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Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy.
Birch, Sharla M; Lawlor, Michael W; Conlon, Thomas J; Guo, Lee-Jae; Crudele, Julie M; Hawkins, Eleanor C; Nghiem, Peter P; Ahn, Mihye; Meng, Hui; Beatka, Margaret J; Fickau, Brittany A; Prieto, Juan C; Styner, Martin A; Struharik, Michael J; Shanks, Courtney; Brown, Kristy J; Golebiowski, Diane; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Clement, Nathalie; Coleman, Kirsten E; Corti, Manuela; Pan, Xiufang; Hauschka, Stephen D; Gonzalez, J Patrick; Morris, Carl A; Schneider, Joel S; Duan, Dongsheng; Chamberlain, Jeffrey S; Byrne, Barry J; Kornegay, Joe N.
Afiliación
  • Birch SM; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
  • Lawlor MW; Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  • Conlon TJ; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
  • Guo LJ; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
  • Crudele JM; University of Washington, Seattle, WA 98109, USA.
  • Hawkins EC; College of Veterinary Medicine, North Carolina State University, Raleigh, NC 27606, USA.
  • Nghiem PP; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
  • Ahn M; University of Nevada-Reno, Reno, NV 89557, USA.
  • Meng H; Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  • Beatka MJ; Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  • Fickau BA; Medical College of Wisconsin, Milwaukee, WI 53226, USA.
  • Prieto JC; University of North Carolina, Chapel Hill, NC 27599, USA.
  • Styner MA; University of North Carolina, Chapel Hill, NC 27599, USA.
  • Struharik MJ; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Shanks C; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Brown KJ; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Golebiowski D; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Bettis AK; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
  • Balog-Alvarez CJ; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
  • Clement N; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
  • Coleman KE; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
  • Corti M; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
  • Pan X; School of Medicine, University of Missouri, Columbia, MO 65212, USA.
  • Hauschka SD; University of Washington, Seattle, WA 98109, USA.
  • Gonzalez JP; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Morris CA; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Schneider JS; Solid Biosciences Inc., Cambridge, MA 02142, USA.
  • Duan D; School of Medicine, University of Missouri, Columbia, MO 65212, USA.
  • Chamberlain JS; University of Washington, Seattle, WA 98109, USA.
  • Byrne BJ; Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA.
  • Kornegay JN; Texas A&M University, College of Veterinary Medicine and Biomedical Sciences, College Station, TX 77843, USA.
Sci Transl Med ; 15(677): eabo1815, 2023 01 04.
Article en En | MEDLINE | ID: mdl-36599002
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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne / Distrofia Muscular Animal Tipo de estudio: Clinical_trials Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2023 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Base de datos: MEDLINE Asunto principal: Distrofia Muscular de Duchenne / Distrofia Muscular Animal Tipo de estudio: Clinical_trials Idioma: En Revista: Sci Transl Med Asunto de la revista: CIENCIA / MEDICINA Año: 2023 Tipo del documento: Article