341-55, 2011 May.

Artículo en Inglés | MEDLINE | ID: mdl-21499295

19.

In vivo genome editing restores haemostasis in a mouse model of haemophilia.

Li, Hojun; Haurigot, Virginia; Doyon, Yannick; Li, Tianjian; Wong, Sunnie Y; Bhagwat, Anand S; Malani, Nirav; Anguela, Xavier M; Sharma, Rajiv; Ivanciu, Lacramiora; Murphy, Samuel L; Finn, Jonathan D; Khazi, Fayaz R; Zhou, Shangzhen; Paschon, David E; Rebar, Edward J; Bushman, Frederic D; Gregory, Philip D; Holmes, Michael C; High, Katherine A.

Nature ; 475(7355): 217-21, 2011 Jun 26.

Artículo en Inglés | MEDLINE | ID: mdl-21706032

20.

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

Vercauteren, Koen; Hoffman, Brad E; Zolotukhin, Irene; Keeler, Geoffrey D; Xiao, Jing W; Basner-Tschakarjan, Etiena; High, Katherine A; Ertl, Hildegund Cj; Rice, Charles M; Srivastava, Arun; de Jong, Ype P; Herzog, Roland W.

Mol Ther ; 24(6): 1042-1049, 2016 06.

Artículo en Inglés | MEDLINE | ID: mdl-27019999

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