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1.

ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.

Pagant, Silvere; Huston, Marshall W; Moreira, Luciana; Gan, Lin; St Martin, Susan; Sproul, Scott; Holmes, Michael C; Meyer, Kathleen; Wechsler, Thomas; Desnick, Robert J; Yasuda, Makiko.

Mol Ther; 29(11): 3230-3242, 2021 11 03.

Artículo en Inglés | MEDLINE | ID: mdl-33775910

2.

ZFN-Mediated In Vivo Genome Editing Corrects Murine Hurler Syndrome.

Ou, Li; DeKelver, Russell C; Rohde, Michelle; Tom, Susan; Radeke, Robert; St Martin, Susan J; Santiago, Yolanda; Sproul, Scott; Przybilla, Michael J; Koniar, Brenda L; Podetz-Pedersen, Kelly M; Laoharawee, Kanut; Cooksley, Renee D; Meyer, Kathleen E; Holmes, Michael C; McIvor, R Scott; Wechsler, Thomas; Whitley, Chester B.

Mol Ther; 27(1): 178-187, 2019 01 02.

Artículo en Inglés | MEDLINE | ID: mdl-30528089

3.

Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.

Laoharawee, Kanut; DeKelver, Russell C; Podetz-Pedersen, Kelly M; Rohde, Michelle; Sproul, Scott; Nguyen, Hoang-Oanh; Nguyen, Tam; St Martin, Susan J; Ou, Li; Tom, Susan; Radeke, Robert; Meyer, Kathleen E; Holmes, Michael C; Whitley, Chester B; Wechsler, Thomas; McIvor, R Scott.

Mol Ther; 26(4): 1127-1136, 2018 04 04.

Artículo en Inglés | MEDLINE | ID: mdl-29580682

4.

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

5.

Aberrant chromosome morphology in human cells defective for Holliday junction resolution.

Wechsler, Thomas; Newman, Scott; West, Stephen C.

Nature; 471(7340): 642-6, 2011 Mar 31.

Artículo en Inglés | MEDLINE | ID: mdl-21399624

6.

AAV9-NGLY1 gene replacement therapy improves phenotypic and biomarker endpoints in a rat model of NGLY1 Deficiency.

Zhu, Lei; Tan, Brandon; Dwight, Selina S; Beahm, Brendan; Wilsey, Matt; Crawford, Brett E; Schweighardt, Becky; Cook, Jennifer W; Wechsler, Thomas; Mueller, William F.

Mol Ther Methods Clin Dev; 27: 259-271, 2022 Dec 08.

Artículo en Inglés | MEDLINE | ID: mdl-36320418

7.

GlcNAc-Asn is a biomarker for NGLY1 deficiency.

Mueller, William F; Zhu, Lei; Tan, Brandon; Dwight, Selina; Beahm, Brendan; Wilsey, Matt; Wechsler, Thomas; Mak, Justin; Cowan, Tina; Pritchett, Jake; Taylor, Eric; Crawford, Brett E.

J Biochem; 171(2): 177-186, 2022 Feb 21.

Artículo en Inglés | MEDLINE | ID: mdl-34697629

8.

AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.

Yasuda, Makiko; Huston, Marshall W; Pagant, Silvere; Gan, Lin; St Martin, Susan; Sproul, Scott; Richards, Daniel; Ballaron, Stephen; Hettini, Khaled; Ledeboer, Annemarie; Falese, Lillian; Cao, Liching; Lu, Yanmei; Holmes, Michael C; Meyer, Kathleen; Desnick, Robert J; Wechsler, Thomas.

Mol Ther Methods Clin Dev; 18: 607-619, 2020 Sep 11.

Artículo en Inglés | MEDLINE | ID: mdl-32775495

9.

Woven fabrics: recent developments and future applications.

Wechsler, Thomas.

Med Device Technol; 14(5): 35-6, 2003 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-12852123

10.

Filters and open-heart surgery.

Wechsler, Thomas.

Med Device Technol; 13(5): 46, 2002 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-12116735

11.

RIDDLE immunodeficiency syndrome is linked to defects in 53BP1-mediated DNA damage signaling.

Stewart, Grant S; Stankovic, Tatjana; Byrd, Philip J; Wechsler, Thomas; Miller, Edward S; Huissoon, Aarn; Drayson, Mark T; West, Stephen C; Elledge, Stephen J; Taylor, A Malcolm R.

Proc Natl Acad Sci U S A; 104(43): 16910-5, 2007 Oct 23.

Artículo en Inglés | MEDLINE | ID: mdl-17940005

12.

DNA-PKcs function regulated specifically by protein phosphatase 5.

Wechsler, Thomas; Chen, Benjamin P C; Harper, Ryan; Morotomi-Yano, Keiko; Huang, Betty C B; Meek, Katheryn; Cleaver, James E; Chen, David J; Wabl, Matthias.

Proc Natl Acad Sci U S A; 101(5): 1247-52, 2004 Feb 03.

Artículo en Inglés | MEDLINE | ID: mdl-14734805

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